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441 Study Matches
Augmented Reality-Cardiopulmonary Resuscitation Support for Pediatric Resuscitation (AR-CPR)
This study evaluates whether an augmented reality cardiopulmonary resuscitation feedback system (AR-CPR) can support health care providers in delivering high-quality chest compressions during pediatric cardiac arrest. AR-CPR provides real-time visual feedback on chest compression rate, depth, and recoil through a head-mounted augmented reality display. In this randomized, multicenter, international, simulation-based non-inferiority study, health care providers perform chest compressions during an 18-minute simulated pediatric cardiac arrest. Participants are assigned to receive either real-time feedback from AR-CPR or coaching from a trained human CPR coach. The primary objective is to determine whether the percentage of chest compressions meeting guideline targets for both rate and depth with AR-CPR is non-inferior to that achieved with human CPR coaching.
studyfinder@utsouthwestern.edu
ALL
18 Years to old
NA
NCT07778823
Inclusion Criteria:
* Actively credentialed pediatric emergency department or pediatric intensive care unit nurse or clinical technician at a participating study site
* Current American Heart Association (AHA) Pediatric Advanced Life Support (PALS) certification
Exclusion Criteria:
* Inability to physically perform chest compressions
* Need for prescription corrective lenses without having those corrective lenses available at the time of study participation DEVICE: AR-CPR
Cardiac Arrest (CA), Pediatric Cardiac Arrest (Simulated), Cardiopulmonary Resuscitation (CPR)
augmented reality, cardiopulmonary resuscitation, pediatric cardiac arrest, cpr quality, chest compressions, cpr coaching, real-time feedback, pediatric advanced life support, simulation
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
Adaptive Dosing of Immune Checkpoint Inhibitors for Hepatocellular Carcinoma
The goal of this clinical trial is to learn if immunotherapy including nivolumab plus ipilimumab is effective and safe in treating hepatocellular carcinoma.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
David Hsieh
ALL
18 Years to old
PHASE2
NCT07689175
STU20261069
Inclusion Criteria:
• Patient must have a diagnosis confirmed by histology or clinically by the American Association for the Study of Liver Diseases (AASLD) criteria in patients with cirrhosis. Known fibrolamellar HCC or combined HCC-cholangiocarcinoma will be excluded.
• Patients may not have received any prior anti-PD-1/L1 or anti-CTLA-4 therapies for the treatment of advanced HCC.
• Patients with locally advanced or metastatic disease must have disease deemed not amenable to surgical and/or locoregional therapies or patients who have progressed following surgical and/or locoregional therapies.
• Child-Pugh Score B7-8
• Measurable disease, as defined as lesions that can accurately be measured in at least one dimension according to RECIST v.1.1.
• Prior locoregional therapy is allowed provided the target lesion has increased in size ≥25% since the cessation of locoregional therapy or the target lesion was not treated with locoregional therapy. Patients treated with palliative radiotherapy for symptoms will be eligible as long as the target lesion is not the treated lesion.
• Age ≥ 18 years.
• ECOG performance score 0-2
• Adequate organ and marrow function as defined below: Platelet count ≥ 40,000/mm3 Hgb ≥ 8 g/dl INR ≤ 2 AST, ALT ≤ 5 times ULN Calculated creatinine clearance (CrCl) ≥ 35 mL/min. CrCl can be calculated using the Cockcroft-Gault method. Albumin ≥ 2.0 g/dl
• 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 120 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. 10a. 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). 11\. Ability to understand and the willingness to sign a written informed consent.
Exclusion Criteria:
• Prior solid organ transplant.
• Hypersensitivity to IV contrast; not suitable for pre-medication.
• Subjects may not be receiving any other investigational agents for the treatment of the cancer under study.
• Active autoimmune disease that requires current systemic treatment (ie, with use of disease-modifying agents, corticosteroids, or immunosuppressive drugs), except for conditions that, in the investigator's opinion, do not have a substantial hazardous risk such as asthma, and cutaneous and musculoskeletal rheumatologic conditions.
• Known human immunodeficiency virus infection (testing not required) in a patient not on antiretroviral therapy and detectable viral load.
• Prior malignancy that required systemic treatment within the previous year except for locally curable cancers that have been apparently cured, such as basal or squamous cell skin cancer, superficial bladder cancer, or carcinoma in situ of the cervix, breast, or prostate cancer. Preneoplastic or malignant diagnoses that are indolent in nature and do not require active systemic treatment are not excluded.
• If a participant has symptomatic or clinically active brain metastases including leptomeningeal disease, they must be excluded if: * Has evidence of progression by neurologic symptoms * Has metastatic brain lesions that require immediate intervention. * Has carcinomatous meningitis, regardless of clinical stability
• Known severe hypersensitivity reactions to monoclonal antibodies (≥Grade 3).
• 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.
• Subjects must not be pregnant or nursing due to the potential for congenital abnormalities and the potential of this regimen to harm nursing infants.
• Prisoners or subjects who are involuntarily incarcerated.
• Has significant dementia or other mental condition that precludes the participant's ability to consent to the study.
DRUG: Nivolumab, DRUG: Ipilimumab (3 mg/kg)
Hepatocellular Carcinoma (HCC)
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
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 Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT) (ENERGIZEKids-T)
The primary objective of this study is to compare the effect of mitapivat versus placebo on transfusion burden in pediatric participants with α- or β-transfusion-dependent thalassemia.
studyfinder@utsouthwestern.edu
ALL
1 Year to 17 Years old
PHASE3
NCT07506863
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.
* Transfusion dependent, defined as 6 to 20 transfusion episodes (also referred to as "transfusion events") and a ≤6-week transfusion-free period during the 24-week period before randomization.
* 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 ≥36 weeks before randomization.
* Currently receiving treatment with hematopoietic stimulating agents; the last dose must have been administered ≥36 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) ≤ 6 months 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 Matched Placebo, DRUG: Mitapivat
Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia
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
AMAZE 12: A Research Study Investigating How Well the Medicine NNC0487-0111 Helps People With Excess Body Weight Maintain Their Weight Loss (AMAZE 12)
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. 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 (a treatment that has no active medicine in it). Which treatment participants get is decided by chance.
studyfinder@utsouthwestern.edu
ALL
18 Years to old
PHASE3
NCT07503210
Inclusion Criteria:
* Male or female (sex at birth).
* Age 18 years or above at the time of signing the informed consent.
Exclusion Criteria:
* Glycated haemoglobin (HbA1c) greater than or equal to ≥ 6.5% \[48 millimoles per mole (mmol/mol)\] as measured by the central laboratory at screening.
* History of type 1 or type 2 diabetes mellitus as declared by the participant or reported in the medical records.
* 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)
Obesity
A Study of Guselkumab Versus Risankizumab in Participants With Moderately to Severely Active Crohn's Disease (CHARGE)
The purpose of this study is to assess how well guselkumab works when compared to risankizumab in participants with moderately to severely active Crohn's Disease (CD; a long-term condition causing severe inflammation of the intestinal tract).
studyfinder@utsouthwestern.edu
ALL
18 Years to old
PHASE3
NCT07499232
Inclusion criteria:
* Has CD or fistulizing Crohn's Disease (CD) of at least 12 weeks' duration, with colitis, ileitis, or ileocolitis, confirmed at some time in the past by radiography, histology, and/or endoscopy
* Have moderately to severely active CD, defined as baseline Crohn's Disease Activity Index (CDAI) score greater than or equal to (\>=) 220 but less than or equal to (\<=) 450
* Baseline endoscopic evidence of active ileal and/or colonic CD as assessed by central endoscopy reading at the screening endoscopy defined as a screening Simple Endoscopic Score for Crohn's Disease (SES CD) \>= 4 (for participants with isolated ileal disease) or \>= 6 (for participants with colonic or ileocolonic disease), based on the presence of ulceration in any 1 of the 5 ileocolonic segments, resulting in the following specified ulceration component scores:
• a minimum score of 1 for the component of "size of ulcers" AND
• a minimum score of 1 for the component of "ulcerated surface" * In the opinion of the investigator, participant's disease is appropriate to treat with the maintenance dosing regimens utilized in the study * Adhere to the requirements for concomitant medications for the treatment of CD as mentioned in the protocol Exclusion criteria * Has complications of CD such as symptomatic strictures or stenoses, short gut syndrome, active draining stoma or significant fistulizing disease or any other manifestation anticipated to require surgery within the next year, could preclude the use of the CDAI to assess response to therapy, or would possibly confound the ability to assess the effect of treatment with guselkumab or risankizumab * Currently has or is suspected to have an abscess * Has an active fistula during screening or at Week 0 with an anticipated need for surgery * Has had any kind of bowel resection within 24 weeks, or any other intra-abdominal or other major surgery within 12 weeks, before first dose of study intervention * Currently has a malignancy or has a history of malignancy within 5 years before screening
• a minimum score of 1 for the component of "size of ulcers" AND
• a minimum score of 1 for the component of "ulcerated surface" * In the opinion of the investigator, participant's disease is appropriate to treat with the maintenance dosing regimens utilized in the study * Adhere to the requirements for concomitant medications for the treatment of CD as mentioned in the protocol Exclusion criteria * Has complications of CD such as symptomatic strictures or stenoses, short gut syndrome, active draining stoma or significant fistulizing disease or any other manifestation anticipated to require surgery within the next year, could preclude the use of the CDAI to assess response to therapy, or would possibly confound the ability to assess the effect of treatment with guselkumab or risankizumab * Currently has or is suspected to have an abscess * Has an active fistula during screening or at Week 0 with an anticipated need for surgery * Has had any kind of bowel resection within 24 weeks, or any other intra-abdominal or other major surgery within 12 weeks, before first dose of study intervention * Currently has a malignancy or has a history of malignancy within 5 years before screening
DRUG: Guselkumab, DRUG: Risankizumab
Crohn Disease
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
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia (ASPEN)
This is a multicenter, multinational, randomized, active-controlled, operationally seamless Phase 2/3 study of BMN 333 in treatment-naïve pediatric participants with achondroplasia (ACH). The study consists of a Phase 2 part and a Phase 3 part.
studyfinder@utsouthwestern.edu
ALL
2 Years to 17 Years old
PHASE2
NCT07441876
Inclusion Criteria:
• Participants must be aged ≥ 2 to \< 11 years (Phase 2) or ≥ 2 to \< 18 years (Phase 3), at the time of signing the informed consent
• Participants must have ACH (confirmed by documented genetic testing) and open epiphyses
• Are Tanner Stage I (Phase 2) or any Tanner stage (Phase 3)
• Are ambulatory and able to stand without assistance
Exclusion Criteria:
• Have any short stature condition other than ACH (eg, hypochondroplasia, trisomy 21, pseudoachondroplasia, GH deficiency)
• Have any of the following disorders: Hypothyroidism or hyperthyroidism, unless treated with evidence of normalized thyroid-stimulating hormone (TSH) levels, diabetes mellitus, unless considered well-controlled, autoimmune inflammatory disease, inflammatory bowel disease, autonomic neuropathy, anemia defined as hemoglobin \< 10 g/dL, vitamin D deficiency, significant hip pathology.
• Have history of any renal insufficiency or cardiac/ cardiovascular disease that places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension.
• Have had bone fractures of the long bones or spine within 6 months prior to screening.
• Have used vosoritide, any other approved product (except GH, as detailed below), investigational product, or investigational medical device for the treatment of ACH or short stature at any time
• Have been treated with GH, insulin-like growth factor 1, or anabolic steroids in the 6 months prior to treatment start
DRUG: BMN 333, DRUG: Vosoritide Injection [Voxzogo]
Achondroplasia
Achondroplasia, ACH, Bone Diseases, Developmental Dwarfism, Bone Diseases, Genetic Diseases, Inborn, Musculoskeletal Diseases, Natriuretic Peptide, C-type, Osteochondrodysplasias, Physiological Effects of Drugs, Skeletal Dysplasias
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
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 Study of Eloralintide (LY3841136) in Participants With Persistent Obesity Who Are Treated With a Weekly Incretin (ENLIGHTEN-6)
The main purpose of this study is to evaluate the efficacy and safety of eloralintide compared with placebo in participants with persistent obesity or overweight, with or without type 2 diabetes, and on stable incretin background therapy. Participation in the study will last about 80 weeks.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Elaine.Konda@UTSouthwestern.edu
Amy Shah
ALL
18 Years to old
PHASE3
NCT07392190
STU20252255
Inclusion Criteria:
* Are on stable incretin therapy at screening
* With persistent obesity or overweight defined as:
* ≥30 kg/m2 OR
* ≥27 kg/m2 with at least one existing obesity related complication at screening:
* hypertension
* dyslipidemia
* obstructive sleep apnea
* cardiovascular disease (for example, ischemic cardiovascular disease, New York Heart Association Functional Class I-III heart failure), or
* type 2 diabetes
* Have a stable body weight (\<5% body weight change) at screening
Exclusion Criteria:
* Have a prior or planned surgical treatment for obesity (liposuction, cryolipolysis, or abdominoplasty allowed if performed \>1 year before screening)
* Have a prior or planned endoscopic procedure and/or device-based therapy for obesity (prior device-based therapy acceptable if device removal was more than 6 months prior to screening)
* Have type 1 diabetes
* Have taken any of the following antihyperglycemic medications within 90 days before screening:
* dipeptidyl peptidase-4 (DPP-4) inhibitors
* amylin analogs
* insulin
* Have had within 90 days prior to screening:
* heart attack
* stroke
* coronary artery revascularization
* unstable angina, or
* hospitalization due to congestive heart failure
* Have a history or diagnosis of New York Heart Association Functional Classification Class IV congestive heart failure DRUG: Eloralintide, DRUG: Placebo
Overweight, Obesity
Amylin receptor agonist
UT Southwestern
A Study Comparing Higher Dose Chemotherapy Over a Shorter Amount of Time to Lower Dose Chemotherapy Plus Maintenance Over a Longer Amount of Time in Patients With Newly Diagnosed Intermediate-Risk Rhabdomyosarcoma (IR RMS)
This phase III trial compares higher dose chemotherapy, with vincristine, dactinomycin and cyclophosphamide, over a shorter amount of time to lower dose chemotherapy plus maintenance, with vincristine, dactinomycin, cyclophosphamide, irinotecan and vinorelbine, over a longer amount of time, along with standard of care surgery and radiation, in patients with newly diagnosed intermediate risk rhabdomyosarcoma. Vincristine and vinorelbine are in a class of medications called vinca alkaloids. They work by stopping tumor cells from growing and dividing and may kill them. Dactinomycin is a type of antibiotic that is only used in cancer chemotherapy (antineoplastic antibiotic). It works by damaging the cell's DNA and may kill tumor cells. Cyclophosphamide is in a class of medications called alkylating agents. It works by damaging the cell's DNA and may kill tumor cells. It may also lower the body's immune response. Irinotecan is in a class of antineoplastic medications called topoisomerase I inhibitors. It blocks a certain enzyme needed for cell division and DNA repair and may kill tumor cells. It is not yet known if the higher dose chemotherapy over a shorter amount of time or the lower dose chemotherapy with maintenance over a longer amount of time is more effective in the treatment of patient with newly diagnosed, intermediate risk rhabdomyosarcoma.
studyfinder@utsouthwestern.edu
ALL
to 50 Years old
PHASE3
NCT07466316
Inclusion Criteria:
* Patient must be ≤ 50 years of age at the time of enrollment
* Patients with newly diagnosed soft tissue RMS of any subtype, except adult-type pleomorphic, based upon institutional histopathologic classification, are eligible to enroll on the study based upon FOXO1 fusion status, Stage, Intergroup Rhabdomyosarcoma Study (IRS) group, and age, as below. FOXO1 fusion status must be determined prior to enrollment. RMS types included under embryonal rhabdomyosarcoma (ERMS) include those which are reclassified in the 2020 World Health Organization (WHO) classification as ERMS (typical, dense and botryoid variants) and spindle cell/sclerosing RMS (encompassing the historical spindle cell ERMS variant and the newly recognized sclerosing RMS variant). Classification of alveolar Rhabdomyosarcoma (ARMS) in the 2020 WHO Classification is the same as in the International Classification of Rhabdomyosarcoma (ICR) and includes classic and solid variants.
* FOXO1 fusion negative (FN)
* Stage 2/3, Group III
* Stage 4, Group IV, \< 10 years old
* FOXO1 fusion positive (FP)
* Stages 1-3, Groups I-III
* Disease/staging imaging studies, if applicable, must be obtained within 21 days prior to enrollment and start of protocol therapy (repeat if necessary)
* FOXO1 status results must be available to enroll. All patients will undergo institutional pathology review and institutional FOXO1 fusion determination regardless of histology prior to enrollment. FOXO1 status may confirmed by cytogenetic, fluorescence in situ hybridization (FISH), or next generation sequencing techniques. FOXO1 fusion results should be SUBMITTED as an upload to RAVE at study enrollment because this information is required for randomization.
Please note the following:
* Institutional PAX3 versus (vs.) PAX7 determination is not required but should be submitted if available. Institutional FOXO1 testing may be performed at a contract or commercial lab as long as reports can be submitted and uploaded to RAVE. Additional molecular pathology reports, including the MCI report, are not required but should be submitted if available.
* Patients who are \< 10 years old with distant metastatic disease (Stage 4) who have institutional molecular testing indicating fusion negative (FN) RMS but are later found to have fusion positive (FP) RMS by MCI or other testing will be considered to have metastatic FP disease and will go off study
* Appropriate lymph node sampling based on primary site of disease is required
* Patients must have a performance status of Lansky performance status score ≥ 50 for patients ≤ 16 years of age or Karnofsky performance status score ≥ 50 for patients \> 16 years of age
* Peripheral absolute neutrophil count (ANC) ≥ 750/μL (All laboratory studies to determine eligibility must be performed within 7 days prior to enrollment unless otherwise indicated. Laboratory studies must be repeated prior to the start of protocol therapy if \> 7 days have elapsed from their most recent prior assessment. Laboratory tests need not be repeated if therapy starts within seven (7) days of their most recent prior assessment. If the result of a laboratory study that is repeated at any time post-enrollment and prior to the start of protocol therapy is outside the limits for eligibility, then the evaluation must be rechecked within 48 hours prior to initiating protocol therapy. The results of the recheck must be within the limits for eligibility to proceed. If the result of the recheck is outside the limits of eligibility, the patient may not receive protocol therapy and will be considered off protocol therapy.)
* Platelet count ≥ 75,000/μL (All laboratory studies to determine eligibility must be performed within 7 days prior to enrollment unless otherwise indicated. Laboratory studies must be repeated prior to the start of protocol therapy if \> 7 days have elapsed from their most recent prior assessment. Laboratory tests need not be repeated if therapy starts within seven (7) days of their most recent prior assessment. If the result of a laboratory study that is repeated at any time post-enrollment and prior to the start of protocol therapy is outside the limits for eligibility, then the evaluation must be rechecked within 48 hours prior to initiating protocol therapy. The results of the recheck must be within the limits for eligibility to proceed. If the result of the recheck is outside the limits of eligibility, the patient may not receive protocol therapy and will be considered off protocol therapy.)
* For pediatric patients \< 18 years of age:
* A serum creatinine based on age/sex as follows:
* 1 month to \< 6 months: Maximum serum creatinine 0.4 mg/dL (male), 0.4 mg/dL (female)
* 6 months to \< 1 year: Maximum serum creatinine 0.5 mg/dL (male), 0.5 mg/dL (female)
* 1 to \< 2 years: Maximum serum creatinine 0.6 mg/dL (male), 0.6 mg/dL (female)
* 2 to \< 6 years: Maximum serum creatinine 0.8 mg/dL (male), 0.8 mg/dL (female)
* 6 to \< 10 years: Maximum serum creatinine 1 mg/dL (male), 1 mg/dL (female)
* 10 to \< 13 years: Maximum serum creatinine 1.2 mg/dL (male), 1.2 mg/dL (female)
* 13 to \< 16 years: Maximum serum creatinine 1.5 mg/dL (male), 1.4 mg/dL (female)
* ≥ 16 years: Maximum serum creatinine 1.7 mg/dL (male),1.4 mg/dL (female)
* OR a 24-hour urine Creatinine clearance ≥ 50 mL/min/1.73 m\^2
* OR a glomerular filtration rate (GFR) ≥ 70 mL/min/1.73 m\^2. GFR must be performed using direct measurement with a nuclear blood sampling method OR direct small molecule clearance method (iothalamate or other molecule per institutional standard).
* Note: Estimated GFR (eGFR) from serum creatinine, cystatin C or other estimates are not acceptable for determining eligibility.
* Patients with an elevated serum creatinine due to obstructive hydronephrosis secondary to tumor are still eligible. However, patients with urinary tract obstruction by tumor must have unimpeded urinary flow established via diversion (ie, percutaneous nephrostomies or ureteric stents) of the urinary tract.
For adult patients (aged 18 years or older):
* Creatinine clearance ≥ 50 mL/min, as estimated by the Cockcroft and Gault formula or as a 24-hour urine collection. Estimated creatinine clearance is based on actual body weight.
(All laboratory studies to determine eligibility must be performed within 7 days prior to enrollment unless otherwise indicated. Laboratory studies must be repeated prior to the start of protocol therapy if \> 7 days have elapsed from their most recent prior assessment. Laboratory tests need not be repeated if therapy starts within seven (7) days of their most recent prior assessment. If the result of a laboratory study that is repeated at any time post-enrollment and prior to the start of protocol therapy is outside the limits for eligibility, then the evaluation must be rechecked within 48 hours prior to initiating protocol therapy. The results of the recheck must be within the limits for eligibility to proceed. If the result of the recheck is outside the limits of eligibility, the patient may not receive protocol therapy and will be considered off protocol therapy.)
* Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age (All laboratory studies to determine eligibility must be performed within 7 days prior to enrollment unless otherwise indicated. Laboratory studies must be repeated prior to the start of protocol therapy if \> 7 days have elapsed from their most recent prior assessment. Laboratory tests need not be repeated if therapy starts within seven (7) days of their most recent prior assessment. If the result of a laboratory study that is repeated at any time post-enrollment and prior to the start of protocol therapy is outside the limits for eligibility, then the evaluation must be rechecked within 48 hours prior to initiating protocol therapy. The results of the recheck must be within the limits for eligibility to proceed. If the result of the recheck is outside the limits of eligibility, the patient may not receive protocol therapy and will be considered off protocol therapy.)
* If there is evidence of biliary obstruction by tumor, then total bilirubin must be \< 3 x ULN for age
* Serum glutamic pyruvic transaminase (SGPT) (alanine aminotransferase \[ALT\]) ≤ 135 U/L (All laboratory studies to determine eligibility must be performed within 7 days prior to enrollment unless otherwise indicated. Laboratory studies must be repeated prior to the start of protocol therapy if \> 7 days have elapsed from their most recent prior assessment. Laboratory tests need not be repeated if therapy starts within seven (7) days of their most recent prior assessment. If the result of a laboratory study that is repeated at any time post-enrollment and prior to the start of protocol therapy is outside the limits for eligibility, then the evaluation must be rechecked within 48 hours prior to initiating protocol therapy. The results of the recheck must be within the limits for eligibility to proceed. If the result of the recheck is outside the limits of eligibility, the patient may not receive protocol therapy and will be considered off protocol therapy.)
* Note: For the purpose of this study, the ULN for SGPT (ALT) has been set to the value of 45 U/L.
* Known HIV-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial
Exclusion Criteria:
* Patients with evidence of uncontrolled infection are not eligible
* Previous or concurrent cancer(s) that is/was being treated with chemotherapy and/or radiation.
* Note: Surgical resection alone of previous or concurrent cancer(s) is allowed
* Patients with central nervous system involvement of RMS as defined below:
* Malignant cells detected in cerebrospinal fluid
* Intra-parenchymal brain metastases separate and distinct from primary tumor (i.e., direct extension from parameningeal primary tumors is allowed)
* Diffuse leptomeningeal disease
* Patients with known Charcot-Marie-Tooth disease
* Patients who have received any chemotherapy (excluding steroids) and/or radiation therapy for RMS prior to enrollment. Note: the following exception:
* Patients requiring emergency radiation therapy for life-threatening complications of tumor burden due to RMS. These patients are eligible, provided they are consented to ARST2531 prior to administration of radiation.
* Note: Patients who have received or are receiving chemotherapy or radiation for non-malignant conditions (eg, autoimmune diseases) are eligible. Patients must discontinue chemotherapy for non-malignant conditions prior to starting protocol therapy
* Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential
* Lactating females who plan to breastfeed their infants
* Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation PROCEDURE: Biospecimen Collection, PROCEDURE: Bone Marrow Aspiration, PROCEDURE: Bone Marrow Biopsy, PROCEDURE: Bone Scan, PROCEDURE: Computed Tomography, DRUG: Cyclophosphamide, BIOLOGICAL: Dactinomycin, DRUG: Irinotecan Hydrochloride, PROCEDURE: Lumbar Puncture, PROCEDURE: Lymph Node Biopsy, PROCEDURE: Magnetic Resonance Imaging, PROCEDURE: Positron Emission Tomography, RADIATION: Radiation Therapy, PROCEDURE: Resection, OTHER: Survey Administration, DRUG: Vincristine Sulfate, DRUG: Vinorelbine Tartrate
Rhabdomyosarcoma
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
A Study of CX11 Tablets in Patients With Type 2 Diabetes Mellitus
This study is testing whether a new medication called CX11 works and is safe for participants with type 2 diabetes who have not reached good blood sugar control while taking a steady dose of metformin, with or without a steady dose of an SGLT2 inhibitor, for at least 90 days. The study is being done at multiple medical centers. Participants are assigned by chance (randomized) to different groups, and neither the participants nor the study staff know which group they're in (double-blind). The groups are compared side by side (parallel), and some participants will receive inactive pills (placebo) to help measure the true effect of the study drug. After screening, participants will be randomly placed into one of six groups, with equal chances of being in any group. Each group will receive a different dose of CX11 or a placebo. Treatment will last 24 weeks. After that, all participants will have a 2-week follow-up period to check on safety.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Emily.Zhang@UTSouthwestern.edu
Emily Zhang
ALL
18 Years to 75 Years old
PHASE2
NCT07340320
STU20252558
Inclusion Criteria
Participants who meet all of the following criteria will be eligible to participate in this study:
* Adults aged 18 to 75.
* Diagnosis of type 2 diabetes for at least 6 months.
* HbA1c between 7.0% and 10.5%.
* Body mass index (BMI) between 23 and 50 kg/m².
* Body weight stable for the past 3 months before joining.
* Stable dose of metformin (≥1000 mg/day), with or without SGLT2i, for ≥3 months.
* Women of childbearing potential (WOCBP): highly effective contraception ≥6 months prior to screening, throughout study, and 90 days post-last dose; negative pregnancy test within 24 hrs of first dose; no intent to donate sperm/ova
* Agrees to avoid grapefruit/grapefruit products
Exclusion Criteria
Participants who meet any of the following criteria will be excluded from this study:
* Anticipated initiation or change in concomitant medications (for more than 14 consecutive days) known to affect weight or glucose metabolism (e.g. treatment with orlistat, thyroid hormones, or systemic corticosteroids).
* Type 1 diabetes or a history of diabetic ketoacidosis.
* Use of any GLP-1 receptor agonist within the past 6 months, or any prior exposure to CX11.
* Use of insulin to control blood sugar within the past 12 months.
* More than one episode of severe low blood sugar, with awareness of hypoglycemia symptoms.
* Cardiovascular or cerebrovascular conditions within the past 6 months:
* Heart attack, coronary angioplasty, or bypass surgery (diagnostic angiography allowed).
* Valvular heart disease or prior heart valve repair surgery.
* Unstable angina.
* Transient ischemic attack (TIA) or stroke.
* Decompensated heart failure (NYHA Class III or IV).
* ECG abnormalities indicating significant safety risk, such as supraventricular tachycardia, torsades de pointes, second- or third-degree AV block, myocardial infarction, QTcF \> 450 ms in males or \> 470 ms in females, PR interval \> 220 ms.
* Poorly controlled hypertension at screening: systolic ≥ 180 mmHg or diastolic ≥ 100 mmHg.
* Pancreatic or gallbladder conditions:
* Acute or chronic pancreatitis.
* Symptomatic gallbladder disease (previous cholecystectomy is allowed).
* Pancreatic injury or risk factors that increase pancreatitis risk.
* Thyroid conditions:
* Poorly controlled abnormal thyroid function on a stable dose before screening.
* Clinically significant abnormal thyroid test results at screening.
* Personal or first-degree family history of medullary thyroid carcinoma or multiple endocrine neoplasia (MEN) type 2A or 2B.
* Cancer history:
* Malignancy within the past 5 years, regardless of recurrence or metastasis. Exceptions: localized basal cell skin cancer, low-risk prostate cancer, cervical carcinoma in situ, or high-grade prostatic intraepithelial neoplasia.
* Gastrointestinal conditions or treatments that may affect drug absorption:
* Abnormal gastric emptying (e.g., gastric outlet obstruction).
* Severe chronic gastrointestinal disease, including active ulcer within 6 months.
* Crohn's disease, ulcerative colitis, or other inflammatory bowel diseases.
* Prior gastrointestinal surgery (except polypectomy and appendectomy).
* Long-term use of drugs that directly affect gastrointestinal motility (e.g., mosapride, cisapride).
* Liver disease:
* Active liver disease other than nonalcoholic fatty liver.
* Chronic active hepatitis B or C.
* Primary biliary cirrhosis.
* Eye disease:
* Uncontrolled or potentially unstable diabetic retinopathy or maculopathy.
* Abnormal lab results at screening:
* eGFR \< 60 mL/min/1.73 m² (CKD-EPI).
* ALT or AST \> 2.5 × upper limit of normal (ULN).
* Total bilirubin \> 1.5 × ULN (except known Gilbert's syndrome).
* Serum amylase or lipase \> 1.5 × ULN.
* Fasting triglycerides \> 5.7 mmol/L.
* TSH \> 1.5 × ULN or \< 1.0 × LLN.
* Calcitonin ≥ 20 ng/L.
* Hemoglobin \< 110 g/L (male) or \< 100 g/L (female).
DRUG: CX11, OTHER: Placebo
Type II Diabetes Mellitus
Type 2 Diabetes Mellitus, HbA1C, Percentage change in HbA1C, Change in Fasting Plasma Glucose, Change in body weight, Body weight loss, Hypoglycemics episodes, Hypoglycemia, Continuous Glucose Monitoring, Blood glucose, Health Survey Short Form-36, Diabetes treatment satisfaction questionnaire, Columbia-suicide severity rating scale
UT Southwestern
A Study of Eloralintide (LY3841136) in Participants With Obesity, or Overweight Without Type 2 Diabetes (ENLIGHTEN-1)
The purpose of this study is to evaluate the efficacy and safety of eloralintide in adults with obesity or overweight who do not have type 2 diabetes. The study has two phases: a main phase and an extension phase. Participation in the main phase of the study will last about 75 weeks. Participants with prediabetes will continue in the extension phase for another 2 years.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Lina.GonzalezDuarte@UTSouthwestern.edu
Amy Shah
ALL
18 Years to old
PHASE3
NCT07321886
STU20252251
Inclusion Criteria:
* Have Body Mass Index (BMI) at screening of the following:
* 30 kilogram per square meter (kg/m2) OR
* 27 kg/m2 with at least one of the following weight-related health conditions at screening:
* high blood pressure
* dyslipidemia
* obstructive sleep apnea, or
* heart disease
* Have a stable body weight (\<5% body weight change) for 90 days prior to screening.
* Have a history of at least one self-reported unsuccessful dietary effort to reduce body weight
Exclusion Criteria:
* Have a prior or planned surgical treatment for obesity (liposuction, cryolipolysis, or abdominoplasty allowed if performed \>1 year before screening)
* Have a prior or planned endoscopic procedure and/or device-based therapy for obesity (prior device-based therapy acceptable if device removal was more than 6 months prior to screening)
* Have type 1 diabetes or type 2 diabetes
* Have had within 90 days prior to screening:
* heart attack
* stroke
* coronary artery revascularization
* unstable angina, or
* hospitalization due to congestive heart failure
* Have a history or diagnosis of New York Heart Association Functional Classification Class IV congestive heart failure
* Have taken medications or alternative remedies intended for weight loss within 90 days of screening DRUG: Eloralintide, DRUG: Placebo
Obesity, Overweight
UT Southwestern
Phase II Trial of PSA Response-based Androgen Deprivation Therapy and Nodal Coverage for Prostate Cancer Early Salvage Radiotherapy (RANGER) ((RANGER))
This Phase II, single arm study evaluates a PSA-response-adapted approach to salvage radiotherapy after radical prostatectomy for prostate cancer. All participants will receive hypo-fractionated stereotactic radiotherapy to the prostate fossa. At 5 weeks, biochemical response will be assessed. responders will proceed to observation, while non responders will receive sequential pelvic nodal radiotherapy and 4 months of androgen deprivation therapy (ADT). The study aims to determine whether this response base approach achieves non inferior 2 year freedom from progression compared with historical outcomes using routine pelvic nodal radiotherapy and ADT in all patients.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Aurelie Garant
MALE
18 Years to old
PHASE2
NCT07313241
STU20251220
Inclusion Criteria:
* Men aged ≥18 years with histologically confirmed prostate adenocarcinoma treated with prostatectomy in the localized setting within 10 years, with post-operative PSA (persistent or rising) of ≥0.05ng/mL.
* Radical prostatectomy ≥4 months prior to enrollment without nodal involvement (pN0 or pNx)
* Performance status ECOG 0-2
* No definite evidence of regional or distant metastatic disease by at least pelvic imaging within 90 days of registration. Equivocal findings are allowed at investigator discretion. Imaging is specified as follows:
* PSA\>=0.2ng/mL: positron emission tomography (PET) with FDA-approved advanced imaging agent for prostate cancer (e.g. PSMA) required.
* PSA \<0.2 n/gm: PET with above noted agents OR conventional CT or MRI at investigator discretion.
* All sexually active men must agree to use adequate contraception for the duration of study therapies and a period of 60 days thereafter. Should a female partner of a trial participant become pregnant or suspect she is pregnant while the subject is participating in this study, the patient should inform his treating physician immediately.
* Ability to understand and the willingness to sign a written informed consent.
Exclusion Criteria:
* Prior androgen deprivation therapy (ADT) \> 3 months OR anti-androgen therapy (AAT) of \> 30 days. For shorter courses of either, at least 30 day "wash out" period is required with confirmation of resolved castration of testosterone to \>50ng/mL.
* Ongoing testosterone replacement therapy (TRT) with refusal to discontinue (must be stopped with demonstration of detectable PSA ≥0.05ng/mL and non-castrate testosterone \>50ng/mL after 14 days of TRT cessation)
* Prior pelvic radiotherapy
* 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.
* History of bladder neck or urethral stricture requiring procedural intervention.
* Patients with a prior or concurrent malignancy whose natural history or treatment has the potential to actively interfere with the safety or efficacy assessments of this study in the investigator's view.
* Active inflammatory bowel disease requiring recurring systemic or steroid/enema therapy RADIATION: Prostate Fossa Radiotherapy, RADIATION: Pelvic nodal Radiotherapy, DRUG: Androgen Deprivation Therapy (ADT)
Prostate Cancer, Prostate
UT Southwestern; Parkland Health & Hospital System
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
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: * Take filgrastim subcutaneously x 5 doses and subsequently undergo a leukapheresis 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 \~6 weeks after the first DOC1021 administration
• One or more lesions available for biopsy or resection to yield at least 50 mg (e.g., 5 core biopsies) 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 filgrastim (e.g. Neupogen), leukapheresis and perinodal injections of DOC1021 near regional nodes + weekly pIFN x 4 weeks.
• 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 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