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A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia (FORWARD-FA)
To evaluate the efficacy and safety of subcutaneous nomlabofusp in adult and pediatric subjects with Friedreich's ataxia
studyfinder@utsouthwestern.edu
ALL
12 Years to 40 Years old
PHASE3
NCT07778836
Key
• Subject must provide genetically confirmed FRDA diagnosis report and is homozygous for GAA repeat expansions documented on the genetic diagnostic report, with repeat sizing (if available).
• Subject must complete 1 trial at Screening and 1 trial at Day -1 of the T25-FW test, using their customary assistive device (e.g., cane, 2 canes/crutches \[Canadian crutches\], wheeled walker/rollator or canine assistance) if needed. Each trial must be completed within 3 minutes.
• Subject must have the following at Screening and Day -1 per the following upright stability items from Module E of the mFARS:
• Item #E1, Sitting Posture - no more than a maximum score of 2
• Item #E2A, Stance Feet Apart - no more than a maximum score of 2 (average of 3 attempts)
• Subject must have an mFARS score ≥ 20 and \< 60 at Screening and Day -1.
• Subject must have a Functional Staging for Ataxia score of 4 or less at Screening.
• Subject demonstrates sufficient dexterity and visual acuity to prepare and self-administer SC injections of study drug daily (QD) or has an identified caregiver who will be trained and committed to prepare and administer the injections.
• Subject has a Screening HbA1c ≤ 7.0%.
• If the subject is taking permitted concomitant medication(s), subject must have been on a stable dose and frequency of medication(s) over the past 28 days prior to initiation of Screening. Subjects taking niacin and resveratrol must have been on a stable dose and frequency for 90 days prior to initiation of Screening and subjects taking omaveloxolone must have been on a stable dose and frequency for 1 years prior to initiation of Screening. Key Exclusion Criteria Subjects are excluded from the study if any of the following exclusion criteria are met:
• Subject who is confirmed as compound heterozygous (GAA repeat expansion on only 1 allele) for FRDA.
• Subject previously participated in a clinical trial involving nomlabofusp. Participation is defined as the subject having signed the informed consent for the study and received at least 1 dose of study drug (nomlabofusp or placebo).
• The subject has any condition, disease, or situation that could confound the results of the study or put the subject at undue risk, making participation inadvisable in the opinion of the PI.
• Women of childbearing potential who are pregnant (have a positive pregnancy test at Screening or Day -1), lactating, or planning to attempt to become pregnant during this study or within 90 days after the last dose of study drug (this includes male subjects with partners of childbearing potential who are attempting to become pregnant).
• Subject used any investigational drug or device within 90 days prior to the initiation of Screening.
• Subject previously received a gene therapy (investigational or approved) at any time in the past.
• Subject requires use of amiodarone.
• Subject used erythropoietin, etravirine, or gamma interferon within 90 days prior to the initiation of Screening.
• Subject's use of biotin supplementation exceeds 30 μg/day, either as part of a multivitamin or as a standalone supplement, within 7 days prior to the first dose of study drug. Biotin supplementation ≤ 30 μg/day is permitted if taken at a stable dose and frequency for at least 28 days prior to the initiation of Screening and there is a commitment from the subject to maintain the biotin dose throughout the study (due to interference with assays).
• Subject receives medication that requires SC injection in the abdomen or thigh.
• Subject has a Screening ECHO left ventricular ejection fraction \< 45%.
• Subject has a QTcF on an ECG as specified below:
• For subjects ≥ 12 and \< 18 years of age, a male or female subject with a QTcF \> 460 ms.
• For subjects ≥ 18 years of age, a male subject with a QTcF \> 450 ms or a female subject has a QTcF \> 470 ms.
• Subject has suicidal ideation as determined by a "yes" to item #2 on the C-SSRS at Screening (within the last 28 days) or at Day -1.
Inclusion Criteria:
Subjects who meet all of the following criteria are potentially eligible for study participation:
• Subject must provide genetically confirmed FRDA diagnosis report and is homozygous for GAA repeat expansions documented on the genetic diagnostic report, with repeat sizing (if available).
• Subject must complete 1 trial at Screening and 1 trial at Day -1 of the T25-FW test, using their customary assistive device (e.g., cane, 2 canes/crutches \[Canadian crutches\], wheeled walker/rollator or canine assistance) if needed. Each trial must be completed within 3 minutes.
• Subject must have the following at Screening and Day -1 per the following upright stability items from Module E of the mFARS:
• Item #E1, Sitting Posture - no more than a maximum score of 2
• Item #E2A, Stance Feet Apart - no more than a maximum score of 2 (average of 3 attempts)
• Subject must have an mFARS score ≥ 20 and \< 60 at Screening and Day -1.
• Subject must have a Functional Staging for Ataxia score of 4 or less at Screening.
• Subject demonstrates sufficient dexterity and visual acuity to prepare and self-administer SC injections of study drug daily (QD) or has an identified caregiver who will be trained and committed to prepare and administer the injections.
• Subject has a Screening HbA1c ≤ 7.0%.
• If the subject is taking permitted concomitant medication(s), subject must have been on a stable dose and frequency of medication(s) over the past 28 days prior to initiation of Screening. Subjects taking niacin and resveratrol must have been on a stable dose and frequency for 90 days prior to initiation of Screening and subjects taking omaveloxolone must have been on a stable dose and frequency for 1 years prior to initiation of Screening. Key Exclusion Criteria Subjects are excluded from the study if any of the following exclusion criteria are met:
• Subject who is confirmed as compound heterozygous (GAA repeat expansion on only 1 allele) for FRDA.
• Subject previously participated in a clinical trial involving nomlabofusp. Participation is defined as the subject having signed the informed consent for the study and received at least 1 dose of study drug (nomlabofusp or placebo).
• The subject has any condition, disease, or situation that could confound the results of the study or put the subject at undue risk, making participation inadvisable in the opinion of the PI.
• Women of childbearing potential who are pregnant (have a positive pregnancy test at Screening or Day -1), lactating, or planning to attempt to become pregnant during this study or within 90 days after the last dose of study drug (this includes male subjects with partners of childbearing potential who are attempting to become pregnant).
• Subject used any investigational drug or device within 90 days prior to the initiation of Screening.
• Subject previously received a gene therapy (investigational or approved) at any time in the past.
• Subject requires use of amiodarone.
• Subject used erythropoietin, etravirine, or gamma interferon within 90 days prior to the initiation of Screening.
• Subject's use of biotin supplementation exceeds 30 μg/day, either as part of a multivitamin or as a standalone supplement, within 7 days prior to the first dose of study drug. Biotin supplementation ≤ 30 μg/day is permitted if taken at a stable dose and frequency for at least 28 days prior to the initiation of Screening and there is a commitment from the subject to maintain the biotin dose throughout the study (due to interference with assays).
• Subject receives medication that requires SC injection in the abdomen or thigh.
• Subject has a Screening ECHO left ventricular ejection fraction \< 45%.
• Subject has a QTcF on an ECG as specified below:
• For subjects ≥ 12 and \< 18 years of age, a male or female subject with a QTcF \> 460 ms.
• For subjects ≥ 18 years of age, a male subject with a QTcF \> 450 ms or a female subject has a QTcF \> 470 ms.
• Subject has suicidal ideation as determined by a "yes" to item #2 on the C-SSRS at Screening (within the last 28 days) or at Day -1.
DRUG: Nomlabofusp, DRUG: Placebo
Friedreich Ataxia, Friedreich's Ataxia
FA, FRDA
A Study to Test Whether Survodutide Helps People With Type 2 Diabetes Control Their Blood Sugar
This study aims to find out whether a study medicine called survodutide helps people control their blood sugar. Adults who live with type 2 diabetes and with a body mass index (BMI) of 23 kg/m2 or higher can join. The study has 3 parts. In each part the study compares survodutide with placebo. Survodutide is being developed to treat several health problems including type 2 diabetes. Placebo looks like survodutide but does not contain any medicine. Depending on a person's diabetes treatment, a person will be assigned either to * Part A: healthy eating and physical activity * Part B: diabetes tablets (no injection of insulin) * Part C: injection of insulin (with or without diabetes tablets) Participants are randomly put into 1 of 3 groups, which means the group is chosen by chance. Two groups of participants get survodutide at different dose levels, and the third group gets placebo as injection under the skin once a week. You have a 2 in 3 chance of getting survodutide. During the study, participants continue their regular diabetes treatment. Participants are in the study for about 1 year and 2 months. During this time, they attend up to 12 visits at the site and receive at least 9 phone calls. Study doctors regularly test participants' blood sugar by checking their HbA1c values and other laboratory test results. The study doctor also regularly checks participants' health and takes note of any changes. For each study part, the results will be compared between the survodutide and the placebo group to see whether the treatment works.
studyfinder@utsouthwestern.edu
ALL
18 Years to old
PHASE3
NCT07754461
Inclusion criteria:
• Male or female, age ≥18 years at the time of signing informed consent, and at least the legal age of consent in countries where it is \>18 years
• Diagnosed with type 2 diabetes mellitus (T2D) (e.g. American diabetes association (ADA)/European association for the study of diabetes (EASD) guidelines) and must meet requirements for one of background therapy parts A, B or C:
• Part A: Naïve to insulin therapy and have not used oral or injectable anti-hyperglycaemic (diabetes) medication for at least 90 days prior to screening (Visit 1) and glycosylated haemoglobin A1c (HbA1c) ≥7.0 (≥53 mmol/mol) and \<9.5% (\<80 mmol/mol) as measured by the central laboratory at screening (Visit 1);
• Part B: Treated with stable dose of oral antidiabetic medication (OAD) for 90 days prior to screening (Visit 1) as monotherapy or combination of: Maximum tolerated dose of metformin and/or Maximum tolerated dose of sulfonylurea and/or Maximum tolerated dose of sodium-glucose cotransporter 2 inhibitor (SGLT2i) and HbA1c ≥7.0% (≥53 mmol/mol) and \<10.5% (\<91 mmol/mol) as measured by the central laboratory at screening (Visit 1);
• Part C: Stable use of basal insulin at a dose of ≥20 international units (IU)/day with or without stable dose of metformin and/or SGLT2i for 90 days prior to screening (Visit 1) and HbA1c ≥7.0% (≥53 mmol/mol) and \<10.5% (\<91 mmol/mol) as measured by the central laboratory at screening (Visit 1)
• Body mass index (BMI) ≥23 kg/m2 at screening (Visit 1)
• Signed and dated written informed consent in accordance with international council for harmonisation - good clinical practice (ICH-GCP) and local legislation prior to admission to the trial
• Woman (or women) of childbearing potential (WOCBP) must be ready and able to use highly effective methods of birth control per international council for harmonisation of technical requirements for pharmaceuticals for human use M3 (R2) that result in a low failure rate of less than 1% per year when used consistently and correctly.
• In the investigator's opinion, participants are well-motivated, capable, and willing to: Learn how to self-inject the investigational medicinal product (IMP), as required for this protocol (persons with physical limitations who are not able to perform the injections must have the assistance of an individual trained to inject the IMP); Inject the IMP or accept injection from a designated person; Follow study procedures for the duration of the study, including, but not limited to: follow lifestyle advice (for example, dietary restrictions and exercise plan), maintain a diary, complete required questionnaires, and handle the IMP as described in the instructions for use (IFU) Exclusion criteria:
• Type 1 diabetes mellitus (T1D) or latent autoimmune diabetes in adults (LADA)
• Treatment within 90 days before screening (Visit 1) up to and including randomisation (Visit 2) with any: glucagon-like peptide-1 receptor (GLP-1R) agonists including GLP-1R agonist/glucose-dependent insulinotropic polypeptide (GIP) combinations, amylin or incretin combinations; Glucose-lowering drugs other than stated in the inclusion criteria for each part (i.e. metformin, Sodium-Glucose Cotransporter 2 Inhibitor (SGLT2i), basal insulin, depending on the background therapy part); Glucose-lowering investigational drug; Any anti-obesity medication including bupropion/naltrexone, orlistat, phentermine, and phentermine/topiramate
• History of ketoacidosis or hyperosmolar state or coma within the last 6 months before screening (Visit 1) up to and including randomisation (Visit 2)
• Hypoglycaemia unawareness or a history of severe hypoglycaemia within the last 3 months before screening (Visit 1) up to and including randomisation (Visit 2)
• Diabetic retinopathy or maculopathy currently under treatment, or that is reasonably anticipated to require such treatment over the duration of the study
• Body weight change (self-reported) \>5% within 90 days before screening (Visit 1)
• Previous or planned (during the trial period) treatment for obesity with surgery or a weight loss device, including any prior bariatric surgery. The following are allowed: (1) liposuction and/or abdominoplasty, if performed \>1 year before screening (Visit 1), (2) lap banding, if the band has been removed \>1 year before screening (Visit 1), (3) intragastric balloon, if the balloon has been removed \>1 year before screening (Visit 1), (4) duodenal-jejunal bypass sleeve, if the sleeve has been removed \>1 year before screening (Visit 1).
• Answered "yes" to any of the suicide-related behaviours (Actual attempt, Interrupted attempt, Aborted attempt, Preparatory act or behaviour) or to the non-suicidal self-injurious behaviour question on the "Suicidal Behaviour" section of the Columbia-Suicide Severity Rating Scale (C-SSRS) related to the past 2 years before screening (Visit 1) up to and including randomisation (Visit 2)
• Further exclusion criteria apply.
• Male or female, age ≥18 years at the time of signing informed consent, and at least the legal age of consent in countries where it is \>18 years
• Diagnosed with type 2 diabetes mellitus (T2D) (e.g. American diabetes association (ADA)/European association for the study of diabetes (EASD) guidelines) and must meet requirements for one of background therapy parts A, B or C:
• Part A: Naïve to insulin therapy and have not used oral or injectable anti-hyperglycaemic (diabetes) medication for at least 90 days prior to screening (Visit 1) and glycosylated haemoglobin A1c (HbA1c) ≥7.0 (≥53 mmol/mol) and \<9.5% (\<80 mmol/mol) as measured by the central laboratory at screening (Visit 1);
• Part B: Treated with stable dose of oral antidiabetic medication (OAD) for 90 days prior to screening (Visit 1) as monotherapy or combination of: Maximum tolerated dose of metformin and/or Maximum tolerated dose of sulfonylurea and/or Maximum tolerated dose of sodium-glucose cotransporter 2 inhibitor (SGLT2i) and HbA1c ≥7.0% (≥53 mmol/mol) and \<10.5% (\<91 mmol/mol) as measured by the central laboratory at screening (Visit 1);
• Part C: Stable use of basal insulin at a dose of ≥20 international units (IU)/day with or without stable dose of metformin and/or SGLT2i for 90 days prior to screening (Visit 1) and HbA1c ≥7.0% (≥53 mmol/mol) and \<10.5% (\<91 mmol/mol) as measured by the central laboratory at screening (Visit 1)
• Body mass index (BMI) ≥23 kg/m2 at screening (Visit 1)
• Signed and dated written informed consent in accordance with international council for harmonisation - good clinical practice (ICH-GCP) and local legislation prior to admission to the trial
• Woman (or women) of childbearing potential (WOCBP) must be ready and able to use highly effective methods of birth control per international council for harmonisation of technical requirements for pharmaceuticals for human use M3 (R2) that result in a low failure rate of less than 1% per year when used consistently and correctly.
• In the investigator's opinion, participants are well-motivated, capable, and willing to: Learn how to self-inject the investigational medicinal product (IMP), as required for this protocol (persons with physical limitations who are not able to perform the injections must have the assistance of an individual trained to inject the IMP); Inject the IMP or accept injection from a designated person; Follow study procedures for the duration of the study, including, but not limited to: follow lifestyle advice (for example, dietary restrictions and exercise plan), maintain a diary, complete required questionnaires, and handle the IMP as described in the instructions for use (IFU) Exclusion criteria:
• Type 1 diabetes mellitus (T1D) or latent autoimmune diabetes in adults (LADA)
• Treatment within 90 days before screening (Visit 1) up to and including randomisation (Visit 2) with any: glucagon-like peptide-1 receptor (GLP-1R) agonists including GLP-1R agonist/glucose-dependent insulinotropic polypeptide (GIP) combinations, amylin or incretin combinations; Glucose-lowering drugs other than stated in the inclusion criteria for each part (i.e. metformin, Sodium-Glucose Cotransporter 2 Inhibitor (SGLT2i), basal insulin, depending on the background therapy part); Glucose-lowering investigational drug; Any anti-obesity medication including bupropion/naltrexone, orlistat, phentermine, and phentermine/topiramate
• History of ketoacidosis or hyperosmolar state or coma within the last 6 months before screening (Visit 1) up to and including randomisation (Visit 2)
• Hypoglycaemia unawareness or a history of severe hypoglycaemia within the last 3 months before screening (Visit 1) up to and including randomisation (Visit 2)
• Diabetic retinopathy or maculopathy currently under treatment, or that is reasonably anticipated to require such treatment over the duration of the study
• Body weight change (self-reported) \>5% within 90 days before screening (Visit 1)
• Previous or planned (during the trial period) treatment for obesity with surgery or a weight loss device, including any prior bariatric surgery. The following are allowed: (1) liposuction and/or abdominoplasty, if performed \>1 year before screening (Visit 1), (2) lap banding, if the band has been removed \>1 year before screening (Visit 1), (3) intragastric balloon, if the balloon has been removed \>1 year before screening (Visit 1), (4) duodenal-jejunal bypass sleeve, if the sleeve has been removed \>1 year before screening (Visit 1).
• Answered "yes" to any of the suicide-related behaviours (Actual attempt, Interrupted attempt, Aborted attempt, Preparatory act or behaviour) or to the non-suicidal self-injurious behaviour question on the "Suicidal Behaviour" section of the Columbia-Suicide Severity Rating Scale (C-SSRS) related to the past 2 years before screening (Visit 1) up to and including randomisation (Visit 2)
• Further exclusion criteria apply.
DRUG: Placebo, DRUG: Survodutide
Type 2 Diabetes
A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in Pediatric Participants
The primary purpose of the study is to evaluate the efficacy of olezarsen administered by subcutaneous injection to pediatric participants with FCS.
studyfinder@utsouthwestern.edu
ALL
2 Years to 17 Years old
PHASE3
NCT07727538
Key
• Parental or legally authorized representative consent must be obtained, and the participants must provide age-appropriate or cognition-appropriate assent, as determined by the Investigator. The parent or legal guardian must be able to understand and comply with the study visit schedule and all other study procedures.
• Must be able to comply with all study procedures.
• Age 12 to less than 18 years at the time of informed consent/assent (Cohort 1); age 2 to less than 12 years at time of informed consent/assent (Cohort 2).
• Willing to fast for at least 10 hours before visits requiring fasted blood sampling.
• A diagnosis of Familial Chylomicronemia Syndrome (type 1 Hyperlipoproteinemia) by documentation of confirmed homozygote, compound heterozygote or double heterozygote for loss-of-function mutations in type 1-causing genes.
• Fasting TGs ≥880 mg/dL at screening. If fasting TG is \< 880 mg/dL, up to two additional tests may be performed during the screening period with any single test used to qualify. Key
• Diabetes mellitus with any of the following:
• Newly diagnosed within 12 weeks prior to screening or during the screening period.
• Hemoglobin A1c (HbA1c) ≥9.5% at screening.
• Change in basal insulin regimen \>20% within 3 months prior to screening or during the screening period.
• Type 1 diabetes.
• History of bleeding, diathesis, or coagulopathy.
• Major surgery within 3 months of screening.
• Plasma apheresis within 4 weeks prior to screening or planned during the study.
• Treatment with another investigational drug, biological agent, or device within one month of screening, or 5 half-lives of investigational agent, whichever is longer.
• Active pancreatitis within 4 weeks prior to screening or during the screening period.
• Malignancy diagnosed or treated within 5 years prior to screening or during the screening period. Note: Other protocol-specified inclusion/exclusion criteria may apply.
Inclusion Criteria:
• Parental or legally authorized representative consent must be obtained, and the participants must provide age-appropriate or cognition-appropriate assent, as determined by the Investigator. The parent or legal guardian must be able to understand and comply with the study visit schedule and all other study procedures.
• Must be able to comply with all study procedures.
• Age 12 to less than 18 years at the time of informed consent/assent (Cohort 1); age 2 to less than 12 years at time of informed consent/assent (Cohort 2).
• Willing to fast for at least 10 hours before visits requiring fasted blood sampling.
• A diagnosis of Familial Chylomicronemia Syndrome (type 1 Hyperlipoproteinemia) by documentation of confirmed homozygote, compound heterozygote or double heterozygote for loss-of-function mutations in type 1-causing genes.
• Fasting TGs ≥880 mg/dL at screening. If fasting TG is \< 880 mg/dL, up to two additional tests may be performed during the screening period with any single test used to qualify. Key
Exclusion Criteria:
• Diabetes mellitus with any of the following:
• Newly diagnosed within 12 weeks prior to screening or during the screening period.
• Hemoglobin A1c (HbA1c) ≥9.5% at screening.
• Change in basal insulin regimen \>20% within 3 months prior to screening or during the screening period.
• Type 1 diabetes.
• History of bleeding, diathesis, or coagulopathy.
• Major surgery within 3 months of screening.
• Plasma apheresis within 4 weeks prior to screening or planned during the study.
• Treatment with another investigational drug, biological agent, or device within one month of screening, or 5 half-lives of investigational agent, whichever is longer.
• Active pancreatitis within 4 weeks prior to screening or during the screening period.
• Malignancy diagnosed or treated within 5 years prior to screening or during the screening period. Note: Other protocol-specified inclusion/exclusion criteria may apply.
DRUG: Olezarsen
Familial Chylomicronemia Syndrome
Perioperative FLOT(D) With Personalized Ultrafractionated Stereotactic Adaptive Radiotherapy (PULSAR) in Esophageal Cancer
The goal of this clinical trial is to learn the safety of the addition of PULSAR radiation to standard of care chemotherapy and immunotherapy in people with esophageal and gastroesophageal junction cancer and to determine the safest dose of radiation that can be used. Participants in this study will be undergoing clinically scheduled procedures. In addition to the standard of care visits and treatments, there will be additional visits and assessments with radiation.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Shahed Badiyan
ALL
18 Years to old
PHASE1
NCT07700420
STU20250653
Inclusion Criteria:
• At least 18 years of age at date of enrollment. Both men and women and members of all races and ethnic groups will be included.
• Willing and able to provide written informed consent.
• Pathologic diagnosis of esophageal or gastroesophageal junction adenocarcinoma. GEJ cancer includes Siewart types 1 and 2 tumors. Siewart type 3 is also eligible as long as the patient is intended to be treated in the same way as for type 1 and 2 tumors (i.e. candidate for FLOT-(D), resectable, amenable to radiation)
• T1N+ or T2-4N(any) by the American Joint Committee on Cancer staging manual 8th edition
• Primary tumor (and lymph nodes) that appear to be resectable in the opinion of an experienced thoracic surgeon or surgical oncologist 19.
• Measurable disease by RECIST 1.1 is not a requirement in order to enroll on this study. Those who have measurable disease at baseline will be followed by RECIST 1.1 criteria at time of restaging.
• Primary tumor (and applicable nodal sites) that appears amenable to radiation in the opinion of an experienced radiation oncologist.
• Eastern cooperative Oncology Group (ECOG) performance status of 0-1
• No prior systemic treatment or radiation for esophageal/GEJ adenocarcinoma. Patients who have received prior endoscopic therapies with subsequent recurrence necessitating curative-intent surgery will be eligible for enrollment.
• Adequate organ and marrow function as defined below:
• 0 ANC ≥1500/mL 10.1 Platelet ≥100,000/mL 10.2 Total Bilirubin ≤ 1.0 x the upper limit of normal (ULN). This will not apply to patients with confirmed Gilbert's syndrome, who will be allowed in consultation with their physician.
• 3 AST and/or ALT both ≤ 1.5x ULN with alkaline phosphatase ≤2.5x ULN 10.4 Creatinine Clearance ≥ 30 mL/min by Cockroft Gault
• All men, as well as women of child-bearing potential (WOCBP) must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) starting with the first dose of radiation through 120 days after completion of adjuvant chemotherapy or immunotherapy. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately. 11a. A woman of child-bearing potential is any woman (regardless of sexual orientation, marital status, having undergone a tubal ligation, or remaining celibate by choice) who meets the following criteria: * Has not undergone a hysterectomy or bilateral oophorectomy; or * Has not been naturally postmenopausal for at least 12 consecutive months (i.e., has had menses at any time in the preceding 12 consecutive months).
Exclusion Criteria:
• Patients with adenosquamous cell carcinoma, squamous cell carcinoma, GI stromal tumor, or neuroendocrine tumor (any grade) of the esophagus or GEJ.
• Receipt of prior chemotherapy, radiotherapy, or both for esophageal or gastroesophageal junction cancer (any type).
• Any other active malignancies besides localized skin cancers or in situ carcinomas. Patients with localized prostate or breast cancers who have been stable for ≥ 6 months on adjuvant therapy without evidence of active disease will be eligible to enroll on this study. Patients with a history of cancer that has not been active in the last 5 years may be included but only after consultation with the principal investigator.
• Prior RT to the chest or abdomen (not esophagus/GEJ) that would, in the opinion of a radiation oncologist, limit the safety of PULSAR.
• Known dihydropyrimidine dehydrogenase (DPYD) intermediate or poor metabolizer phenotype where administration of full dose 5-fluoruracil would be prohibitively toxic. It is not a requirement to test DPYD deficiency prior to enrollment. Patients found to be DPYD intermediate or poor metabolizers through the course of standard of care evaluation will be removed from the protocol and followed for DLTs. These patients will be replaced.
• Uncontrolled comorbid illness or condition including congestive heart failure, unstable angina, cardiac arrhythmia, or psychiatric illness that would limit compliance with study requirements or that would be prohibitive of a surgical resection in the opinion of a surgical oncologist or thoracic surgeon.
• Chemotherapy, or other systemic cancer therapy for non-esophageal or GEJ cancer within 52 weeks prior to starting study treatment (with the exception of long-term adjuvant therapy for curative-intent prostate or breast cancer, as defined above).
• History of allergic reactions attributed to compounds of similar chemical or biologic composition to FLOT chemotherapy, durvalumab or other agents used in study.
• Subjects must not be pregnant or nursing due to the potential for congenital abnormalities and the potential of this regimen to harm nursing infants.
• Unwilling or unable to undergo placement of chest wall mediport for chemotherapy administration.
• Any autoimmune condition requiring immunosuppression beyond physiologic doses of steroids will not be eligible for receipt of durvalumab. Patient would still be eligible for enrollment for receipt of perioperative FLOT only and can enroll on this study while receiving FLOT.
• Requirement of prednisone 10 mg (or prednisone equivalent) or more daily for any reason (Inhalational steroids for chronic obstructive pulmonary disease, asthma, or the like, is allowable). Patient would still be eligible for enrollment for receipt of perioperative FLOT only if they are meeting all other eligibility criteria.
• Judgment by the Investigator that the patient should not participate in the study if the patient is unlikely to comply with study procedures, restrictions, and requirements.
RADIATION: PULSAR- Personalized Ultra-fractionated Stereotactic Ablative Radiotherapy, DRUG: FLOT (Fluorouracil+Leucovorin+Oxaliplatin+Docetaxel)(D)
Gastro-esophageal Junction (GEJ) Cancer, Esophagus Cancer, Esophagus, Other Digestive Organ
UT Southwestern
Privosegtor Investigation in Optic Neuropathies Efficacy Evaluation Research (PIONEER-1)
The goal of this clinical trial is to evaluate the safety and efficacy of privosegtor, a neuroprotective candidate, in patients diagnosed with optic neuritis (ON). Researchers will compare privosegtor and the standard of care (methylprednisolone) to a placebo and standard of care (methylprednisolone).
Call 214-648-5005
studyfinder@utsouthwestern.edu, Stephanie.Morales@UTSouthwestern.edu
Ruben Jauregui
ALL
18 Years to 50 Years old
PHASE3
NCT07623668
STU20260769
Key
Inclusion Criteria:
* Adult men and women (aged 18 to 50 years) with the first episode of ON in the study eye, associated with unilateral visual loss.
* Onset of visual loss symptoms in the previous 12 days before first administration of study treatment.
Key Exclusion Criteria:
\- Have a history or presence of any disorder or condition that may, in the opinion of the Investigator, likely interfere with the interpretation of the study results or participant safety DRUG: Privosegtor, DRUG: Methylprednisolone, OTHER: Placebo
Optic Neuritis
Optic Neuritis, ON, AON, Optic Neuropathy, Multiple Sclerosis, MS, Vision Loss, LCVA, Low Contrast Visual Acuity, Low Contrast Sensitivity, Privosegtor, OCS-05
UT Southwestern
TG-INSIGHT With Joint POCUS, Hemostatic Potential in Patients With Severe Hemophilia A on Novel Replacement and Substitution FVIII Therapies
This is an observational research study to find out if there is a difference in the way children with moderate or severe hemophilia A, treated on two different types of factor replacement, form a clot and also evaluate if they develop tiny bleeds within the joint and subsequently early joint changes when receiving extended half-life factor VIII.
studyfinder@utsouthwestern.edu
ALL
6 Months to old
NCT07692217
Inclusion Criteria:
* Participants with moderate or severe HA who are on prophylaxis with either weekly, biweekly, or every 4-weeks emicizumab or weekly efanesoctocog alfa for at least 2 months.
* \>6 months of age
Exclusion Criteria:
* Participants with active FVIII inhibitor (\>0.5 BU/mL)
* Presence of an additional bleeding disorder other than hemophilia A DRUG: Half-life factor VIII based replacement therapy, DRUG: Non-FVIII based replacement therapy
Hemophilia A, Factor VIII (FVIII)
A Phase 2, Open-Label, Single-Arm Trial of FT819 in Participants With Lupus Nephritis (RECLAIM-LN)
The primary objective of this trial is to evaluate the efficacy and safety of FT819, comprised of allogeneic T cells that express a CD19-targeted CAR, following bendamustine administration in participants with refractory moderate-to-severe lupus nephritis, as assessed by the proportion of participants who achieve complete renal response (CRR) at Week 26.
Call 214-648-5005
studyfinder@utsouthwestern.edu, ivan.sy@childrens.com
Samuel John
ALL
12 Years to 70 Years old
PHASE2
NCT07570862
STU20260716
INCLUSION CRITERIA:
* Age ≥12 to ≤70 years
* Diagnosis of SLE per EULAR/ACR 2019 classification criteria
* Biopsy-proven proliferative Class III or IV LN, with or without concomitant Class V involvement, based on the 2003/2018 ISN/RPS classification
* Positivity for at least one of the following autoantibodies at screening:
• Antinuclear antibody (ANA)
• Anti-double-stranded DNA (anti-dsDNA) or
• Anti-Smith antibody * Active disease, defined as: a. Evidence of SLE activity, defined as either: i. SLEDAI-2K ≥6 or ii. At least 1 BILAG A or 2 BILAG B scores for SLE-related organ involvement; and b. Evidence of renal involvement, defined as UPCr ≥1 g/g; and c. Moderate-to-severe renal disease with investigator's impression that improvement is possible * Refractory to ≥2 systemic immunosuppressive therapies for the treatment of LN EXCLUSION CRITERIA: * Evidence of inadequate organ function during the screening period * Active central nervous system (CNS) symptoms attributable to autoimmune disease within 12 months prior to trial intervention * History of or current renal diseases (other than LN) that, in the opinion of the investigator, could interfere with assessment of LN or confound evaluation of disease activity * Receipt of dialysis (hemodialysis or peritoneal dialysis) within 12 weeks of trial intervention * Irreversible organ damage related to underlying disease (e.g., ESRD) where, in the opinion of the investigator, CD19 CAR T-cell therapy would be unlikely to benefit the participant * History of malignancy in the prior 5 years * Known allergy to the following FT819 components: albumin (human) or DMSO * History of intolerance or contraindication to bendamustine * Body weight \<30 kg * Any medical condition, clinical laboratory abnormality, or nonmedical/social issue that, per investigator or medical monitor judgement, precludes safe participation in and completion of the trial or that could affect compliance with protocol conduct or interpretation of results
• Antinuclear antibody (ANA)
• Anti-double-stranded DNA (anti-dsDNA) or
• Anti-Smith antibody * Active disease, defined as: a. Evidence of SLE activity, defined as either: i. SLEDAI-2K ≥6 or ii. At least 1 BILAG A or 2 BILAG B scores for SLE-related organ involvement; and b. Evidence of renal involvement, defined as UPCr ≥1 g/g; and c. Moderate-to-severe renal disease with investigator's impression that improvement is possible * Refractory to ≥2 systemic immunosuppressive therapies for the treatment of LN EXCLUSION CRITERIA: * Evidence of inadequate organ function during the screening period * Active central nervous system (CNS) symptoms attributable to autoimmune disease within 12 months prior to trial intervention * History of or current renal diseases (other than LN) that, in the opinion of the investigator, could interfere with assessment of LN or confound evaluation of disease activity * Receipt of dialysis (hemodialysis or peritoneal dialysis) within 12 weeks of trial intervention * Irreversible organ damage related to underlying disease (e.g., ESRD) where, in the opinion of the investigator, CD19 CAR T-cell therapy would be unlikely to benefit the participant * History of malignancy in the prior 5 years * Known allergy to the following FT819 components: albumin (human) or DMSO * History of intolerance or contraindication to bendamustine * Body weight \<30 kg * Any medical condition, clinical laboratory abnormality, or nonmedical/social issue that, per investigator or medical monitor judgement, precludes safe participation in and completion of the trial or that could affect compliance with protocol conduct or interpretation of results
BIOLOGICAL: FT819
Lupus Nephritis, Systemic Lupus Erythematosus, SLE - Systemic Lupus Erythematosus, Lupus Nephritis - WHO Class III, Lupus Nephritis - WHO Class IV
FT819, Fate Therapeutics, Allogeneic CAR T, CD19 - targeted therapy, Lupus Nephritis, Systemic Lupus Erythematosus
Children’s Health
ReneuRx™ (Nerve Selective Therapy) Utilized in Lateral Hip Pain (RENEU)
The goal of this clinical trial is to evaluate the safety and analgesic performance of the ReneuRx™ device in subjects with moderate to severe chronic lateral hip pain compared to subjects receiving treatment with corticosteroid injection in adults aged 22-80 years. Participants will attend study visits and complete quality of life questionnaires. Participants will be followed for approximately 6-12 months after the study procedure.
Jonathan Thompson jonathan.thompson@utsouthwestern.edu
ALL
22 Years to 80 Years old
NA
NCT07568015
Inclusion Criteria:
• Age 22 to 80, inclusive of any gender
• Baseline pain intensity of \>5 of the Numeric Rating Scale (NRS) localized to the lateral aspect of the hip (with or without radiation to lateral thigh or buttock) despite current treatment
• Tenderness to palpation of the peritrochanteric space of the lateral hip in adults with Greater Trochanteric Pain Syndrome.
• At least 6 months of previous conservative treatments for subject's lateral hip pain (NSAID, acetaminophen, physical therapy, and/or cortisone injections) that are not currently providing relief
• Agree to see study investigator and study team for hip pain during the study period
• Willing/able to understand the informed consent form and provide written informed consent
• Able to complete outcome measures (including electronic patient reported outcome measures)
Exclusion Criteria:
• Known allergy to glycerol, hyaluronic acid, poloxamer 407, or phosphate buffered saline
• History of cryoglobulinemia
• History of cold-induced auto-immune hemolytic anemia (e.g. paroxysmal cold hemoglobinuria or cold agglutinin disease)
• History of cold urticaria
• History of Chilblain's (pernio) disease in the lower extremities
• History of Raynaud's disease
• Open and/or infected wounds or active tumor at or near the treatment site
• History of vascular surgery involving femoral vessels on theinjection side
• History of hip surgery of any kind to the index side within the past 6 months (e.g., arthroscopy, arthroplasty, etc.)
• History of trochanteric osteotomy, femoral osteotomy, amputation, or pseudoarthrosis.
• Active bacterial or fungal infection that, at the discretion of the investigator, would preclude study participation
• Currently taking \>60 MME/day
• History of systemic inflammatory conditions such as rheumatoid arthritis
• Bleeding disorders, unless appropriately stopped or reversed for the procedure at investigator's discretion
• Any neuropathic pain condition (e.g. - complex regional pain syndrome, lumbar radiculopathy) in the area of pain
• Presence of unstable psychiatric disease
• Cryoneurolysis, thermal or pulsed radiofrequency ablation, or phenol injection for the index hip(s) within the past 12 months
• Corticosteroid injection within the previous 3 months or orthobiologic therapy such as platelet rich plasma injections, or shockwave therapy for the index hip(s) within the previous 6 months
• Known contraindication to use of a regional anesthetic block
• Pregnant, nursing, or intent of becoming pregnant during the study period
• Documented evidence of an overlapping pain pattern from another source that the Investigator believes to be indistinguishable from lateral hip pain
• Lumbar or sacroiliac fusion within the previous 6 months
• Any other condition (such as history of deep vein thrombosis, significant cardiovascular, renal failure/dialysis, hepatic or other systemic comorbidity/chronic pain condition including cancer) or circumstance that, in the opinion of the investigator, would compromise the safety of the subject or the quality of study data
• Body habitus/hip anatomy that would preclude the use of the product injection needle size
• Participation in any clinical study of a therapeutic investigational product within 30 days prior to enrollment
• Pending litigation or disability status
• Unwilling to refrain from any scheduled surgeries that would impact the collection of study endpoint data during the duration of the study
• Subject is a prisoner
DRUG: Triamcinolone acetate, DEVICE: ReneuRx
Greater Trochanteric Pain Syndrome, Greater Trochanteric Pain Syndrome of Both Lower Limbs, Lateral Hip Pain
RENEU, ReneuRx™, ReneuRx, GTPS
AMAZE 2: A Research Study Investigating How Well the Medicine NNC0487-0111 Helps People With Excess Body Weight and Type 2 Diabetes Lose Weight (AMAZE 2)
The purpose of this clinical study is to find out if NNC0487-0111 is safe and effective for treating people who have excess body weight and type 2 diabetes. There are 2 study treatments in this study taken as injections under the skin once a week. Participants will either get NNC0487-0111 (the treatment being tested) or Placebo (treatment that has no active medicine in it). Which treatment participants get is decided by chance.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Rama.Mortada@UTSouthwestern.edu
Ildiko Lingvay
ALL
18 Years to old
PHASE3
NCT07533175
STU20252461
Inclusion Criteria:
* Male or female (sex at birth).
* Age 18 years or above at the time of signing informed consent.
* Diagnosed with type 2 diabetes mellitus more than equal to (≥) 180 days before screening.
* Treatment with lifestyle intervention, and/or 0-3 marketed oral antidiabetic drugs (OAD)s (metformin, α-glucosidase inhibitors (AGI), glinides, sodium-glucose cotransporter 2 inhibitor (SGLT2i), thiazolidinediones, or sulfonylureas (SU) as a single agent or in combination) according to local label. Treatment with oral antidiabetic drugs should be stable (same drug(s), dose and dosing frequency) before screening.
* Haemoglobin A1c (HbA1c) 7-10% \[53-86 (millimoles per mole) mmol/mol\] (both inclusive) as measured by the central laboratory at screening.
Exclusion Criteria:
* Renal impairment with estimated Glomerular Filtration Rate (eGFR) less than (\<) 30 milliliter per minute per meter square (mL/min/1.73 m\^2) \[2021 Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula\], at screening.
* Participant with diabetic retinopathy or maculopathy who received treatment with retinal photocoagulation, vitrectomy or anti-Vascular Endothelial Growth Factor (anti-VEGF) before screening or are expected to require treatment after screening. Diabetic retinopathy or maculopathy must be verified by an eye examination performed within 90 days before screening or in the period between screening and randomisation. Pharmacological pupil-dilation is a requirement unless using a digital fundus photography camera specified for non-dilated examination.
* Known hypoglycaemic unawareness as indicated by the investigator according to Clarke's questionnaire question 8.
* Recurrent severe hypoglycaemic episodes within the last year as judged by the investigator.
* Treatment with glucagon-like peptide-1 (GLP-1) receptor agonists (RA), dual GLP-1/gastric inhibitory peptide (GIP) RAs (or any other GLP-1 based treatment), or amylin analogues before screening. DRUG: NNC0487-0111, DRUG: Placebo (matched to NNC0487-0111)
Diabetes Mellitus, Overweight, Obesity
UT Southwestern
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia (ENERGIZEKids)
The primary objective of this study is to compare the effect of mitapivat versus placebo on anemia in pediatric participants with alpha- or beta-non-transfusion-dependent thalassemia.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Laurie.Rodgers-Augustyniak@childrens.com
Kathryn Dickerson
ALL
1 Year to 17 Years old
PHASE3
NCT07517133
STU20260644
Inclusion Criteria:
* Written informed consent/assent from the participant (or their legally authorized representative, parent(s), or legal guardian) must be obtained before any study-related procedures are conducted and participants must be willing to comply with all study procedures for the duration of the study.
* Aged 1 to \<18 years and weighing at least 7 kilograms (kg) at the time of providing informed consent/assent.
* Documented diagnosis of thalassemia (β-thalassemia with or without α-globin gene mutations, hemoglobin E (HbE)/β-thalassemia, or α-thalassemia/hemoglobin H (HbH) disease) based on Hemoglobin (Hb) electrophoresis, Hb high-performance liquid chromatography, and/or DNA analysis from the participant's medical record. If this information is not available from the participant's medical record, the test(s) can be performed by a local laboratory during the Screening Period. If a local laboratory is unable to perform the test(s), results from the comprehensive α- and β-globin genotyping performed by the study central laboratory can be used.
* Hb concentration ≤10.0 grams per deciliter (g/dL) \[100.0 grams per liter (g/L)\], based on an average of at least 2 Hb concentration measurements (separated by ≥7 days) collected during the Screening Period.
* Non-transfusion dependent, defined as ≤5 transfusion episodes (also referred to as "transfusion events") during the 24-week period before randomization and no red blood cells (RBC) transfusions ≤8 weeks before providing informed consent/assent and no RBC transfusions during the Screening Period.
* If taking hydroxyurea, the hydroxyurea dose must be stable for ≥16 weeks before randomization.
* Female participants who have attained menarche must be abstinent of sexual activities that may induce pregnancy as part of their usual lifestyle, or agree to use 2 forms of contraception, 1 of which must be considered highly effective, from the time of informed consent/assent, throughout the study, and for 28 days after the last dose of study drug. The second form of contraception can include an acceptable barrier method.
Exclusion Criteria:
* Pregnant or breastfeeding.
* Documented history of homozygous or heterozygous hemoglobin S (HbS) or hemoglobin C (HbC).
* Prior exposure to gene therapy or prior bone marrow or stem cell transplantation, including any prior exposure to myeloablative chemotherapy.
* Any conditions other than thalassemia expected to affect sexual maturation.
* Currently receiving treatment with luspatercept; the last dose must have been administered ≥18 weeks before randomization.
* Currently receiving treatment with hematopoietic stimulating agents; the last dose must have been administered ≥18 weeks before randomization.
* History of malignancy (active or treated) ≤5 years before providing informed consent/assent, except for nonmelanomatous skin cancer in situ, cervical carcinoma in situ, or breast carcinoma in situ.
* History of active and/or uncontrolled cardiac or pulmonary disease or clinically relevant QT prolongation within 6 months before providing informed consent/assent.
* Hepatobiliary disorders, including but not limited to:
* Liver disease with histopathological evidence or clinical diagnosis of cirrhosis or severe fibrosis.
* Clinically symptomatic cholelithiasis or cholecystitis (prior cholecystectomy is not exclusionary).
* History of drug-induced cholestatic hepatitis.
* Aspartate Aminotransferase (AST) \>2.5\*Upper Limit of Normal (ULN) (unless due to hemolysis and hepatic iron deposition) and Alanine Transaminase (ALT) \>2.5\*ULN (unless due to hepatic iron deposition).
* Renal dysfunction as defined by an estimated glomerular filtration rate \<60 milliliters per minute (mL/min)/1.73-meter square (m\^2).
* Nonfasting triglycerides \>215 milligrams per deciliter (mg/dL) \[5 millimole per liter (mmol/L)\].
* Active infection requiring systemic antimicrobial therapy at the time of providing informed consent/assent. If antimicrobial therapy is required during the Screening Period, screening procedures should not be performed while antimicrobial therapy is being administered, and the last dose of antimicrobial therapy must be administered ≥7 days before randomization.
* Participants with known active hepatitis B or hepatitis C virus infection.
* Participants with known human immunodeficiency virus (HIV) infection.
* History of major surgery (including splenectomy) ≤16 weeks before providing informed consent/assent and/or a major surgical procedure planned during the study.
* Current enrollment or past participation (within ≤12 weeks or a timeframe equivalent to 5 half-lives of the investigational study drug before administration of the first dose of study drug, whichever is longer) in any other clinical study involving an investigational treatment or device.
* Receiving strong Cytochrome3A4/5 (CYP3A4/5) inhibitors that have not been stopped for ≥5 days or a timeframe equivalent to 5 half-lives (whichever is longer), or strong CYP3A4/5 inducers that have not been stopped for ≥4 weeks or a timeframe equivalent to 5 half-lives (whichever is longer), before randomization.
* Receiving anabolic steroids that have not been stopped for at least 4 weeks before randomization. Testosterone replacement therapy to treat hypogonadism is allowed; the testosterone dose and preparation must be stable for ≥12 weeks before randomization.
* Known allergy, or other contraindication, to mitapivat or its excipients (microcrystalline cellulose, croscarmellose sodium, sodium stearyl fumarate, mannitol, magnesium stearate, and the Opadry filmcoat \[hypromellose, titanium dioxide, lactose monohydrate triacetin, and FD\&C Blue #2\]).
* Any medical, hematological, psychological, or behavioral condition(s) or prior or current therapy that, in the opinion of the Investigator, may confer an unacceptable risk to participating in the study and/or could confound the interpretation of the study data. Also excluded are:
* Participants who are institutionalized by regulatory or court order.
* Participants with any condition(s) that could create undue influence (including but not limited to incarceration, involuntary psychiatric confinement, and financial or familial affiliation with the Investigator or Sponsor). DRUG: Mitapivat, DRUG: Placebo Matching Mitapivat
Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
Children’s Health
Immune Modulation During Palynziq® Treatment in Adults (IMPALA)
Study 165-401 is a Phase 4, open-label study designed to examine the concomitant use of methotrexate (MTX) to suppress immune responses to Palynziq and improve tolerability and efficacy in adults with PKU.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Juana.Luevano@UTSouthwestern.edu
Markey McNutt
ALL
18 Years to 65 Years old
PHASE4
NCT07477691
STU20252279
Inclusion Criteria:
* Adults between 18 and 65 years old
* Have a confirmed diagnosis of phenylketonuria (PKU)
* Are in generally good health based on medical evaluation
* Are willing and medically eligible to receive Palynziq and methotrexate (MTX) Cohort A: Have never taken Palynziq before and are willing to start it during the study Cohort B: Have blood \> 600 μmol/L after taking Palynziq for at least 24 weeks, are on a daily dose of at least 20mg and unable to increase the dose further
* Agree to use required contraception if they or their partner could become pregnant
* Are willing to carry two epinephrine devices at all times during Palynziq treatment
Exclusion Criteria:
* Pregnant, breastfeeding, planning to become pregnant, planning to father a child, or not using effective birth control if applicable
* Have a known severe allergy or hypersensitivity reaction to methotrexate (MTX), Palynziq, or other PEG-containing medications
* Have a serious active infection or a history of severe or recurrent infections
* Have significant medical conditions that may affect safety or participation (such as serious heart, lung, liver, kidney, immune, neurological, psychiatric, or cancer-related conditions)
* Have a history of substance or alcohol abuse within the past 12 months
* Have had an organ transplant or are taking chronic immunosuppressive medications
* Are currently taking medications that are not allowed in the study, including other PKU treatments besides Palynziq
* Are using, or plan to use, injectable PEG-containing medications other than Palynziq during the study
* Have major surgery planned during the study participation period
* Are currently participating in another clinical study involving Palynziq
* In the opinion of the study doctor, are not a suitable candidate for the study or may have difficulty complying with study requirements BIOLOGICAL: Pegvaliase, DRUG: Methotrexate
Phenylketonuria
PKU, Phenylketonuria, Pegvaliase, Palynziq, Methotrexate, Immune Response, Safety, Immune Modulation, BMN 165-401, Hyperphenylalaninemia, Enzyme Substitution Therapy, Pharmacologic Immunosuppression
UT Southwestern; Children’s Health
A First-in-human (FIH), Phase 1 Study of ML261, an Autologous Potency Enhanced Anti-DLL3 CAR T Cell Therapy, in Participants With R/R SCLC or Select NECs (SPECTRAL-1)
This is a first-in-human (FIH), open-label, Phase 1 study designed to evaluate the safety, pharmacokinetics (PK), and preliminary efficacy of ML261, an autologous potency enhanced anti-DLL3 CAR T cell therapy, in participants with R/R SCLC or select NECs
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Benjamin Drapkin
ALL
18 Years to old
PHASE1
NCT07488923
STU20260523
Inclusion Criteria
* ≥18 years of age at the time of signing the ICF
* Have been previously treated with at least one line of systemic standard of care (SOC) anti-cancer therapy for their respective cancer indication. Participants with locally advanced disease who are eligible for curative resection will be excluded.
* Have documented radiological disease progression/relapse during or after their most recent line of anti-cancer therapy with measurable disease on imaging, as assessed by RECIST v1.1
* Have histologically and/or cytologically confirmed diagnosis of select advanced or metastatic R/R solid tumor malignancy in one of the following: R/R SCLC, R/R GEP-NEC, R/R high-grade NEPC, R/R epNEC with biopsy-documented DLL3 expression on archival tissue or fresh biopsy by local or central assessment. CNS NEC is excluded. Participants with mixed histologies for any of these indications qualify if the small cell/neuroendocrine tumor cell percentage is \> 50%, except for high-grade NEPC where neuroendocrine component must be \> 20%.
* Eastern Cooperative Oncology Group (ECOG) Performance Score (PS) 0 or 1
* Life expectancy ≥12 weeks
* Have adequate hematologic and end-organ function
Exclusion Criteria:
* Previous systemic anti-cancer therapies within the timeframes, as specified in the protocol.
* Prior exposure/treatment with DLL3-targeted CAR T therapy or any other genetically engineered adoptive T cell therapy.
* Prior allogeneic organ transplant (including allogeneic bone marrow transplant).
* Major surgical procedure within 4 weeks of the first dose of any study drug administration or anticipated to be in need of a major surgical procedure during the course of study.
* Participants with toxicities (as a result of prior anti-cancer therapy) which have not recovered to baseline or CTCAE v5.0 \ BIOLOGICAL: ML261
Small Cell Lung Cancer (SCLC ), Extrapulmonary Neuroendocrine Carcinoma (EP-NEC), Gastroenteropancreatic NEC (GEP NEC), Neuroendocrine Prostate Cancer (NEPC)
Neuroendocrine carcinoma, DLL3, CAR T
UT Southwestern
A Study Comparing Siplizumab With Rabbit Anti-Thymocyte Globulin to Help the Body Accept a Kidney Transplant (MODERNIZE1)
The goal of this clinical trial is to learn if siplizumab can prevent rejection of a kidney transplant in adult participants with end stage kidney disease. The main questions it aims to answer are: How many adverse events do participants receiving two different doses of siplizumab have compared to rabbit anti-thymocyte globulin (rATG)? How many participants successfully keep their kidney transplant after receiving siplizumab or rATG? This will be calculated as those participants that did not: die; have their kidney fail to work properly (graft loss); their body rejects their transplant (tissue sample (biopsy)-proven acute rejection: BPAR); or who were lost to follow-up. How does the body respond to siplizumab after dosing at each of the 2 dose levels? How does siplizumab work in the body compared to rATG? Selected participants will be divided into 3 groups. In 2 of the groups, participants will be given siplizumab at one of the two study medicine doses. Participants in the third group will be given rATG. All participants will take the usual anti-rejection medicines given before, during, and after a kidney transplant. All participants will also be given medicines before the study drug to lower the risk of reactions, and medicines used to prevent or treat infections after the transplant. Participants will be asked to provide their medical history at the first visit at the study site where you will have your kidney transplant. At the first visit and other visits participants will be asked to provide their medication history, have a physical exam, check vital signs, have blood drawn for tests, and have non-invasive tests that record the electrical activity of your heart (ECG) and blood draws. Participants will be monitored for 12 months after transplant surgery.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Sarah.Joseph@UTSouthwestern.edu
David Wojciechowski
ALL
18 Years to 70 Years old
PHASE2
NCT07508787
STU20260532
Inclusion Criteria:
* Recipients of a renal allograft from a non-HLA identical living or deceased donor.
* Recipients of a kidney with a cold ischemia time \< 30 hours; hypothermic machine perfusion within the same timeframe is acceptable.
* Women of childbearing potential, defined as all women physiologically capable of becoming pregnant, must agree to use highly effective methods of contraception.
Exclusion Criteria:
* Transplant recipients seronegative for EBV.
* Transplant recipients receiving a kidney from a non-heart beating donor (ie, DCD) whose organ is retrieved from an uncontrolled DCD or whose donor age \> 55 years of age.
* Multi-organ transplant recipients (eg, kidney-pancreas, organ other than kidney), dual-kidney transplant recipients, recipients with more than one prior kidney transplant, or hematopoietic stem cell transplantation recipients.
* Presence of current or historical DSA via single-antigen bead assay or local SoC. The MFI cut-off value used to determine the presence of DSA will be defined as per the institutional SoC. Results within 3 months prior to transplant are acceptable.
* Crossmatch positive (isolated positive B cell crossmatches are not an exclusion criterion).
* ABO-incompatible recipient.
* Administration of complement inhibitor therapy within 6 months prior to the transplant, or likely to require treatment with complement inhibitor therapy during the study.
* Participants receiving immunosuppressive therapies prior to transplant for pre-existing conditions must be candidates for the protocol-defined regimen.
* History of malignancy of any organ system, except for localized excised non-melanomatous skin lesions or carcinoma in situ of the cervix.
* Seropositive for human immunodeficiency virus or hepatitis B surface antigen. Participants who are seropositive for hepatitis C virus (HCV) are excluded without proof of sustained viral response after anti-HCV treatment.
* Recipient of a kidney from a donor who tests positive for human immunodeficiency virus or hepatitis B surface antigen/hepatitis B core protein.
* History of hypersensitivity to any of the study drugs or to drugs of similar chemical classes (eg, siplizumab, rATG, TAC, MPA derivatives, CS).
* Evidence of active tuberculosis (TB) infection (participants with a history of latent TB may become eligible after treatment with anti-TB therapy according to national guidelines).
* Participants with severe systemic infection(s), at the time of screening or within 2 weeks prior to randomization DRUG: Siplizumab, DRUG: Rabbit anti-thymocyte globulin
Renal Transplant Failure and Rejection
UT Southwestern
Examining the Feasibility of Using Pressure Gradient Regulated Automated Cerebral Spinal Fluid Drainage During External Lumbar Drain Trials (UPGRADE)
The intellidrop device is an FDA-approved system that automates safe, small volume of cerebral spinal fluid drainage with continuous pressure monitoring, reducing nursing workload and human error while improving patient mobility and comfort
Call 214-648-5005
studyfinder@utsouthwestern.edu, Haben.Mehari@UTSouthwestern.edu
Padraig O'Suilleabhain
ALL
60 Years to 99 Years old
NA
NCT07494812
STU20260216
Inclusion Criteria:
* All patients will be adults aged 60 years or older with suspected iNPH who are being admitted to the hospital for the purpose of an LDT specifically to evaluate for benefit from shunt placement.
Exclusion Criteria:
* Patients who are under 18 years of age, pregnant, or are currently incarcerated will be excluded from participating in this study. DEVICE: Intellidrop Automated CSF Drainage System, DEVICE: Intellidrop Automated CSF Drainage System
NPH (Normal Pressure Hydrocephalus)
UPGRADE, Cerebrospinal fluid buildup (CSF buildup)
UT Southwestern
[18F]FPyQCP PET Imaging of Fibroblast Activation Protein in Selected Oncology Indications
This is a multi-center, open-label, single-arm, Phase 1/2 study designed to evaluate the safety, radiation dosimetry, and preliminary diagnostic performance of \[18F\]FPyQCP in detecting colorectal cancer (CRC), gastric cancer (GC), pancreatic ductal adenocarcinoma (PDAC), invasive lobular breast cancer (ILC), and epithelial ovarian cancer (EOC).
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Steven Rowe
ALL
18 Years to 80 Years old
PHASE1
NCT07432633
STU20250096
Inclusion Criteria:
• Participants provide informed consent and confirm that they are able and willing to comply with all protocol requirements.
• Participants must be ≥ 18 years and \< 80 years of age and competent to give informed consent.
• Eastern Cooperative Oncology Group performance status ≤ 2.
• Diagnosis of either CRC (confirmed by histopathology), GC (confirmed by histopathology), PDAC (confirmed by cytology or histopathology), ILC (confirmed by histopathology), or EOC (suspected or confirmed by cytology or histopathology).
• Women of childbearing potential (WOCBP) should have a negative serum test at screening (Visit 1) and a negative urine pregnancy test at the PET/CT imaging visit (Visit 2) prior to \[18F\]FPyQCP administration.
• WOCBP, and men who are sexually active with WOCBP, must agree to use a highly effective method(s) of contraception for the duration of the study
• Cohort A specific: Participants with stage I-III (see Appendix 3) CRC, GC, PDAC, ILC, or EOC (stage IV disease is allowed in the setting of oligometastatic disease \[5 or fewer known metastases) as assessed by conventional imaging within 8 weeks of \[18F\]FPyQCP administration.
• Cohort B specific: Conventional imaging performed within 8 weeks of screening and no later than 24 hours before \[18F\]FPyQCP administration and available for upload to the central imaging vendor, including, at a minimum, a contrast-enhanced CT that includes the abdomen and pelvis.
• Either:
• Treatment-naïve with at least stage IIB disease. Available biopsy sample or scheduled biopsy or surgical resection no later than Day 42.
• Following neoadjuvant therapy (with at least stage IIB disease at initial presentation) with scheduled biopsy or surgical resection no later than Day 42. \[18F\]FPyQCP PET/CT imaging should be performed as close to scheduled procedure as clinically feasible.
• Suspected recurrence after definitive therapy
Exclusion Criteria:
• Participants administered any radioisotope within 5 physical half-lives prior to \[18F\]FPyQCP administration.
• Participants administered any other IMP within 2 weeks or 5 half-lives, whichever is longest, prior to \[18F\]FPyQCP administration.
• Participants who have recently received any other contrast agent (\< 24 hours for intravenous agents and \< 5 days for oral agents) before the day of \[18F\]FPyQCP administration.
• Participants with a history of severe claustrophobia or panic attacks when in confined spaces.
• Known hypersensitivity to \[18F\]FPyQCP or any of its constituents.
• Participants with any medical condition or other circumstances at screening or in their past medical history that, in the opinion of the investigator, compromise obtaining reliable data, achieving study objectives, or study completion.
• Known diagnosis of an autoimmune or inflammatory disorder that is expected to confound image interpretation per investigator judgment, excluding disorders directly related to the index cancer (e.g. tumor-associated pancreatitis or biliary stasis for PDAC).
• Medical history of abdomino-pelvic or breast irradiation in the last 3 months.
• Presence of any current implanted foreign material (e.g. stents, surgical clips) that may confound image interpretation per investigator judgment.
• Significant renal or hepatic impairment.
• Female participants who are breastfeeding, unless the participant commits to pumping breast milk and discarding it from injection to ≥ 24 hours from the time of the \[18F\]FPyQCP administration.
• Cohort A Specific
Exclusion Criteria:
12. No prior history of any other cancer, unless treated with curative-intent and disease-free for at least 5 years before Visit 1 (at least 1 year before Visit 1 for basal cell carcinoma, localized squamous cell carcinoma of the skin and in situ carcinoma of cervix). DRUG: [18F]FPyQCP
Colorectal Cancer, Epithelial Ovarian Cancer, Gastric Cancer, Invasive Lobular Breast Carcinoma, Pancreatic Ductal Adenocarcinoma
Positron emission tomography, Fibroblast activation protein
UT Southwestern
A Study of TORL-5-700 in Relapsed/Refractory Non Hodgkin's Lymphoma
A Phase 1/2 study to evaluate safety, tolerability, and anticancer activity of TORL-5-700 as a monotherapy and in combination in R/R NHL
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Praveen Ramakrishnan Geethakumari
ALL
18 Years to old
PHASE1
NCT07439653
STU20261050
Inclusion Criteria:
* Confirmed B-NHL, including but not limited to de novo DLBCL; FL, Grades 1 to 3A; MCL; transformed lymphoma (tFL, RT) and FL Grade 3B; MZL and MALT.
* Eastern Cooperative Oncology Group (ECOG) performance status less than or equal to 2.
* At least 1 measurable lesion per Lugano criteria.
* Tumor tissue is available.
* Adequate organ function
Exclusion Criteria:
* T-cell lymphoma
* CLL or SL
* Burkitt lymphoma and high-grade B-cell lymphoma
* CNS involvement
* Peripheral neuropathy \> Grade 2
* Uncontrolled medical conditions
* Viral infections DRUG: TORL-5-700, DRUG: TORL-5-700 at MTD/RP2D, DRUG: TORL-5-700 at MTD/RP2D in combination with another agent
Histologically Confirmed Relapsed or Refractory B-cell Non-Hodgkin Lymphoma, Non-Hodgkins Lymphoma
UT Southwestern
A Study to Evaluate the Effectiveness and Safety of Setidegrasib, Given With Either mFOLFIRINOX or NALIRIFOX Chemotherapies, in People With Pancreatic Cancer
Pancreatic cancer is difficult to diagnose early. By the time people have been diagnosed, the cancer has usually spread to other parts of the body (metastatic). The standard treatment is chemotherapy, but other treatments are needed to improve outcomes in people with pancreatic cancer. The first treatment that people usually receive is chemotherapy. At the time this study started, some of the main standard chemotherapies for pancreatic cancer were mFOLFIRINOX or NALIRIFOX. Genes give your body instructions on how to make proteins. Proteins are needed to keep the body working properly. Many types of cancer are caused by changes in certain genes, making them faulty. Many people with pancreatic cancer have a faulty KRAS gene. One such change in the KRAS gene is called a G12D mutation. Researchers are looking for ways to stop the actions of abnormal proteins made from the KRAS G12D mutation. This study is about setidegrasib given with chemotherapy in people with pancreatic cancer who have the KRAS G12D mutation. Before setidegrasib can become an approved treatment, clinical studies need to be completed to understand how it works and how safe it is. The main aim is to learn if people who are given setidegrasib with chemotherapy live for longer than people who are given placebo with chemotherapy. Other aims are to learn if setidegrasib delays the cancer and symptoms returning, how the body processes setidegrasib, and its safety, when given with chemotherapy. People in this study will be adults with metastatic pancreatic cancer with the G12D mutation in their KRAS gene. Surgery or radiotherapy will not be an option to cure their cancer. People cannot take part if the cancer cells have spread to the thin tissue covering the brain and spinal cord (leptomeningeal disease), have symptoms of cancer in the brain or nervous system, or have recently had some other cancers that required treatment. In this study, people are given either setidegrasib with mFOLFIRINOX or NALIRIFOX chemotherapy, or a placebo with mFOLFIRINOX or NALIRIFOX chemotherapy. Whether people receive setidegrasib or placebo is decided by chance. The study doctor decides which chemotherapy (mFOLFIRINOX or NALIRIFOX) people receive. People will only receive NALIRIFOX chemotherapy (with setidegrasib or placebo) after the safety of setidegrasib with NALIRIFOX chemotherapy has been confirmed in another ongoing setidegrasib study. All of the study treatments are given slowly through a tube into a vein (infusion). People will continue to receive study treatment until their cancer gets worse, they can't tolerate the study treatment, they start other cancer treatment, they or the doctor decides the person should stop receiving study treatment, or sadly they pass away. There will be safety checks at each visit, and the doctors will continue to check for medical problems and people's wellbeing throughout the study.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Syed Kazmi
ALL
18 Years to old
PHASE3
NCT07409272
STU20260137
Inclusion Criteria:
* Participant has histologically confirmed metastatic pancreatic ductal adenocarcinoma (PDAC) with documented Kirsten rat sarcoma viral oncogene homolog (KRAS) G12D mutation based on local or central testing (confirmation of a participant's positive KRAS G12D mutation result must be available prior to randomization).
* Participant has no option for surgical resection or radiotherapy with curative intent.
* Participant consents to and provides a baseline tumor tissue specimen for the study during screening. The sample must meet the requirements described in the laboratory manual and the tumor sample guidance.
* Participant has an Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0 or 1 within 7 days prior to randomization.
* Participant has adequate organ function as indicated by the following laboratory values within 7 days prior to randomization (if a participant has received a recent blood transfusion, the latest laboratory tests must be obtained ≥ 14 days after any blood transfusion). The laboratory values prior to the initiation of the first dose of setidegrasib/placebo (or mFOLFIRINOX/NALIRIFOX, if chemotherapy is administered during the screening period) should be used to determine eligibility. Participants who receive mFOLFIRINOX/NALIRIFOX during the screening period must meet these criteria within 7 days prior to the start of on-treatment chemotherapy (i.e., C1D1).
* Participant agrees not to participate in another interventional study while receiving study intervention in the present study (participant who is currently in the follow-up period of an interventional clinical trial is allowed).
Exclusion Criteria:
* Participant has neuroendocrine, acinar pancreatic carcinoma or pancreatic cancer with squamous/adenosquamous features.
* Participant has another prior malignancy active (i.e., requiring treatment, including hormonal therapies, or intervention) within the previous 2 years different from the primary malignancy for this study, except for local malignancies that have been apparently cured, such as basal or squamous cell skin cancer, superficial bladder cancer or carcinoma in situ of the cervix or breast, which are allowed.
* Participant has chronic inflammatory bowel disease, bowel obstruction and/or severe uncontrolled diarrhea.
* Participant has peripheral sensory neuropathy with functional impairment.
* Participant has ascites and/or pleural effusion that require invasive interventions within 30 days prior to randomization or have an indwelling drainage catheter.
* Participant has symptomatic pulmonary embolism or pulmonary embolism not being treated with anticoagulation.
* Participant has a history of interstitial lung disease or pulmonary fibrosis.
* Participant has uncontrolled seizure disorder or refractory to antiepileptics.
* Participant has known homozygous uridine diphosphate glucuronosyltransferase 1A1 (UGT1A1) polymorphism.
* Participant has had a myocardial infarction, unstable angina or coronary artery bypass surgery within 6 months prior to randomization or currently has an uncontrolled illness including but not limited to symptomatic congestive heart failure, clinically significant cardiac disease (e.g., cardiomyopathy, infiltrative cardiac disease, etc.), unstable angina pectoris, cardiac arrhythmia, obligate use of a cardiac pacemaker or long QT interval (QT) syndrome.
* Participant has received any prior systemic therapy for their metastatic PDAC (except with up to 2 doses \[i.e., 28 days; 1 cycle\] of mFOLFIRINOX or NALIRIFOX during the screening period. If a participant received \[neo\]adjuvant chemotherapy, tumor recurrence or disease progression must have occurred ≥ 6 months after completing the last dose of the \[neo\]adjuvant therapy).
* Participant has had prior treatment with a KRAS G12D-targeted agent.
* Participant has a corrected QT interval by Fridericia (QTcF) (single electrocardiogram \[ECG\]) \> 470 msec during the screening period. DRUG: Setidegrasib, DRUG: Oxaliplatin, DRUG: Leucovorin, DRUG: Irinotecan, DRUG: fluorouracil, DRUG: liposomal irinotecan, DRUG: Placebo
Pancreatic Cancer, Metastatic Pancreatic Cancer, Metastatic Pancreatic Adenocarcinoma
Pancreatic Cancer, Metastatic Pancreatic Cancer, Metastatic Pancreatic Adenocarcinoma, ASP3082, setidegrasib, KRAS G12D, PDAC, mFOLFIRINOX, NALIRIFOX
UT Southwestern
A Phase 1b Study of HC-7366, an Agonist of ISR With Immunotherapy in Kidney Cancer (SHARK)
To find out if the combination of HC-7366 and nivolumab (with or without ipilimumab) can help to control ccRCC. The
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Hans Hammers
ALL
18 Years to old
PHASE1
NCT07401875
STU20252524
Eligibility Criteria
• Ability to understand and the willingness to sign a written informed consent document
• Male or female ≥ 18 years of age
• Confirmed diagnosis of clear cell RCC
• Stage IV metastatic RCC per American Joint Committee on Cancer
• Triplet Cohort (IO/IO): No prior systemic therapy for advanced RCC or prior adjuvant therapy allowed.
• Doublet Cohort: Participant must have progressed on at least one PD1 based doublet regimen (IO/IO or IO/TKI). Prior adjuvant therapy is allowed and does count as one line of systemic therapy.
• Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1 )
• At least one measurable lesion as defined by RECIST 1.1 • A tumor lesion situated in a previously irradiated area is considered a measurable/target lesion only if subsequent disease progression has been documented in the lesion
• Has pathology-confirmed RCC. Extra tissue should be submitted if available for correlatives. Formalin-fixed paraffin-embedded tissue blocks are preferred to slides. Details pertaining to tumor tissue submission can be found in the Lab Procedures Manual.
• Willing and able to undergo bone and brain scans at baseline and continue to have scans performed if positive at screening.
• Adequate organ function within 28 days prior to first dose of protocol-indicated treatment, including: * White blood cell (WBC) ≥ 2,000 /μL * Absolute neutrophil count (ANC) ≥ 1,000/μL * Platelet count ≥ 100,000/μL * Hemoglobin (Hgb) ≥ 9.0 g/dL in prior 4 weeks. Blood transfusions are allowed to achieve this. * Serum creatinine ≤ upper limit of normal (ULN), or calculated creatinine clearance ≥ 30 mL/min (per the Cockcroft-Gault formula,) * Total bilirubin ≤ ULN (except subjects with Gilbert Syndrome, who must have total bilirubin \< 3.0 mg/dL) * Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 3 x ULN
• Women must not be breastfeeding while taking the study drug and for up to five months after the last dose of study drug
• Women of childbearing potential (WOCBP) must have a negative serum or urine pregnancy test within 24 hours prior to receiving first dose of protocol-indicated treatment * "Women of childbearing potential" (WOCBP) is defined as any female who has experienced menarche who has not undergone surgical sterilization (hysterectomy or bilateral oophorectomy) or is not postmenopausal * Menopause is defined clinically as 12 months of amenorrhea in a woman over 45 years of age in the absence of other biological or physiological causes * If menopausal status is considered for the purpose of evaluating childbearing potential, women \< 62 years of age must have a documented serum follicle stimulating hormone (FSH) level within laboratory reference range for postmenopausal women, in order to be considered postmenopausal and not of childbearing potential
• Women of childbearing potential (WOCBP) must agree to follow instructions for acceptable contraception prior to the study and from the time of signing consent, for the duration of the study participation and for 23 weeks after their last dose of protocol-indicated treatment * The effects of HC-7366 on the developing human fetus are unknown. For this reason and because first-in-class, first-in-human agents as well as other therapeutic agents used in this trial are known to be teratogenic, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. (Refer to Pregnancy Assessment Policy MD Anderson Institutional Policy # CLN1114). * Approved methods of birth control are as follows: Hormonal contraception (i.e. birth control pills, injection, implant, transdermal patch, vaginal ring), Intrauterine device (IUD), Tubal Ligation or hysterectomy, Subject/Partner post vasectomy, Implantable or injectable contraceptives, and condoms plus spermicide. Not engaging in sexual activity for the total duration of the study and the drug washout period is an acceptable practice; however periodic abstinence, the rhythm method, and the withdrawal method are not acceptable methods of birth control. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately.
• Men not azoospermic who are sexually active with WOCBP must agree to follow instructions for acceptable contraception prior to the study and from the time of signing consent, for the duration of the study participation, and for 31 weeks after their last dose of protocol-indicated treatment
• Participant s with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated
• Participant s with a history of hepatitis C virus (HCV) infection must have been treated and cured. For participants with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load
• Participant s with previously treated brain metastases may be eligible provided they are radiologically (by MRI) and clinically stable (i.e., without evidence of disease progression) for at least 4 weeks (28 days) by repeat imaging (repeat imaging should be performed during study screening), with no evidence of new or enlarging brain metastases, and without requirement for steroid treatment for at least 28 days prior to the first dose of study drug or study therapy. (CT is acceptable if MRI is contraindicated)
• Participant s with a prior or concurrent malignancy whose natural history or treatment does not interfere with the safety or efficacy assessment of the investigational regimen are eligible for this study
• Participant s with known history or current symptoms of cardiac disease, or history of treatment with cardiotoxic agents, should have a clinical risk assessment of cardiac function using the New York Heart Association Functional Classification. To be eligible for this study, participant s should be class 2B or better Exclusion Criteria 1\. For the Triplet Cohort (Nivo/Ipi/HC-7366): * Prior systemic treatment including neoadjuvant or adjuvant therapy including an immune checkpoint inhibitor or TKI 2. For the Doublet Cohort (Nivo/HC-7366): * No more than 3 prior lines of systemic therapy allowed * Has received any type of small molecule kinase inhibitor (including investigational kinase inhibitor) ≤ 2 weeks before start of study drug or study therapy 3. ≤ 28 days before first dose of protocol-indicated treatment: * Major surgery requiring general anesthesia 4. ≤ 14 days before first dose of protocol-indicated treatment: * Radiosurgery or radiotherapy * Minor surgery. (Note: Placement of a vascular access device is not considered minor or major surgery) * Active infection requiring systemic treatment 5. Known or suspected clinically significant active bleeding including active hemoptysis 6. Inability to swallow oral medication; or the presence of a poorly controlled gastrointestinal disorder that could significantly affect the absorption of oral study drug - e.g. Crohn's disease, ulcerative colitis, chronic diarrhea (defined as \> 4 loose stools per day), malabsorption, or bowel obstruction 7. Central nervous system (CNS) metastasis, unless asymptomatic and radiologically (by MRI) and clinically stable (i.e., without evidence of disease progression) for at least 4 weeks (28 days) by repeat imaging (repeat imaging should be performed during study screening), with no evidence of new or enlarging brain metastases, and without requirement for steroid treatment for at least 28 days prior to the first dose of study drug or study therapy. (CT is acceptable if MRI is contraindicated 8. Any condition requiring systemic treatment with either corticosteroids (\> 10 mg/day prednisone or equivalent daily) or other immunosuppressive medications within 14 days prior to initiating protocol-indicated treatment * In the absence of active autoimmune disease: Subjects are permitted the use of corticosteroids with minimal systemic absorption (e.g. topical, ocular, intraarticular, intranasal, and inhalational) ≤ 10 mg/day prednisone or equivalent daily; and physiologic replacement doses of systemic corticosteroids ≤ 10 mg/day prednisone or equivalent daily (e.g. hormone replacement therapy needed in participants with hypophysitis) 9. Active, known or suspected autoimmune disease * Subjects with type I diabetes mellitus; hypothyroidism only requiring hormone replacement; skin disorders such as vitiligo, psoriasis or alopecia not requiring systemic treatment; or conditions not expected by the investigator to recur in the absence of an external trigger are permitted to enroll 10. Known psychiatric condition, social circumstance, or other medical condition reasonably judged by the investigator to unacceptably increase the risk of study participation; or to prohibit the understanding or rendering of informed consent or anticipated compliance with and interpretation of scheduled visits, treatment schedule, laboratory tests and other study requirements 11. Pregnant women are excluded from this study because HC-7366 is novel, first-in-class small molecule agent with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with HC-7366, breastfeeding should be discontinued if the mother is treated with HC-7366. These potential risks may also apply to other agents used in this study 12. Human immunodeficiency virus (HIV)-infected participants on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this study 13. Participants who are receiving any other investigational agents
• Ability to understand and the willingness to sign a written informed consent document
• Male or female ≥ 18 years of age
• Confirmed diagnosis of clear cell RCC
• Stage IV metastatic RCC per American Joint Committee on Cancer
• Triplet Cohort (IO/IO): No prior systemic therapy for advanced RCC or prior adjuvant therapy allowed.
• Doublet Cohort: Participant must have progressed on at least one PD1 based doublet regimen (IO/IO or IO/TKI). Prior adjuvant therapy is allowed and does count as one line of systemic therapy.
• Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1 )
• At least one measurable lesion as defined by RECIST 1.1 • A tumor lesion situated in a previously irradiated area is considered a measurable/target lesion only if subsequent disease progression has been documented in the lesion
• Has pathology-confirmed RCC. Extra tissue should be submitted if available for correlatives. Formalin-fixed paraffin-embedded tissue blocks are preferred to slides. Details pertaining to tumor tissue submission can be found in the Lab Procedures Manual.
• Willing and able to undergo bone and brain scans at baseline and continue to have scans performed if positive at screening.
• Adequate organ function within 28 days prior to first dose of protocol-indicated treatment, including: * White blood cell (WBC) ≥ 2,000 /μL * Absolute neutrophil count (ANC) ≥ 1,000/μL * Platelet count ≥ 100,000/μL * Hemoglobin (Hgb) ≥ 9.0 g/dL in prior 4 weeks. Blood transfusions are allowed to achieve this. * Serum creatinine ≤ upper limit of normal (ULN), or calculated creatinine clearance ≥ 30 mL/min (per the Cockcroft-Gault formula,) * Total bilirubin ≤ ULN (except subjects with Gilbert Syndrome, who must have total bilirubin \< 3.0 mg/dL) * Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 3 x ULN
• Women must not be breastfeeding while taking the study drug and for up to five months after the last dose of study drug
• Women of childbearing potential (WOCBP) must have a negative serum or urine pregnancy test within 24 hours prior to receiving first dose of protocol-indicated treatment * "Women of childbearing potential" (WOCBP) is defined as any female who has experienced menarche who has not undergone surgical sterilization (hysterectomy or bilateral oophorectomy) or is not postmenopausal * Menopause is defined clinically as 12 months of amenorrhea in a woman over 45 years of age in the absence of other biological or physiological causes * If menopausal status is considered for the purpose of evaluating childbearing potential, women \< 62 years of age must have a documented serum follicle stimulating hormone (FSH) level within laboratory reference range for postmenopausal women, in order to be considered postmenopausal and not of childbearing potential
• Women of childbearing potential (WOCBP) must agree to follow instructions for acceptable contraception prior to the study and from the time of signing consent, for the duration of the study participation and for 23 weeks after their last dose of protocol-indicated treatment * The effects of HC-7366 on the developing human fetus are unknown. For this reason and because first-in-class, first-in-human agents as well as other therapeutic agents used in this trial are known to be teratogenic, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. (Refer to Pregnancy Assessment Policy MD Anderson Institutional Policy # CLN1114). * Approved methods of birth control are as follows: Hormonal contraception (i.e. birth control pills, injection, implant, transdermal patch, vaginal ring), Intrauterine device (IUD), Tubal Ligation or hysterectomy, Subject/Partner post vasectomy, Implantable or injectable contraceptives, and condoms plus spermicide. Not engaging in sexual activity for the total duration of the study and the drug washout period is an acceptable practice; however periodic abstinence, the rhythm method, and the withdrawal method are not acceptable methods of birth control. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately.
• Men not azoospermic who are sexually active with WOCBP must agree to follow instructions for acceptable contraception prior to the study and from the time of signing consent, for the duration of the study participation, and for 31 weeks after their last dose of protocol-indicated treatment
• Participant s with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated
• Participant s with a history of hepatitis C virus (HCV) infection must have been treated and cured. For participants with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load
• Participant s with previously treated brain metastases may be eligible provided they are radiologically (by MRI) and clinically stable (i.e., without evidence of disease progression) for at least 4 weeks (28 days) by repeat imaging (repeat imaging should be performed during study screening), with no evidence of new or enlarging brain metastases, and without requirement for steroid treatment for at least 28 days prior to the first dose of study drug or study therapy. (CT is acceptable if MRI is contraindicated)
• Participant s with a prior or concurrent malignancy whose natural history or treatment does not interfere with the safety or efficacy assessment of the investigational regimen are eligible for this study
• Participant s with known history or current symptoms of cardiac disease, or history of treatment with cardiotoxic agents, should have a clinical risk assessment of cardiac function using the New York Heart Association Functional Classification. To be eligible for this study, participant s should be class 2B or better Exclusion Criteria 1\. For the Triplet Cohort (Nivo/Ipi/HC-7366): * Prior systemic treatment including neoadjuvant or adjuvant therapy including an immune checkpoint inhibitor or TKI 2. For the Doublet Cohort (Nivo/HC-7366): * No more than 3 prior lines of systemic therapy allowed * Has received any type of small molecule kinase inhibitor (including investigational kinase inhibitor) ≤ 2 weeks before start of study drug or study therapy 3. ≤ 28 days before first dose of protocol-indicated treatment: * Major surgery requiring general anesthesia 4. ≤ 14 days before first dose of protocol-indicated treatment: * Radiosurgery or radiotherapy * Minor surgery. (Note: Placement of a vascular access device is not considered minor or major surgery) * Active infection requiring systemic treatment 5. Known or suspected clinically significant active bleeding including active hemoptysis 6. Inability to swallow oral medication; or the presence of a poorly controlled gastrointestinal disorder that could significantly affect the absorption of oral study drug - e.g. Crohn's disease, ulcerative colitis, chronic diarrhea (defined as \> 4 loose stools per day), malabsorption, or bowel obstruction 7. Central nervous system (CNS) metastasis, unless asymptomatic and radiologically (by MRI) and clinically stable (i.e., without evidence of disease progression) for at least 4 weeks (28 days) by repeat imaging (repeat imaging should be performed during study screening), with no evidence of new or enlarging brain metastases, and without requirement for steroid treatment for at least 28 days prior to the first dose of study drug or study therapy. (CT is acceptable if MRI is contraindicated 8. Any condition requiring systemic treatment with either corticosteroids (\> 10 mg/day prednisone or equivalent daily) or other immunosuppressive medications within 14 days prior to initiating protocol-indicated treatment * In the absence of active autoimmune disease: Subjects are permitted the use of corticosteroids with minimal systemic absorption (e.g. topical, ocular, intraarticular, intranasal, and inhalational) ≤ 10 mg/day prednisone or equivalent daily; and physiologic replacement doses of systemic corticosteroids ≤ 10 mg/day prednisone or equivalent daily (e.g. hormone replacement therapy needed in participants with hypophysitis) 9. Active, known or suspected autoimmune disease * Subjects with type I diabetes mellitus; hypothyroidism only requiring hormone replacement; skin disorders such as vitiligo, psoriasis or alopecia not requiring systemic treatment; or conditions not expected by the investigator to recur in the absence of an external trigger are permitted to enroll 10. Known psychiatric condition, social circumstance, or other medical condition reasonably judged by the investigator to unacceptably increase the risk of study participation; or to prohibit the understanding or rendering of informed consent or anticipated compliance with and interpretation of scheduled visits, treatment schedule, laboratory tests and other study requirements 11. Pregnant women are excluded from this study because HC-7366 is novel, first-in-class small molecule agent with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with HC-7366, breastfeeding should be discontinued if the mother is treated with HC-7366. These potential risks may also apply to other agents used in this study 12. Human immunodeficiency virus (HIV)-infected participants on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this study 13. Participants who are receiving any other investigational agents
DRUG: Nivolumab, DRUG: HC-7366, DRUG: ipilimumab
Phase 1b, HC-7366, SHARK, Kidney Cancer, Kidney
UT Southwestern
Quality of End-of-Life Care for Children With Cancer
This study examines the role of access to care, patient/family interactions with the healthcare system, and stress in explaining variations in quality of end-of-life care. The data collected from this study may help researchers develop a model for identifying patients at risk of low-quality end-of-life care as well as recommendations for potential future interventions.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Gabriella Nguyen
ALL
18 Years to old
NCT07412002
STU20261254
Inclusion Criteria:
* Index Child (COG Registered Patient):
* Must be deceased
* \< 18 years old at time of death
* Diagnosed with any oncologic condition
* History of enrollment on Stratum 1 of APEC14B1, Project:EveryChild
* Note: history of treatment on a COG therapeutic trial is not required
* Resided in the United States, including Puerto Rico, as evidenced by most recent address, at the time of death
Bereaved Parent(s):
* Must be a parent, legal guardian, or caregiver (biological or non-biological) of an eligible Index Child enrolled on APEC14B1 with current Consent to Future Contact (e.g., from the APEC14B1 Part B Consent)
* Self-reported confirmation of familiarity with the care received by the Index Child in the last month of their life
* Bereaved parent must understand English or Spanish (written and/or spoken)
* Must be a parent/guardian/key contact from the Index Child's APEC14B1 Future Contact study record or must be referred by one of the APEC14B1 contacts
* Bereaved parent must be \>= 18 years old at the time of ALTE24C1 study enrollment
Regulatory Requirements:
* Bereaved parent must provide verbal or implied informed consent
* For all participants, all institutional, FDA, and NCI requirements for human studies must be met OTHER: Non-Interventional Study
Childhood Hematopoietic and Lymphatic System Neoplasm, Childhood Malignant Solid Neoplasm
Children’s Health
A Study to Assess How Well and Safely Elafibranor Works in Adult Participants With Primary Sclerosing Cholangitis (ELASCOPE)
The purpose of this study is to find out how well and safely elafibranor works compared to placebo in adult participants with Primary sclerosing cholangitis (PSC). PSC is a rare disease that causes inflammation and scarring of the bile ducts in the liver. Over time, this can lead to liver damage and serious health problems, including the need for a liver transplant and death. In this study, about 350 participants with large duct PSC will take part. Participants will be randomized to receive either elafibranor 120 mg once daily or a placebo (a tablet with no active medicine). The study includes a screening period, an treatment period, and a post-treatment safety follow-up. During the study, participants will undergo routine clinical assessments, laboratory testing, imaging evaluations, and complete patient-reassessments to evaluate liver disease progression, symptoms, quality of life and safety. Following the end of treatment, participants will complete a safety follow-up period at approximately four weeks. Participants may withdraw from the study at any time. Each participant may be in the study for several years, as the treatment period will continue until the study reaches enough health events among participants, which is expected to take about 5 years.
studyfinder@utsouthwestern.edu
ALL
18 Years to 75 Years old
PHASE3
NCT07387549
Inclusion Criteria:
* Adults participants aged 18 years or older
* Confirmed diagnosis of primary sclerosing cholangitis based on standard clinical, biochemical, and imaging criteria
* Compensated liver disease at screening
* Stable background therapy, where applicable prior to study entry
* Women of childbearing potential have to apply during the entire duration of the study a highly effective method of birth control
* Ability to provide written informed consent and comply with study procedures.
Exclusion Criteria:
\- History or presence of other concomitant chronic liver disease
\- History of hepatic decompensation, including: i) History of liver transplantation, current MELD 3.0 score ≥12 due to hepatic impairment.
ii) Evidence of complications of cirrhosis
* Participants with cirrhosis who are also classified as Child-Pugh B or C based on the Child Pugh score.
* History of biliary intervention within 60 days prior to the screening period, and/or presence of percutaneous drain or bile duct stent at SV.
* History of bacterial cholangitis, and/or participant on antibiotics for prophylaxis of recurrent cholangitis within 60 days prior to the SV.
* History or any current suspicion of cholangiocarcinoma or hepatocellular carcinoma
* Known malignancy or history of malignancy within the last 5 years, with the exception of local, successfully treated basal cell carcinoma or in-situ carcinoma of the uterine cervix.
* Medical conditions that may cause non-hepatic increases in ALP (e.g. Paget's disease).
* Administration of the following medications are prohibited as specified below:
i) 3 months prior to baseline: norucholic acid, fibrates, seladelpar and glitazones.
ii) 3 months prior to baseline: cyclosporine, mycophenolate, pentoxifylline, and chronic systemic corticosteroids (except as part of management of IBD at an ongoing stable dose); potentially hepatotoxic drugs (including α-methyl-dopa, sodium valproic acid, isoniazid, or nitrofurantoin).
* Participants who are currently participating in, plan to participate in, or have participated in an investigational drug or medical device study containing active substance within 30 days or five half-lives, whichever is longer, prior to the SV. - Participants with previous exposure to elafibranor.
* Electrocardiogram (ECG) with QT interval corrected by Fridericia's formula (QTcF) \>450 msec in males or QTcF \>470 msec in females for participants without bundle branch block.
* Significant renal disease,
* For female participants: known pregnancy, or has a positive serum pregnancy test, or lactating.
* Regular alcohol intake in excess of the recommended limit of 2 standard drinks per day for men or 1 standard drink per day for women
* History of alcohol abuse, or other substance abuse within 1 year prior to SV.
* Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that contraindicates participation in the study.
* Mental instability or incompetence
* Participant has or is known to have tested positive for human immunodeficiency virus (HIV) type 1 or 2 at SV.
* Medical conditions that may diminish life expectancy to \<2 years. DRUG: Elafibranor, OTHER: Placebo
Primary Sclerosing Cholangitis
Imaging Acetadote Metabolism in Glioblastoma
This goal of this clinical trial is to evaluate how Acetadote affects metabolism in patients with glioblastoma. Drugs like Acetadote, which affect the level of damage in a cell (oxidative stress), may impact brain tumor metabolism and slow the growth of brain tumors. The investigators are evaluating how Acetadote affects glioblastoma metabolism by using MRI-based methods and by determining the changes in metabolism in brain tumor tissue resected from patients with a new diagnosis of glioblastoma.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Evan Noch
ALL
18 Years to old
EARLY_PHASE1
NCT07387666
STU20250529
Inclusion Criteria:
* 1\. Glioblastoma
* 2\. Newly diagnosed with no prior surgery, radiation, chemotherapy, or other tumor-treating agent
* 3\. Age ≥18 years
* 4\. KPS \> 70
* 5\. Adequate organ and marrow function as defined below:
* \- Bilirubin ≤1.5 times upper limit of normal
* \- AST and ALT ≤ 3 times ULN
* \- Creatinine ≤ 1.5 x ULN and/or GFR ≤ 60 mL/min
* -ANC ≥ 1000 cells/ul
* \- Platelet ≥ 100,000/ul
* \- Hemoglobin ≥ 9 g/dl
* 6\. All men, as well as women of child-bearing potential must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation, and for 7 days following completion of therapy. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
* 6a. A female of child-bearing potential is any woman (regardless of sexual orientation, marital status, having undergone a tubal ligation, or remaining celibate by choice) who meets the following criteria:
* \- Has not undergone a hysterectomy or bilateral oophorectomy; or
* \- Has not been naturally postmenopausal for at least 12 consecutive months (i.e., has had menses at any time in the preceding 12 consecutive months).
* 7\. Ability to understand and the willingness to sign a written informed consent.
Exclusion Criteria:
* 1.Chemotherapy, radiotherapy, or other cancer therapy within 4 weeks prior to starting study treatment.
* 2\. Subjects must have recovered from prior treatment-related toxicities to grade 2 or baseline (excluding alopecia and clinically stable toxicities requiring ongoing medical management, such as hypothyroidism from prior immune checkpoint inhibitor treatment).
* 3\. Subjects may not be receiving any other investigational agents for the treatment of the cancer under study.
* 4\. Brain metastases
* 5\. History of allergic or hypersensitivity reactions attributed to compounds of similar chemical or biologic composition to Acetadote.
* 6\. Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that, in the opinion of the investigator, would limit compliance with study requirements.
* 7\. Subjects must not be pregnant or nursing due to the potential for congenital abnormalities and the potential of this regimen to harm nursing infants. DRUG: Acetadote
Glioblastoma, GBM, Brain and Nervous System
UT Southwestern
IMPACT-MACS: Adrenalectomy vs Semaglutide for Metabolic Outcomes in Mild Autonomous Cortisol Secretion (IMPACT-MACS)
The goal of this study is to learn how two treatments-adrenalectomy (surgical removal of an adrenal gland) and semaglutide (a medication used for weight management)-affect insulin resistance and cortisol regulation in adults with mild autonomous cortisol secretion (MACS). The study will also learn how these treatments impact body composition, blood pressure, cholesterol, inflammation, muscle strength, and quality of life. The main questions the study aims to answer are: 1. Does adrenalectomy or semaglutide improve insulin resistance more in people with MACS? 2. How do these treatments change cortisol patterns and other cardiometabolic risk factors? 3. Do people with MACS respond differently to semaglutide compared to matched adults without MACS? Participants will: 1. Receive either adrenalectomy or semaglutide if they have MACS, or semaglutide if they are matched controls 2. Complete clinic visits and phone visits over about 26-30 weeks 3. Undergo metabolic testing such as blood tests, urine steroid profiling, body composition scans, blood pressure monitoring, muscle strength testing, and questionnaires about health and well-being
Call 214-648-5005
studyfinder@utsouthwestern.edu, MartinTzeWah.Kueh@UTSouthwestern.edu
Oksana Hamidi
ALL
18 Years to old
NA
NCT07361874
STU20251717
Inclusion Criteria:
* Adults ≥18 years
* MACS groups: adrenal adenoma + DST cortisol \>1.8 µg/dL + no overt Cushing + eligible for adrenalectomy
* Willingness to postpone surgery 6 months if randomized
* Controls: no adrenal abnormalities + normal DST + BMI ≥27 + ≥2 cardiometabolic conditions
* Stable medication doses for ≥4 weeks
* Negative pregnancy test if applicable
Exclusion Criteria:
* Prior GLP-1 RA within 90 days
* Weight change \>5 kg in past 90 days
* Prior obesity/diabetes surgery
* Type 1 diabetes or other diabetes types
* Severe organ disease
* Recent pancreatitis
* Pregnancy, breastfeeding
* Contraindication to semaglutide
* Contraindication to surgery delay
* Chronic glucocorticoid use PROCEDURE: Intervention 1: Adrenalectomy, DRUG: Intervention 2: Semaglutide
Mild Autonomous Cortisol Secretion (MACS), Autonomous Cortisol Secretion (ACS), Subclinical Cushing's, Mild Autonomous Cortisol Excess
Mild Autonomous Cortisol Secretion, MACS, Adrenal incidentaloma, Adrenal adenoma, Subclinical Cushing, Hypercortisolism, Cortisol dysregulation, Adrenalectomy, Semaglutide, Weight loss, Body composition, Insulin resistance, GLP-1 receptor agonist, Hyperinsulinemic-euglycemic clamp, Cortisol dynamics, Visceral fat, cardiometabolic risk, randomized controlled trial
UT Southwestern
Pediatric Epilepsy
The purpose of the research is to better understand how the human brain accomplishes the basic cognitive tasks of learning new information, recalling stored information, making decisions or choices about presented information and self-control. These investigations are critical to better understand human cognition and to design treatments for disorders of learning, memory, decision making and cognitive control.
Zhongzheng Fu zhongzheng.fu@utsouthwestern.edu
ALL
3 Years to 25 Years old
NA
NCT07350551
Inclusion Criteria:
* Candidates will be those who are admitted to the Epilepsy Monitoring Unit and are able to participate in a pre-operative evaluation using depth intracranial electrodes. The candidacy is determined independently by the patient's treating physician as part of the patient's routine medical care.
* Patients have drug-intractable epilepsy undergoing invasive monitoring in the EMU.
Exclusion Criteria:
* Determination by clinicians and investigators that a patient is unable to complete the behavioral tasks required for the protocol due to either cognitive limits, psychological limits, or pain. BEHAVIORAL: Cedrus RB series response pad, Adtech Behnke-Fried micro-electrodes, Neuralynx or Blackrock electrophysiology system, Blackrock Cerestim or Natus Nicolet stimulator
Epilepsy Intractable
Epilepsy, Decision-making, Cognitive control, Memory, Depth Electrode, Brain Diseases, Central Nervous System Diseases, Nervous System Diseases, ADHD (Attention-Deficit/Hyperactivity Disorder), Cognitive development
A Study to Assess Adverse Events and How Intravenous (IV) Pivekimab Sunirine Moves Through the Body in Pediatric Participants With Relapsed or Refractory Acute Myeloid Leukemia (AML)
Acute myeloid leukemia (AML) is an aggressive blood cancer, withwith few options for participants who relapse after treatment or who don't respond to treatment. This study will assess the adverse events and how pivekimab sunirine moves through the body in pediatric participants with relapsed or refractory (R/R) AML. Pivekimab sunirine is a drug being evaluated in the treatment of AML. This is an open label, single arm study, participants will be enrolled in 1 of the 3 cohorts based on their age and will receive pivekimab sunirine at a dose based on their weight. Around 18 pediatric participants with a diagnosis of AML will be enrolled in the study at approximately 30 sites around the world. Participants will receive intravenous (IV) pivekimab sunirine alone. The total study duration is approximately 28 months. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, and checking for side effects.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Caroline Smith
ALL
6 Months to 17 Years old
PHASE1
NCT07306832
STU20260749
Inclusion Criteria:
* Must have histologically confirmed acute myeloid leukemia (AML) meeting one of the following disease criteria:
* Second or greater relapse. OR
* Disease refractory to second or subsequent line of therapy (defined as resistant disease after at least one cycle of each treatment regimen).
* Must have myeloid leukemic blasts that are CD123-positive by flow cytometry as determined by the treating institution.
* Has \>= 5% myeloid leukemic blasts in bone marrow at time of relapse or refractory disease and prior to Screening for this study.
* Performance status by Lansky (\< 16 years old at evaluation) or Karnofsky (\>= 16 years old at evaluation) score \>= 50 or ECOG score \<= 2.
* May have status of central nervous system (CNS)1, CNS2, or CNS3 disease without clinical signs or neurologic symptoms suggestive of CNS leukemia, such as facial nerve palsy, brain/eye involvement or hypothalamic syndrome. Participants receiving intrathecal therapy and no additional CNS-directed systemic therapy at study entry are eligible and may continue treatment as clinically indicated in accordance with institutional practice.
* For those participants who have not reached the age of consent, parent or legal guardian with the willingness and ability to provide informed consent and participant willing and able to give assent, as appropriate for age and country.
Exclusion Criteria:
* Known clinically significant cardiac disease.
* Down syndrome.
* Acute promyelocytic leukemia (APL) or juvenile myelomonocytic leukemia (JMML).
* Symptomatic central nervous system (CNS3) disease
* Prior history of any severity veno-occlusive disease/sinusoidal obstructive syndrome (VOD/SOS) of the liver.
* Prior history of hematopoietic stem cell transplant within 6 months prior to Screening without evidence of active GvHD at the time of screening and the participant is off medications to treat or prevent either post-transplant graft-versus-host disease (GvHD) or post-transplant rejection (except for a stable dose of corticosteroids).
* Have received prior Chimeric Antigen Receptor T-cell (CAR-T) therapy.
* Any other known current malignancy requiring therapy.
* Currently receiving anticancer therapy with antineoplastic intent, including radiotherapy, systemic therapy small molecules, monoclonal antibodies, other investigational agents, or high-dose chemotherapy with the exception of intrathecal therapy. DRUG: Pivekimab Sunirine
Acute Myeloid Leukemia
Acute Myeloid Leukemia, Relapsed, Refractory, Pivekimab Sunirine, PVEK, Pediatric
Children’s Health
A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B (ATLAS-KIDS)
This is a parallel, Phase 3, two-arm, open-label study to evaluate the efficacy and safety of treatment with fitusiran prophylaxis administered to male pediatric participants (aged 1 to \<12 years) who have severe hemophilia A or B, with or without inhibitory antibodies to FVIII or FIX. Number of participants: Approximately 85 participants will be enrolled into the study: * Approximately 60 fitusiran-naïve participants with severe hemophilia A or B, with or without inhibitors (fitusiran-naïve arm), and * Approximately 25 participants with severe hemophilia A or B with inhibitors rolling over from the EFC15467\* dose confirmation study (roll-over arm). * Fitusiran has been investigated in the pediatric population in study EFC15467, which enrolled male participants aged 1 to \<12 years with hemophilia A or B with inhibitors to examine the safety and tolerability of fitusiran in the pediatric population. Participants will be enrolled into 1 of 2 arms: * Fitusiran-naïve: these participants have not previously received fitusiran, and they will undergo screening and study eligibility assessments. Once enrolled, they will go through a 24-week standard of care (SOC) period before starting fitusiran prophylaxis. * Roll-over participants from the EFC15467 study: only participants who are still on active treatment in study EFC15467 and consenting to study EFC17905 will be eligible to roll over. They will not need to undergo screening or further eligibility assessments. They will directly enroll into the fitusiran treatment period and continue treatment on their current fitusiran dose. The duration of fitusiran treatment will be up to 160 weeks for the fitusiran-naïve arm and up to 60 weeks for the roll-over arm.
Call 214-648-5005
studyfinder@utsouthwestern.edu, susan.corley@childrens.com
Jessica Garcia
MALE
1 Year to 11 Years old
PHASE3
NCT07285460
STU20252266
Inclusion Criteria:
Participants not previously exposed to fitusiran are eligible to be included in the study only if all of the following criteria apply:
* Participant must be 1 to \<12 years of age at the time of enrollment.
* Participants must have severe hemophilia A or B (FVIII \<1% or FIX ≤2%) as evidenced by a central laboratory measurement at screening or documented medical record evidence.
* Participants must meet inhibitor or non-inhibitor status as defined below:
Inhibitor:
Requiring use of BPA for prophylaxis or BPA as on-demand therapy for any bleeding episodes for at least the last 3 months prior to screening, and meet one of the following Nijmegen-modified Bethesda assay results criteria:
* Inhibitor titer of ≥0.6 BU/mL at screening, OR
* Inhibitor titer of \<0.6 BU/mL at screening with medical record evidence of 2 consecutive titers ≥0.6 BU/mL, OR
* Inhibitor titer of \<0.6 BU/mL at screening with medical record evidence of 1 inhibitor titer ≥0.6 BU/mL and a history of anamnestic response, or severe allergic reaction (eg, anaphylaxis) or nephrotic syndrome
Non-inhibitor:
Requiring use of clotting factor concentrates (CFCs) for prophylaxis or CFCs as on-demand therapy for any bleeding episodes for at least the last 3 months prior to screening, and meet each of the following criterion:
* Nijmegen-modified Bethesda assay inhibitor titer of \<0.6 BU/mL at screening, AND
* No use of BPA to treat bleeding episodes for at least the last 3 months prior to screening
* Participants must have adequate peripheral venous access, as determined by the Investigator, to allow the blood draws required by the study protocol.
* Male: There are no contraceptive requirements for this study except where required by local regulations.
* Capable of giving signed informed consent/assent. A signed written informed consent must be obtained from parent(s)/legal guardian (hereafter referred to as the "parent"), as well as a written or oral assent obtained from participant, per local and national requirements.
Exclusion Criteria:
Participants not previously exposed to fitusiran are excluded from the study if any of the following criteria apply:
* Known co-existing bleeding disorders other than hemophilia A or B.
* Presence of clinically significant liver disease.
* History of antiphospholipid antibody syndrome.
* History of arterial or venous thromboembolism, unrelated to an indwelling venous access
* Any condition (eg, medical concern), which in the opinion of the Investigator, would make the participant unsuitable for dosing or which could interfere with the study compliance, the participant's safety and/or the participant's participation in the completion of the treatment period of the study.
* History of multiple drug allergies or history of allergic reaction to an oligonucleotide or GalNAc.
* Subjects with a central or peripheral indwelling catheter, with a history of venous access complications (such as infections, thrombosis) leading to hospitalization and/or systemic anticoagulation therapy in the last 12 months.
* At screening, anticipated need of surgery during the study or planned surgery scheduled to occur during the study.
* Completion of a surgical procedure within 14 days prior to screening, or currently receiving additional BPA infusion for postoperative hemostasis.
* History of intolerance to SC injection(s).
* Current participation in ITI therapy.
* The use of emicizumab (Hemlibra®) or any non-factor bleed management treatment within 6 months prior to screening
* Prior gene therapy
* Current or future participation in another clinical study, scheduled to occur during this study, involving an investigational product other than fitusiran or an investigational device.
* AT activity \<60% at screening, as determined by central laboratory analysis.
* Co-existing thrombophilic disorder.
* Presence of an active Hepatitis C virus infection
* Presence of acute hepatitis A or Hepatitis E virus infection.
* Presence of acute or chronic hepatitis B virus infection.
* Platelet count ≤100 000/μL.
* Presence of acute infection at screening.
* Human immunodeficiency virus (HIV) positive with a CD4 count of \<400 cells/μL.
* Estimated glomerular filtration rate ≤45 mL/min/1.73 m2 (using the Schwartz formula).
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial. DRUG: Fitusiran, BIOLOGICAL: Clotting factor concentrates (CFC) or bypassing agents (BPA), BIOLOGICAL: Antithrombin concentrate (ATIIIC)
Hemophilia
Children’s Health
A Study to Evaluate the Efficacy and Safety of Standard-of-Care Chemotherapy and Bevacizumab With or Without INCA33890 in the First-Line Treatment of Metastatic Microsatellite Stable Colorectal Cancer
The purpose of this study is to evaluate the efficacy and safety of standard-of-care chemotherapy and bevacizumab with or without INCA33890 in the first-line treatment of metastatic microsatellite stable colorectal cancer.
studyfinder@utsouthwestern.edu
ALL
18 Years to old
PHASE3
NCT07284849
Inclusion Criteria:
* Stage IV colorectal adenocarcinoma not amenable to curative resection.
* No prior systemic treatment for unresectable or metastatic disease. Participants who received adjuvant or neoadjuvant therapy may enroll if there was no recurrence within 12 months of the end of treatment.
* Measurable disease per RECIST v1.1.
* ECOG performance status of 0 or 1.
* Adequate organ function determined by laboratory results.
Exclusion Criteria:
* MSI-H/dMMR per historical data in the medical record.
* BRAF V600E mutation per historical data in the medical record.
* Untreated and/or progressing CNS metastases.
* History of other malignancy within 2 years.
* Treatment with an anti-PD-(L)1 or other immune checkpoint inhibitor for any indication within the last 3 years.
* Active autoimmune disease that has required systemic treatment in the past 2 years.
* Significant concurrent and/or uncontrolled medical condition.
* History of organ transplant, including allogeneic stem cell transplantation.
Other protocol-defined inclusion/exclusion criteria apply. DRUG: INCA33890, DRUG: Placebo, DRUG: Bevacizumab, DRUG: FOLFOX
CRC (Colorectal Cancer)
Metastatic Colorectal Cancer, Colon Cancer, INCA33890
DOC1021 Dendritic Cell Immunotherapy for Refractory Melanoma
The goal of this clinical trial is to learn if DOC1021 + pIFN will be safe and will lead to tumor responses in patients with refractory melanoma. DOC1021 is a dendritic cell immunotherapy derived from a patient's own blood cells and loaded with antigens from the patient's tumor in the form of tumor lysate and mRNA. The goal is to stimulate a T cell immune response that eliminates tumor cells. The study consists of two components: an initial phase I safety study to confirm safety/tolerability of the treatment regimen, and, subsequently, a single-arm phase II cohort to assess efficacy of the treatment regimen. All participants will: * Undergo a leukapheresis collection (take filgrastim subcutaneously x 5 doses, if clinically necessary, leading up to collection) * Receive two doses of DOC1021 under image guidance 2 weeks apart * Receive subcutaneous pIFN injections weekly for a total of 4 doses in parallel with the DOC1021 injections * Undergo an optional image-guided perinodal DOC1021 booster injection approximately 6 months after the first DOC1021 dose along with additional subcutaneous pIFN injections at time of the booster and the subsequent week for a total of 2 pIFN doses * Visit the clinic regularly to assess quality of life, symptoms, medication use, imaging, bloodwork, and to receive optional treatment with anti-PD1 agents
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Sanjay Chandrasekaran
ALL
18 Years to old
PHASE1
NCT07288112
STU20260277
Inclusion Criteria:
• Provision of signed and dated informed consent form
• Stated willingness to comply with all study procedures and avail-ability for the duration of the study
• Age 18 years or older
• Patients diagnosed with unresectable or metastatic melanoma and progressed following ≥1 prior systemic therapy including anti-PD-1 (i.e., refractory to anti-PD-1). Refractory defined as primary or secondary resistance as per SITC guidelines, except that confirmatory scan not required if clinical progression requiring surgery or radiation to relieve symptoms
• Willing and able to withhold anti-PD-1 treatment from the time of enrollment through at least 6 weeks after the first DOC1021 administration
• One or more lesions available for biopsy or resection expected to yield at least 50 mg and preferably 100 mg of tumor for generating DOC1021 and at least 1 measurable target tumor lesion evaluable after DOC1021 by RECIST version 1.1.
• Brain metastases allowed if stable after prior treatment
• Ability to receive leukapheresis (with filgrastim if clinically indicated) and perinodal injections of DOC1021 near regional nodes + weekly pIFN x 4 weeks (i.e., sufficient clinical stability and anticipated life expectancy to complete the treatment period and allow time for an immune response to develop).
• Females of reproductive potential must have a negative serum pregnancy test and agree to use effective contraception (as deter-mined appropriate for the patient by the investigator) during study treatment.
• Adequate kidney, liver, bone marrow function, and immune function, as follows:
• Hemoglobin ≥ 8.0 gm/dL (use of transfusion or other intervention to achieve is acceptable)
• Absolute neutrophil count (ANC) ≥ 1,500 cells/mm3
• Platelet count ≥ 75,000/mm3
• Calculated creatinine clearance (CrCl) \> 30 mL/min using Cockcroft and Gault formula: i. For males = (140 - age\[years\]) x (body weight \[kg\]) / (72 x serum creatinine \[mg/dL\]) ii. For females = 0.85 x value from male formula e. Total bilirubin ≤ 1.5 times upper limit of normal (ULN) except in patients with Gilbert's disease for which total bilirubin must be ≤ 3 times ULN f. Aspartate transaminase AST (SGOT) and alanine aminotransferase ALT (SGPT) ≤ 3 times the ULN (or ≤ 5.0 × ULN if liver metastases)
• Eastern Cooperative Oncology Group (ECOG) Performance Score 0 or 1
Exclusion Criteria:
• Patients who are pregnant or breastfeeding.
• Known active HIV or hepatitis infection. Patients with HIV that is well-controlled and have undetectable viral titers remain eligible. Patients with history of HCV adequately treated such that RNA viral load is negative also remain eligible.
• Any severe or uncontrolled medical condition or other condition that could affect participation in this study as determined by the investigator, including but not limited to uncontrolled or severe cardiac dis-ease, systemic autoimmune disorders requiring immunosuppression\*, autoimmune hyper/hypothyroidism, untreated viral hepatitis, autoimmune hepatitis (\*autoimmune disorders include but are not limited to rheumatoid arthritis, psoriasis and inflammatory bowel disease and immunosuppressive medications include DMARDs like methotrexate, TNF inhibitors, IL-6 receptor blockers, CD80/86 inhibitors, anti-CD20 and JAK inhibitors)
• Residual immune-related toxicities from prior immunotherapy \> Grade 1 severity. However, patients who experienced prior endocrine toxicity are eligible if well-controlled on replacement therapy.
• Treatment with another investigational drug or other experimental intervention within the last 30 days.
BIOLOGICAL: DOC1021, PROCEDURE: Tumor resection, DRUG: pIFN (peginterferon alfa-2a)
Refractory Melanoma, Melanoma, skin
Dendritic Cell Vaccine, Immunotherapy, Tumor Vaccine
UT Southwestern
A Study of Amivantamab in Addition to Standard of Care Agents (SOC) Compared With SOC Alone in Participants With Recurrent/Metastatic Head and Neck Cancer (OrigAMI-5)
The purpose of this study is to compare anti-tumor activity of amivantamab in addition to pembrolizumab and carboplatin versus pembrolizumab, 5-fluorouracil (FU), and platinum therapy (carboplatin or cisplatin) in participants with refractory/metastatic (R/M) head and neck squamous cell carcinoma (HNSCC). HNSCC is a type of cancer that develops in the head and neck regions, including the outer tissue layer of the mouth and throat. This study will focus on participants with HNSCC who are treatment-naive (have not received prior treatment) in the R/M setting.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Yu Cao
ALL
18 Years to old
PHASE3
NCT07276399
STU20260092
Inclusion criteria:
* Be more than or equal to (\>=) 18 years of age (or the legal age of majority in the jurisdiction in which the study is taking place, whichever is greater)
* Have histologically or cytologically confirmed recurrent/metastatic (R/M) HNSCC that is considered incurable by local therapies: a. eligible primary tumor locations are the oral cavity, oropharynx, hypopharynx, or larynx; b. Must not have a primary tumor site of nasopharynx or primary tumor of unknown location; c. Must have documented local testing results per local regulations; d. Human papillomavirus (HPV) status must be known for participants with primary tumor location in oropharynx via p16 test, HPV DNA test, or high-risk HPV in situ hybridization (ISH). Any known p16, HPV DNA, or high-risk HPV ISH status of tumor must be negative
* Be treatment-naive for systemic therapy in the R/M setting
* Have an ECOG performance status of 0 or 1
* Have measurable disease according to Response Evaluation Criteria in Solid Tumors (RECIST) version (v).1.1
Exclusion criteria:
* Have an uncontrolled illness
* Have untreated brain metastases or history of known presence of leptomeningeal disease
* Have a history of clinically significant cardiovascular disease
* Inadequate organ or bone marrow function
* Known allergies, hypersensitivity, contraindications, or intolerance to excipients of: Amivantamab, Pembrolizumab, Carboplatin, Cisplatin, 5-FU and Hyaluronidase
BIOLOGICAL: Amivantamab, BIOLOGICAL: Pembrolizumab, DRUG: Carboplatin, DRUG: 5-Flurouracil, DRUG: Cisplatin
Squamous Cell Carcinoma of Head and Neck, Larynx, Lip, Oral Cavity and Pharynx
UT Southwestern
A Study Comparing JNJ-79635322 and an Anti-B-cell Maturation Antigen (BCMA)xCD3 Bispecific Antibody in Participants With Relapsed or Refractory Multiple Myeloma (TRIlogy-4)
The purpose of this study is to evaluate how well JNJ-79635322 works when compared with an anti-B-cell maturation antigen (BCMA)xCD3 bispecific antibody.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Larry Anderson
ALL
18 Years to old
PHASE3
NCT07258511
STU20260692
Inclusion:
* Documented diagnosis of multiple myeloma (MM) as defined by the criteria below:
• MM diagnosis according to the international myeloma working group (IMWG) diagnostic criteria
• Measurable disease at screening as assessed by central laboratory * Received at least 3 prior lines of antimyeloma therapy including a proteasome inhibitor (PI), an immunomodulatory drug (IMiD), and an anti-cluster of differentiation (CD)38 antibody * Documented evidence of progressive disease (PD) or failure to achieve a response to the last line of therapy based on investigator's determination of response by IMWG criteria * Toxicity related to previous anticancer therapy must have resolved to Grade 1 or better * Have an eastern cooperative oncology group (ECOG) performance status of 0 to 2 at screening and immediately before the start of study treatment administration Exclusion: * Active hepatitis of infectious origin * Known active or prior central nervous system (CNS) involvement or exhibits clinical signs of meningeal involvement of MM * Suspected or known allergies, hypersensitivity, or intolerance to the excipients of JNJ-79635322 and Teclistamab * Major surgery, (example, requiring general anesthesia) within 2 weeks before first dose, or will not have fully recovered from surgery, or has surgery planned during the time the participant is expected to participate in the study * Received or plans to receive any live, attenuated vaccine within 4 weeks before the first dose of study treatment, or during study treatment
• MM diagnosis according to the international myeloma working group (IMWG) diagnostic criteria
• Measurable disease at screening as assessed by central laboratory * Received at least 3 prior lines of antimyeloma therapy including a proteasome inhibitor (PI), an immunomodulatory drug (IMiD), and an anti-cluster of differentiation (CD)38 antibody * Documented evidence of progressive disease (PD) or failure to achieve a response to the last line of therapy based on investigator's determination of response by IMWG criteria * Toxicity related to previous anticancer therapy must have resolved to Grade 1 or better * Have an eastern cooperative oncology group (ECOG) performance status of 0 to 2 at screening and immediately before the start of study treatment administration Exclusion: * Active hepatitis of infectious origin * Known active or prior central nervous system (CNS) involvement or exhibits clinical signs of meningeal involvement of MM * Suspected or known allergies, hypersensitivity, or intolerance to the excipients of JNJ-79635322 and Teclistamab * Major surgery, (example, requiring general anesthesia) within 2 weeks before first dose, or will not have fully recovered from surgery, or has surgery planned during the time the participant is expected to participate in the study * Received or plans to receive any live, attenuated vaccine within 4 weeks before the first dose of study treatment, or during study treatment
DRUG: JNJ-79635322, DRUG: Teclistamab
Multiple Myeloma
UT Southwestern
Phase 2A/B Efficacy and Safety of Dabogratinib in Participants With Low Grade Upper Tract Urothelial Carcinoma (SURF303)
A Phase 2A/B study of Dabogratinib (TYRA-300) in Low Grade Upper Tract Urothelial Carcinoma
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Yair Lotan
ALL
18 Years to old
PHASE2
NCT07265947
STU20260411
• Participants ≥ 18 years of age at the time of informed consent and willing and able to comply with all required study procedures
• Confirmed LOW RISK LG UTUC (both favorable and unfavorable) per AUA
• At least 5mm of marker lesion left behind
• Participants must have previous genomic report or archival/fresh tissue in addition to urine sample for retrospective genomic testing
• Identification of marker lesion(s) within 8 weeks prior to randomization (refer to Inclusion Criterion #2)
• If synchronous NMIBC, NMIBC must be fully resected and low-grade Ta or T1
• No prior BCG administration within 1 year of date of consent.
• No intravesical chemotherapy within 8 weeks prior to C1D1 (including UGN-101).
• No systemic chemotherapy within 3 months prior to C1D1
• ECOG 0-2
• Pathology consists of pure urothelial carcinoma
• Adequate bone marrow, liver, and renal function:
• i. Absolute neutrophil count (ANC) ≥1,500/mm3 ii. Platelet count ≥75,000/mm3 iii. Hemoglobin ≥10.0 g/dL
• i. Total bilirubin ≤ ULN ii. Alanine aminotransferase (ALT) ≤ ULN iii. Aspartate aminotransferase (AST) ≤ ULN
• Estimated glomerular filtration rate \>60 mL/min
• Serum Phosphate level ≤ ULN prior to starting treatment
• International normalized ratio (INR) ≤1.5 × ULN
Exclusion Criteria:
• Evidence or any features of high grade (HG) UTUC
• History of carcinoma in situ (CIS)
• History of prostatic urethral involvement
• Current or previous history of muscle invasive bladder cancer
• Current or previous history of lymph node positive and/or metastatic bladder cancer
• Evidence of squamous cell carcinoma, adenocarcinoma or undifferentiated carcinoma or small cell of the bladder
• Currently receiving systemic cancer therapy (cytotoxic or immunotherapy)
• Current or prior history of pelvic external beam radiotherapy for bladder cancer
• Current or history of receiving a prior FGFR inhibitor
• Systemic immunotherapy within 6 months prior to randomization
• Treatment with an investigational agent within 30 days or 5 half-lives from randomization, whichever is shorter; compounds with an unknown half-life will be default to 30 days.
• Prior treatment with an intravesical or intracavitary agent within 8 weeks of C1D1.
• Current evidence of central serous retinopathy or retinal pigmented epithelial detachment of any grade at time of baseline examination.
• Requiring use of medications that are potential inhibitors or inducers of CYP3A (prohibited list of medications)
DRUG: Dabogratinib (TYRA-300) 60mg, DRUG: Dabogratinib (TYRA-300) 80mg, DRUG: Dabogratinib (TYRA-300) TBD
Low Grade Upper Tract Urothelial Carcinoma
Low-grade UTUC, Upper Tract Urothelial Carcinoma, Low Grade Upper Tract Urothelial Carcinoma, FGFR Gene Amplification, FGFR Gene Alteration, FGFR Gene Alterations, FGFR3 Mutation, FGFR3 Mutations, FGFR3 Gene Fusions
UT Southwestern