Search Results
within category "Digestive Systems & Liver Disease"
Here are the studies that match your search criteria. If you are interested in participating, please reach out to the contact listed for the study. If no contact is listed, contact us and we'll help you find the right person.
Phase 1 Dose-escalating Study of MM-398 (Irinotecan Sucrosofate Liposome Injection) Plus Intravenous Cyclophosphamide in Recurrent or Refractory Pediatric Solid Tumors
This is a Phase 1 study of the combination of two drugs: MM-398 and Cyclophosphamide. The
goal is to find the highest dose of MM-398 that can be given safely when it is used together
with the chemotherapy drug Cyclophosphamide.
• Histologically or cytologically-confirmed Ewing sarcoma, rhabdomyosarcoma,
neuroblastoma, or osteosarcoma
• Disease progression after prior therapy in locally advanced or metastatic setting
• Measurable or evaluable disease based on the Response Evaluation Criteria in Solid
Tumors (RECIST v1.1) criteria
• Age 12 months to <21 years
• Adequate bone marrow reserves, hepatic function, and renal function
• Recovered from effects of any prior surgery or cancer therapy
• Patients 18 years or older will provide written consent. A parent or legal guardian of
a patient <18 years of age will provide informed consent and patients 11 to 18 years
of age will provide written assent or as per participating institutional policy.
Exclusion Criteria:
• Clinically significant gastrointestinal disorders
• NYHA Class III or IV congestive heart failure, ventricular arrhythmias or uncontrolled
blood pressure
• Active infection or unexplained fever
• Known hypersensitivity to any of the components of MM-398 or other liposomal products
• Recent Investigational therapy
• Pregnant or breast feeding; females of child-bearing potential must test negative for
pregnancy at the time of enrollment
Drug: MM-398 (Irinotecan Sucrosofate Liposome Injection) plus cyclophosphamide
Neuroblastoma, Sarcoma, Ewing Sarcoma, Mycosis Fungoides, Rhabdomyosarcoma, Osteosarcoma, Recurrent or Refractory Solid Tumors, Brain and Nervous System, Eye and Orbit, Anus, Bones and Joints, Breast - Female, Breast - Male, Cervix, Colon, Corpus Uteri, Ear, Esophagus, Gall Bladder, Head and Neck, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung/Thoracic, Melanoma, skin, Nose, Other Digestive Organ, Other Endocrine System, Other Female Genital, Other Male Genital, Other Respiratory and Intrathoracic Organs, Other Skin, Other Urinary, Ovary, Pancreas, Prostate, Rectum, Stomach, Throat, Thyroid, Urinary Bladder, Uterine (Endometrial), Vulva, Hodgkins Lymphoma, Kaposis sarcoma, Other Hematopoietic, Small Intestine, Soft Tissue
TransMedics (OCS) Liver Trial: Preserving and Assessing Donor Livers for Transplantation (Liver PROTECT)
A prospective, phased-pivotal, international randomized trial to evaluate the effectiveness
of the OCS™ Liver to preserve and assess donor livers intended for transplantation.
Call 214-648-5005 studyfinder@utsouthwestern.edu
David Wojciechowski
188709
All
18 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT02522871
STU 092015-076
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:
• Registered male or female primary Liver transplant candidate
• Age ≥18 years old
• Signed: 1) written informed consent document and 2) authorization to use and disclose
protected health information
Exclusion Criteria:
• Acute, fulminant liver failure
• Prior solid organ or bone marrow transplant
• Chronic use of hemodialysis or diagnosis of chronic renal failure, defined as chronic
serum creatinine of >3 mg/dl for >2 weeks and/or requiring hemodialysis
• Multi-organ transplant
• Ventilator dependent
• Dependent on > 1 IV inotrope to maintain hemodynamics
Device: OCS™ Liver System, Other: Control
Liver Transplantation, Liver Preservation for Transplant
A Study of Pembrolizumab and Bavituximab in Patients With Advanced Hepatocellular Carcinoma
This is a non-randomized, open-label, multi-site phase II therapeutic trial of pembrolizumab
and bavituximab in patients with locally advanced HCC. Locally advanced or metastatic HCC is
defined as disease that is not amenable to surgical and/or locoregional therapies. Subjects
must not have received prior systemic therapy for advanced HCC in keeping with the first-line
setting of this study.
• Patient must have a histologically confirmed diagnosis hepatocellular carcinoma; known
fibrolamellar HCC, sarcomatoid HCC or mixed cholangiocarcinoma and HCC will be
excluded
• Locally advanced or metastatic disease
• 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.
• Measurable disease, as defined as lesions that can accurately be measured in at least
one dimension according to RECIST version 1.1 at least 1 cm with contrast enhanced
dynamic imaging (magnetic resonance imaging or computed tomography).
• Child-Pugh Score A
• Age ≥ 18 years
• ECOG Performance score of 0-1
• Life expectancy greater than 6 months
• Following baseline laboratory values:
1. Total bilirubin ≤ 2.0 mg/ml
2. INR ≤ 1.7
3. Hgb ≥ 8.5 g/dl
4. AST, ALT ≤5 times ULN
5. Platelet count ≥ 50,000/mm3
6. Serum creatinine ≤ 1.5 mg/dL or creatinine clearance ≥ 50 mL/min
7. Albumin ≥ 2.5 g/dl
8. Absolute neutrophil ≥ 1,500 cells/mm3
• Male and female subjects of child bearing potential must agree to use an adequate
method of contraception for the course of the study through 120 days after the last
dose of study medication
• Women of childbearing potential must have a negative pregnancy test within 72 hours
prior to receiving the first dose of study medication
• Subjects are eligible to enroll if they have non-viral-HCC, or if they have HBV-HCC,
or HCV-HCC defined as follows:
HBV-HCC: Controlled (treated) hepatitis B subjects will be allowed if they meet the
following criteria:
Antiviral therapy for HBV must be given for at least 12 weeks and HBV viral load must be
less than 100 IU/mL prior to first dose of study drug. Subjects on active HBV therapy with
viral loads under 100 IU/ml should stay on the same therapy throughout study treatment.
Subjects who are anti-HBc (+), negative for HBsAg, negative for anti-HBs, and have an HBV
viral load under 100 IU/mL do not require HBV anti-viral prophylaxis.
HCV-HCC: active or resolved HCV infection as evidenced by detectable HCV RNA or antibody.
Patients who have failed HCV therapy as evidenced by detectable HCV RNA will be eligible.
Subjects with chronic infection by HCV who are treated (successfully or treatment failure)
or untreated are allowed on study. In addition, subjects with successful HCV treatment are
allowed as long as there are ≥4 weeks between completion of HCV therapy and start of study
drug.
Successful HCV treatment definition: SVR12.
•Prior therapy is allowed provided the following are met: at least 4 weeks since prior
locoregional therapy including surgical resection, chemoembolization, radiotherapy, or
ablation. Provided target lesion has increased in size by 25% or more or the target lesion
was not treated with locoregional therapy. Patients treated with palliative radiotherapy
for symptoms will be eligible 1 week after treatment as long as the target lesion is not
the treated lesion.
Exclusion Criteria:
• Prior liver transplant;
• Patient who has received previous systemic therapy for HCC;
• Clinically significant, uncontrolled heart disease and/or recent events including any
of the following:
• History of acute coronary syndromes (including myocardial infarction, unstable angina,
coronary artery bypass grafting, coronary angioplasty, or stenting) or symptomatic
pericarditis within 12 months prior to screening;
• History of documented congestive heart failure (New York Heart Association functional
classification III-IV);
• Documented cardiomyopathy;
• Patient has a left ventricular ejection fraction <40% as determined by MUGA scan or
ECHO (MUGA and ECHO are not required prior to enrollment);
• Known human immunodeficiency virus (HIV) positive (testing not required);
• History of thromboembolic events (including both pulmonary embolism and deep venous
thrombus but not including tumor thrombus) within the last 6 months;
• Hypersensitivity to IV contrast; not suitable for pre-medication;
• Active or fungal infections requiring systemic treatment within 7 days prior to
screening;
• Known history of, or any evidence of, interstitial lung disease or active
non-infectious pneumonitis;
• Evidence of poorly controlled hypertension which is defined as systolic blood pressure
>150 mmHg or diastolic pressure >90 mmHg despite optimal medical management;
• Pre-existing thyroid abnormality with thyroid function that cannot be maintained in
the normal range with medication;
• Active, known, or suspected autoimmune disease with the following exceptions i)
Subjects with vitiligo, type I diabetes mellitus, resolved childhood asthma or atopy
are permitted to enroll; ii) Subjects with suspected autoimmune thyroid disorders may
be enrolled if they are currently euthyroid or with residual hypothyroidism requiring
only hormone replacement.
iii) Subjects with psoriasis requiring systemic therapy must be excluded from enrollment
• Patient has any other concurrent severe and/or uncontrolled medical condition that
would, in the investigator's judgment, cause unacceptable safety risks, contraindicate
patient participation in the study or compromise compliance with the protocol (e.g.
chronic pancreatitis, active untreated or uncontrolled fungal, bacterial, or viral
infections, etc.);
• Patient has any other concurrent severe and/or uncontrolled medical condition that
would, in the investigator's judgment, cause unacceptable safety risks, contraindicate
patient participation in the study or compromise compliance with the protocol (e.g.
chronic pancreatitis, active untreated or uncontrolled fungal, bacterial, or viral
infections, etc.);
• Known history of active bacillus tuberculosis;
• Subjects with a condition requiring systemic treatment with either corticosteroids (>
10 mg/day prednisone equivalent) or other immunosuppressive medications within 14 days
of study administration. Inhaled or topical steroids and adrenal replacement doses >10
mg/day prednisone equivalents are permitted in the absence of autoimmune disease;
• Patient who has received radiotherapy ≤ 4 weeks prior to study entry. Palliative
radiotherapy for symptomatic control is acceptable (if completed at least 2 weeks
prior to study drug administration and no additional radiotherapy for the same lesion
is planned);
• Patient has had major surgery within 14 days prior to starting study drug or has not
recovered from major side effects (tumor biopsy is not considered as major surgery);
• Clinically apparent ascites on physical examination, ascites present on imaging
studies is allowed;
• Patient has a known hypersensitivity to any of the excipients of bavituximab or
pembrolizumab or monoclonal antibody;
• Active gastrointestinal bleeding within previous 2 months;
• History of any condition requiring anti-platelet therapy (aspirin >300 mg/day,
clopidogrel >75 mg/day);
• Prisoners or subjects who are involuntarily incarcerated;
• Symptomatic or clinically active brain metastases;
• Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a
female after contraception and until the termination of gestation, confirmed by a
positive hCG laboratory test;
• Prior immunotherapy including anti-PD-1, anti-PD-L1, or anti-PD-L2 agents;
• Has dual active HBV infection (HBsAg (+) and /or detectable HBV DNA) and HCV infection
(anti-HCV Ab(+) and detectable HCV RNA) at study entry.
Drug: Pembrolizumab, Drug: Bavituximab
Hepatocellular Carcinoma, Liver
UT Southwestern; Parkland Health & Hospital System
Basket Study of Neratinib in Participants With Solid Tumors Harboring Somatic HER2 or EGFR Exon 18 Mutations (SUMMIT)
This is an open-label, multicenter, multinational, Phase 2 basket study exploring the
efficacy and safety of neratinib as monotherapy or in combination with other therapies in
participants with HER (EGFR, HER2) mutation-positive solid tumors.
• Provide written informed consent
• Histologically confirmed cancers for which no curative therapy exists
• Documented HER2 or EGFR exon 18 mutation
• Participants must agree and commit to use appropriate methods of contraception as
outlined in the protocol
• At least one measurable lesion, defined by RECIST v1.1
Exclusion Criteria:
• Participants harboring ineligible somatic HER2 mutations
• Prior treatment with any HER2-directed tyrosine kinase inhibitor (e.g., lapatinib,
afatinib, dacomitinib, neratinib) is excluded with the following exception: patients
with EGFR exon 18 mutated NSCLC who may have received afatinib, osimertinib, or other
pan HER or EGFR TKIs remain eligible
• Participants who are receiving any other anticancer agents
• Symptomatic or unstable brain metastases
• Women who are pregnant or breast-feeding
There are additional inclusion and exclusion criteria. The study center will determine if
criteria for participation are met.
A Study of Selpercatinib (LOXO-292) in Participants With Advanced Solid Tumors, RET Fusion-Positive Solid Tumors, and Medullary Thyroid Cancer (LIBRETTO-001) (LIBRETTO-001)
This is an open-label, first-in-human study designed to evaluate the safety, tolerability,
pharmacokinetics (PK) and preliminary anti-tumor activity of selpercatinib (also known as
LOXO-292) administered orally to participants with advanced solid tumors, including
rearranged during transfection (RET)-fusion-positive solid tumors, medullary thyroid cancer
(MTC) and other tumors with RET activation.
For Phase 1:
• Participants with a locally advanced or metastatic solid tumor that:
• Has progressed on or is intolerant to standard therapy, or
• For which no standard therapy exists, or in the opinion of the Investigator, are not
candidates for or would be unlikely to tolerate or derive significant clinical benefit
from standard therapy, or
• Decline standard therapy
• Prior multikinase inhibitors (MKIs) with anti-RET activity are allowed
• A RET gene alteration is not required initially. Once adequate PK exposure is
achieved, evidence of RET gene alteration in tumor and/or blood is required as
identified through molecular assays, as performed for clinical evaluation
• Measurable or non-measurable disease as determined by RECIST 1.1 or RANO as
appropriate to tumor type
• Eastern Cooperative Oncology Group (ECOG) score of 0, 1, or 2 or Lansky Performance
Score (LPS) greater than or equal to (≥) 40 percent (%) (age less than [<] 16 years)
with no sudden deterioration 2 weeks prior to the first dose of study treatment
• Adequate hematologic, hepatic and renal function
• Life expectancy of at least 3 months
For Phase 2: As for phase 1 with the following modifications:
• For Cohort 1: Participants must have received prior standard therapy appropriate for
their tumor type and stage of disease, or in the opinion of the Investigator, would be
unlikely to tolerate or derive clinical benefit from appropriate standard of care
therapy
• Cohorts 1 and 2:
• Enrollment will be restricted to participants with evidence of a RET gene
alteration in tumor
• At least one measurable lesion as defined by RECIST 1.1 or RANO, as appropriate
to tumor type and not previously irradiated
• Cohorts 3 and 4: Enrollment closed
• Cohort 5:
• Cohorts 1-4 without measurable disease
• MCT not meeting the requirements for Cohorts 3 or 4
• MTC syndrome spectrum cancers (e.g., MTC, pheochromocytoma), cancers with
neuroendocrine features/differentiation, or poorly differentiated thyroid cancers
with other RET alteration/activation may be allowed with prior Sponsor approval
• cfDNA positive for a RET gene alteration not known to be present in a tumor
sample
• Cohort 6: Participants who otherwise are eligible for Cohorts 1, 2 or 5 who
discontinued another RET inhibitor may be eligible with prior Sponsor approval
• Cohort 7: Participants with a histologically confirmed stage IB-IIIA NSCLC and a RET
fusion; determined to be medically operable and tumor deemed resectable by a thoracic
surgical oncologist, without prior systemic treatment for NSCLC
Key Exclusion Criteria (Phase 1 and Phase 2):
• Phase 2 Cohorts 1 and 2: an additional known oncogenic driver
• Cohorts 3 and 4: Enrollment closed
• Cohorts 1, 2 and 5: prior treatment with a selective RET inhibitor Notes: Participants
otherwise eligible for Cohorts 1, 2, and 5 who discontinued another selective RET
inhibitor may be eligible for Phase 2 Cohort 6 with prior Sponsor approval
• Investigational agent or anticancer therapy (including chemotherapy, biologic therapy,
immunotherapy, anticancer Chinese medicine or other anticancer herbal remedy) within 5
half-lives or 2 weeks (whichever is shorter) prior to planned start of LOXO-292
(selpercatinib). In addition, no concurrent investigational anti-cancer therapy is
permitted Note: Potential exception for this exclusion criterion will require a valid
scientific justification and approval from the Sponsor
• Major surgery (excluding placement of vascular access) within 2 weeks prior to planned
start of LOXO-292 (selpercatinib)
• Radiotherapy with a limited field of radiation for palliation within 1 week of planned
start of LOXO-292 (selpercatinib), with the exception of participants receiving
radiation to more than 30% of the bone marrow or with a wide field of radiation, which
must be completed at least 4 weeks prior to the first dose of study treatment
• Any unresolved toxicities from prior therapy greater than Common Terminology Criteria
for Adverse Events (CTCAE) Grade 1 at the time of starting study treatment with the
exception of alopecia and Grade 2, prior platinum-therapy related neuropathy
• Symptomatic primary CNS tumor, metastases, leptomeningeal carcinomatosis, or untreated
spinal cord compression. Participants are eligible if neurological symptoms and CNS
imaging are stable and steroid dose is stable for 14 days prior to the first dose of
LOXO-292 (selpercatinib) and no CNS surgery or radiation has been performed for 28
days, 14 days if stereotactic radiosurgery (SRS)
• Clinically significant active cardiovascular disease or history of myocardial
infarction within 6 months prior to planned start of LOXO-292 (selpercatinib) or
prolongation of the QT interval corrected (QTcF) greater than (>) 470 milliseconds
(msec)
• Participants with implanted pacemakers may enter the study without meeting QTc
criteria due to nonevaluable measurement if it is possible to monitor for QT
changes.
• Participants with bundle branch block may be considered for study entry if QTc is
appropriate by a formula other than Fridericia's and if it is possible to monitor
for QT changes.
• Required treatment with certain strong cytochrome P450 3A4 (CYP3A4) inhibitors or
inducers and certain prohibited concomitant medications
• Phase 2 Cohort 7 (neoadjuvant treatment): Participant must not have received prior
systemic therapy for NSCLC.
Drug: LOXO-292
Lymphoma, Non-Small Cell Lung Cancer, Colon Cancer, Medullary Thyroid Cancer, Any Solid Tumor, Brain and Nervous System, Eye and Orbit, Anus, Bones and Joints, Breast - Female, Breast - Male, Cervix, Colon, Corpus Uteri, Esophagus, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung/Thoracic, Melanoma, skin, Other Digestive Organ, Other Endocrine System, Other Female Genital, Other Male Genital, Other Respiratory and Intrathoracic Organs, Other Skin, Other Urinary, Ovary, Pancreas, Prostate, Stomach, Thyroid, Urinary Bladder, Hodgkins Lymphoma, Small Intestine, Soft Tissue
LOXO-292, KIF5B-RET, M918T, CCDC6-RET, RET-PTC1, NCOA4-RET, RET-PTC, RET-PTC3, RET-PTC4, PRKAR1A-RET, RET-PTC2, GOLGA5-RET, RET-PTC5, ERC1-RET, KTN1-RET, RET-PTC8, HOOK3-RET, PCM1-RET, TRIM24-RET, RET-PTC6, TRIM27-RET, TRIM33-RET, RET-PTC7, AKAP13-RET, FKBP15-RET, SPECC1L-RET, TBL1XR1-RET, BCR-RET, FGRF1OP-RET, RFG8-RET, RET-PTC9, ACBD5-RET, MYH13-RET, CUX1-RET, KIAA1468-RET, FRMD4A-RET, SQSTM1-RET, AFAP1L2-RET, PPFIBP2-RET, EML4-RET, PARD3-RET, G533C, C609F, C609G, C609R, C609S, C609Y, C611F, C611G, C611S, C611Y, C611W, C618F, C618R, C618S, C620F, C620R, C620S, C630R, C630Y, D631Y, C634F, C634G, C634R, C634S, C634W, C634Y, K666E, E768D, L790F, V804L, V804M, A883F, S891A, R912P, CLIP1-RET, Y806C, RET fusion, RET alteration, RET mutation, RET rearrangement, RET translocation, Neoplasms by Site, Neoplasms, Non-Small Cell Lung Cancer, Lung Neoplasms, Carcinoma, Non-Small-Cell Lung, Cancer of Lung, Cancer of the Lung, Lung Cancer, Neoplasms, Lung, Neoplasms, Pulmonary, Pulmonary Cancer, Pulmonary Neoplasms, Respiratory Tract Neoplasms, Lung Diseases, Respiratory Tract Diseases, Carcinoma, Bronchogenic, Bronchial Neoplasms, Medullary Thyroid Cancer, Papillary Thyroid Cancer, Thyroid Diseases, Thyroid Neoplasms, Cancer of the Thyroid, Cancer of Thyroid, Neoplasms, Thyroid, Thyroid Ademona, Thyroid Cancer, Thyroid Carcinoma, Endocrine System Diseases, Endocrine Gland Neoplasms, Head and Neck Neoplasms, Thoracic Neoplasms, CNS tumor, Primary CNS tumor, Cancer of Colon, Cancer of the Colon, Colon Cancer, Colon Neoplasms, Colonic Cancer, Neoplasms, Colonic, Malignant tumor of Breast, Mammary Cancer, Mammary Carcinoma, Human, Mammary Neoplasm, Human, Neoplasms, Breast, Tumors, Breast, Human Mammary Carcinoma, Malignant Neoplasm of Breast, Breast Carcinoma, Breast Tumors, Cancer of the Breast, Breast Neoplasms, Breast Cancer, RET Inhibitor, MTC, NSCLC, selpercatinib, neo-adjuvant treatment in early stage NSCLC
MEDI9447(Oleclumab) Pancreatic Chemotherapy Combination Study
The objective of this study is to evaluate the safety, tolerability, and antitumor activity
of oleclumab (MEDI9447) in combination with or without durvalumab plus chemotherapy in
subjects with metastatic pancreatic cancer.
1. Age ≥ 18
2. Written and signed informed consent must be obtained
3. ECOG Performance Status 0 or 1
4. Weight ≥ 35 kg
5. Subjects must have histologically or cytologically, confirmed pancreatic
adenocarcinoma:
Cohort A: Subjects with previously untreated metastatic pancreatic adenocarcinoma (1st
line metastatic disease) not previously treated with systemic therapies.
Cohort B: Subjects with metastatic pancreatic adenocarcinoma previously treated with
gemcitabine-based chemotherapy (without exposure to 5-FU, capecitabine, oxaliplatin)
2nd line metastatic disease
6. Subjects must have at least 1 measurable lesion according to RECIST v1.1
7. All subjects must consent to providing archival tumor specimens
Exclusion Criteria:
1. Receipt of any conventional or investigational anticancer therapy within 21 days or
palliative radiotherapy within 14 days prior to the scheduled first dose of study
treatment.
2. Prior receipt of any immune-related therapy
3. Concurrent enrollment in another therapeutic clinical study. Enrollment in
observational studies will be allowed
4. Subjects with a history of venous thrombosis within the past 3 months
5. Subjects with prior history of myocardial infarction, transient ischemic attack, or
stroke in the last 3 months prior to start of treatment
6. Active or prior documented autoimmune or inflammatory disorders within the past 3
years prior to the start of treatment
7. Other invasive malignancy within 2 years.
8. Any history of leptomeningeal disease or cord compression.
9. Current or prior use of immunosuppressive medication within 14 days prior to the first
dose
A Study of Zolbetuximab (IMAB362) Plus CAPOX Compared With Placebo Plus CAPOX as First-line Treatment of Subjects With Claudin (CLDN) 18.2-Positive, HER2-Negative, Locally Advanced Unresectable or Metastatic Gastric or Gastroesophageal Junction (GEJ) Adenocarcinoma (GLOW)
The purpose of this study is to evaluate the efficacy of zolbetuximab plus capecitabine and
oxaliplatin (CAPOX) compared with placebo plus CAPOX (as first-line treatment) as measured by
Progression Free Survival (PFS).
This study will also evaluate efficacy, physical function, safety, and tolerability of
zolbetuximab, as well as its effects on quality of life. Pharmacokinetics (PK) of
zolbetuximab and the immunogenicity profile of zolbetuximab will be evaluated as well.
• A female subject is eligible to participate if she is not pregnant (negative serum
pregnancy test at screening; female subjects with elevated serum beta human chorionic
gonadotropin (βhCG) and a demonstrated non-pregnant status through additional testing
are eligible) and at least 1 of the following conditions applies:
• Not a woman of childbearing potential (WOCBP) OR
• WOCBP who agrees to follow the contraceptive guidance throughout the treatment
period and for 9 months after the final administration of oxaliplatin and 6
months after the final administration of all other study drugs.
• Female subject must agree not to breastfeed starting at screening and throughout the
study period, and for 6 months after the final study treatment administration.
• Female subject must not donate ova starting at screening and throughout the study
period, and for 9 months after the final administration of oxaliplatin and 6 months
after the final administration of all other study drugs.
• A male subject with female partner(s) of childbearing potential:
• must agree to use contraception during the treatment period and for 6 months
after the final study treatment administration.
• A male subject must not donate sperm during the treatment period and for 6 months
after the final study treatment administration.
• Male subject with a pregnant or breastfeeding partner(s) must agree to remain
abstinent or use a condom for the duration of the pregnancy or time partner is
breastfeeding throughout the study period and for 6 months after the final study
treatment administration.
• Subject has histologically confirmed diagnosis of Gastric or GEJ adenocarcinoma.
• Subject has radiologically confirmed locally advanced unresectable or metastatic
disease within 28 days prior to randomization.
• Subject has radiologically evaluable disease (measurable and/or non-measurable disease
according to RECIST 1.1), per local assessment, ≤ 28 days prior to randomization. For
subjects with only 1 evaluable lesion and prior radiotherapy ≤ 3 months before
randomization, the lesion must either be outside the field of prior radiotherapy or
have documented progression following radiation therapy.
• Subject's tumor expresses CLDN18.2 in ≥ 75% of tumor cells demonstrating moderate to
strong membranous staining as determined by central IHC testing.
• Subject has a HER2-negative tumor as determined by local or central testing on a
gastric or GEJ tumor specimen.
• Subject has ECOG performance status 0 or 1.
• Subject has predicted life expectancy ≥ 12 weeks.
• Subject must meet all of the following criteria based on the centrally or locally
analyzed laboratory tests collected within 14 days prior to randomization. In the case
of multiple sample collections within this period, the most recent sample collection
with available results should be used to determine eligibility.
• Hemoglobin (Hb) ≥ 9 g/dl. Subjects requiring transfusions are eligible if they
have a post-transfusion Hgb ≥ 9 g/dL.
• Absolute Neutrophil Count (ANC) ≥ 1.5x10^9/L
• Platelets ≥ 100x10^9/L
• Albumin ≥ 2.5 g/dL
• Total Bilirubin ≤ 1.5 x upper limit of normal (ULN) without liver metastases (or
< 3.0 x ULN if liver metastases are present)
• Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5 x ULN
without liver metastases (or ≤ 5 x ULN if liver metastases are present)
• Estimated creatinine clearance ≥ 30 mL/min
• Prothrombin time/international normalized ratio (PT/INR) and partial
thromboplastin time (PTT) ≤ 1.5 x ULN (except for subjects receiving
anticoagulation therapy)
Exclusion Criteria:
• Subject has received prior systemic chemotherapy for locally advanced unresectable or
metastatic gastric or GEJ adenocarcinoma. However, subject may have received either
neo-adjuvant or adjuvant chemotherapy, immunotherapy or other systemic anticancer
therapies as long as it was completed at least 6 months prior to randomization.
• Subject has received radiotherapy for locally advanced unresectable or metastatic
gastric or GEJ adenocarcinoma ≤ 14 days prior to randomization and has not recovered
from any related toxicity.
• Subject has received treatment with herbal medications or other treatments that have
known antitumor activity within 28 days prior to randomization.
• Subject has received systemic immunosuppressive therapy, including systemic
corticosteroids within 14 days prior to randomization. Subjects using a physiologic
replacement dose of hydrocortisone or its equivalent (defined as up to 30 mg per day
of hydrocortisone or up to 10 mg per day of prednisone), receiving a single dose of
systemic corticosteroids or receiving systemic corticosteroids as premedication for
radiologic imaging contrast use are allowed.
• Subject has received other investigational agents or devices within 28 days prior to
randomization.
• Subject has prior severe allergic reaction or intolerance to known ingredients of
zolbetuximab or other monoclonal antibodies, including humanized or chimeric
antibodies.
• Subject has known immediate or delayed hypersensitivity, intolerance or
contraindication to any component of study treatment.
• Subject has prior severe allergic reaction or intolerance to any component of CAPOX.
• Subject has known dihydropyrimidine dehydrogenase (DPD) deficiency.
• Subject has a complete gastric outlet syndrome or a partial gastric outlet syndrome
with persistent/recurrent vomiting.
• Subject has significant gastric bleeding and/or untreated gastric ulcers that exclude
the subject from participation.
• Subject has a known history of a positive test for human immunodeficiency virus (HIV)
infection or known active hepatitis B (positive hepatitis B surface antigen (HBs Ag))
or C infection. NOTE: Screening for these infections should be conducted per local
requirements.
• For subjects who are negative for HBs Ag, but hepatitis B core antibody (HBc Ab)
positive, an HB deoxyribonucleic acid (DNA) test will be performed and if
positive, the subject will be excluded.
• Subjects with positive hepatitis C virus (HCV) serology, but negative HCV
ribonucleic acid (RNA) test are eligible.
• Subjects treated for HCV with undetectable viral load results are eligible.
• Subject has an active autoimmune disease that has required systemic treatment within
the past 3 months prior to randomization.
• Subject has active infection requiring systemic therapy that has not completely
resolved within 7 days prior to randomization.
• Subject has significant cardiovascular disease, including any of the following:
• Congestive heart failure (defined as New York Heart Association Class III or IV),
myocardial infarction, unstable angina, coronary angioplasty, stenting, coronary
artery bypass graft, cerebrovascular accident (CVA) or hypertensive crisis within
6 months prior to randomization.
• History of clinically significant ventricular arrhythmias (i.e., sustained
ventricular tachycardia, ventricular fibrillation or Torsades de Pointes
• QTc interval > 450 msec for male subjects; QTc interval > 470 msec for female
subjects
• History or family history of congenital long QT syndrome
• Cardiac arrhythmias requiring anti-arrhythmic medications (Subject with rate
controlled atrial fibrillation for > 1 month prior to randomization are
eligible).
• Subject has a history of central nervous system (CNS) metastases and/or carcinomatous
meningitis from gastric/GEJ cancer..
• Subject has known peripheral sensory neuropathy > grade 1 unless the absence of deep
tendon reflexes is the sole neurological abnormality.
• Subject has had a major surgical procedure ≤ 28 days prior to randomization.
• Subject is without complete recovery from a major surgical procedure ≤ 14 days
prior to randomization.
• Subject has psychiatric illness or social situations that would preclude study
compliance.
• Subject has another malignancy for which treatment is required.
• Subject has any concurrent disease, infection, or co-morbid condition that interferes
with the ability of the subject to participate in the study, which places the subject
at undue risk or complicates the interpretation of data.
Safety and Efficacy Study of IMSA101 in Refractory Malignancies
Open-label, dose escalation (Phase I) and dose expansion (Phase IIA) study of patients
receiving intra-tumoral IMSA101 alone or in combination with an immune checkpoint inhibitor
(ICI) (Phase I and II)
1. Signed informed consent and mental capability to understand the informed consent
2. Male or female patients > 18 years of age
3. Histologically or cytologically documented locally advanced or metastatic solid tumor
malignancies refractory to or otherwise ineligible for treatment with standard-of-care
agents/regimens, including but not limited to:
• Malignant melanoma
• Hormone receptor negative breast cancer
• Gastro-esophageal cancer
• Non-small cell lung cancer
• Head and neck cancer
• Hepatoma
• Renal cell carcinoma
4. Eastern Cooperative Oncology Group (ECOG) performance status of ≤ 1
5. Evaluable or measurable disease as follows:
• A minimum of 3 RECIST-evaluable lesions: one that is suitable for injection and
biopsied; one non-injected that will be biopsied for abscopal effect; and one
measurable lesion that will be followed for response only.
• Injectable tumors shall be accessed by intralesional (cutaneous) or percutaneous
injection only, including those lesions that are visible, palpable, or detectable
by standard radiographic or ultrasound methods. Neither surgical procedures nor
endoscopically-guided injections including those to endobronchial, endoluminal,
or endosinusial spaces shall be allowed. While no anatomic locations are required
or disallowed, lesions selected for intratumoral injection must, in the opinion
of the investigator:
• Not be immediately adjacent to blood vasculature or other physiologic landmarks
in such a way that will accrue undue safety risk to the patient
• Have longest diameter ≥ 10 mm and ≤ 50 mm
• Be fully efficacy evaluable per RECIST v1.1 criteria
6. Life expectancy > 3 months (Phase I) and > 6 months (Phase IIA)
7. ECG without evidence of clinically meaningful conduction abnormalities or active
ischemia as determined by the investigator
8. Acceptable organ and marrow function as defined below:
• Absolute neutrophil count > 1,500 cells/μL
• Platelets > 50,000 cells/μL
• Total bilirubin ≤ 1.5 times the upper limit of normal (ULN)
• Aspartate aminotransferase (AST)/alanine transaminase (ALT) ≤ 2.5 times ULN. If
liver metastases are present, AST/ALT < 5 times ULN
• Serum creatinine < 1.5 mg/dL and a measured creatinine clearance ≥ 50 mL/min
using the Cockcroft-Gault formula
• Prothrombin time (PT)/partial thromboplastin time (PTT) ≤ 1.5 times ULN
9. Women of child-bearing potential (defined as a female who has experienced menarche and
who has not undergone successful surgical sterilization (hysterectomy, bilateral
salpingectomy, or bilateral oophorectomy) or is not postmenopausal (defined as
amenorrhea for at least 12 consecutive months with an appropriate clinical profile at
the appropriate age, e.g., greater than 45 years) must have a negative serum pregnancy
test prior to first dose of study drug
10. Male and female patients with reproductive potential must agree to use two forms of
highly effective contraception throughout the study
11. Phase I combination only: Demonstrated RECIST stable disease through ≥ 4 consecutive
cycles of an approved PD-1 or PD-L1 targeted ICI with no Grade ≥ 3 CTCAE events
considered by the investigator to be drug-related.
Exclusion Criteria:
1. Anti-cancer therapy within 4 weeks or < 5 half-lives of the first dose of study drug.
2. Failure to recover to Grade 1 or less from clinically significant AEs due to prior
anti-cancer therapy.
3. Known untreated brain metastases or treated brain metastases that have not been stable
(scan showing no worsening of central nervous system (CNS) lesion[s] and no
requirement of corticosteroids) ≥ 4 weeks prior to study enrollment
4. Baseline prolongation of QT/QTc interval (QTc interval > 470)
5. 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 opinion of the
investigator would limit compliance with study requirements
6. Women who are pregnant or breastfeeding
7. Phase I combination only: Prior tumor progression through PD-1 or PD-L1 targeted ICI
therapy.
A Study of Atezolizumab Plus Bevacizumab Versus Active Surveillance as Adjuvant Therapy in Patients With Hepatocellular Carcinoma at High Risk of Recurrence After Surgical Resection or Ablation (IMbrave050)
This study will evaluate the efficacy and safety of adjuvant therapy with atezolizumab plus
bevacizumab compared with active surveillance in participants with completely resected or
ablated hepatocellular carcinoma (HCC) who are at high risk for disease recurrence.
• Participants with a first diagnosis of HCC who have undergone either a curative
resection or ablation (radiofrequency ablation [RFA] or microwave ablation [MVA] only)
within 4-12 weeks prior to randomization
• Documented diagnosis of HCC that has been completely resected or ablated (RFA or MVA
only)
• Absence of major macrovascular invasion (except Vp1/Vp2) and extrahepatic spread
• Absence of extrahepatic spread as confirmed by CT or MRI scan of the chest, abdomen,
pelvis, and head prior to and following curative procedure
• Full recovery from surgical resection or ablation within 4 weeks prior to
randomization
• High risk for HCC recurrence after resection or ablation
• For patients who received post-operative transarterial chemoembolization: full
recovery from the procedure within 4 weeks prior to randomization
• For patients with resected HCC, availability of a representative baseline tumor tissue
sample
• ECOG Performance Status of 0 or 1
• Child-Pugh Class A status
• Adequate hematologic and end-organ function
• For women of childbearing potential: agreement to remain abstinent (refrain from
heterosexual intercourse) or use contraceptive methods
• For men: agreement to remain abstinent (refrain from heterosexual intercourse) or use
a condom, and agreement to refrain from donating sperm
Exclusion Criteria:
• Known fibrolamellar HCC, sarcomatoid HCC, or mixed cholangiocarcinoma and HCC
• Evidence of residual, recurrent, or metastatic disease at randomization
• Clinically significant ascites
• History of hepatic encephalopathy
• Prior bleeding event due to untreated or incompletely treated esophageal and/or
gastric varices within 6 months prior to randomization
• Have received more than 1 cycle of adjuvant TACE following surgical resection
• Active or history of autoimmune disease or immune deficiency
• History of idiopathic pulmonary fibrosis, organizing pneumonia, drug-induced
pneumonitis, or idiopathic pneumonitis, or evidence of active pneumonitis on screening
chest CT scan
• Significant cardiovascular disease within 3 months prior to Day 1 of Cycle 1, unstable
arrhythmia, or unstable angina
• History of malignancy other than HCC within 5 years prior to screening, with the
exception of malignancies with a negligible risk of metastasis or death
• Active tuberculosis
• Any other disease, metabolic dysfunction, physical examination finding, or clinical
laboratory finding that contraindicates the use of an investigational drug, may affect
the interpretation of the results, or may render the patient at high risk from
treatment complications
• Pregnant or breastfeeding, or intending to become pregnant during the study or within
5 months after the final dose of atezolizumab or within 6 months after the final dose
of bevacizumab. Women of childbearing potential must have a negative serum pregnancy
test result within 14 days prior to Day 1 of Cycle 1.
• Co-infection with HBV and HCV
• Co-infection with HBV and hepatitis D viral infection
• Clinical significant uncontrolled or symptomatic hypercalcemia
• Any treatment for HCC prior to resection or ablation, including systemic therapy and
locoregional therapy such as TACE
• Treatment with systemic immunostimulatory or immunosuppressive agents
• Inadequately controlled arterial hypertension
• History of hypertensive crisis or hypertensive encephalopathy
• Significant vascular disease
• Evidence of bleeding diathesis or significant coagulopathy
• Current or recent use of aspirin or full-dose oral or parenteral anticoagulants
• Core biopsy within 3 days of Day 1 of Cycle 1
• History of GI fistula, GI perforation, or intra-abdominal abscess
• Serious non-healing or dehiscing wound
• Major surgical procedure within four weeks
• Chronic daily treatment with a non-steroidal anti-inflammatory drug
Drug: Atezolizumab, Drug: Bevacizumab
Carcinoma, Hepatocellular, Liver
UT Southwestern; Parkland Health & Hospital System
A Phase 1/2, Study Evaluating the Safety, Tolerability, PK, and Efficacy of Sotorasib (AMG 510) in Subjects With Solid Tumors With a Specific KRAS Mutation (CodeBreaK 100)
Evaluate the safety and tolerability of sotorasib in adult subjects with KRAS p.G12C mutant
advanced solid tumors.
Estimate the maximum tolerated dose (MTD) and/or a recommended phase 2 dose (RP2D) in adult
subjects with KRAS p.G12C mutant advanced solid tumors.
• Men or women greater than or equal to 18 years old.
• Pathologically documented, locally-advanced or metastatic malignancy with, KRAS p.G12C
mutation identified through molecular testing.
Exclusion Criteria
• Active brain metastases from non-brain tumors.
• Myocardial infarction within 6 months of study day 1.
• Gastrointestinal (GI) tract disease causing the inability to take oral medication.
Drug: sotorasib, Drug: Anti PD-1/L1, Drug: Midazolam
INO-3107 With Electroporation (EP) in Participants With HPV-6- and/or HPV-11-Associated Recurrent Respiratory Papillomatosis (RRP)
This is a Phase 1/2 open-label, multicenter trial to evaluate the safety, tolerability,
immunogenicity, and efficacy of INO-3107 in subjects with HPV-6 and/or HPV-11-associated
recurrent respiratory papillomatosis (RRP). The trial population will include participants
who have been diagnosed with either Juvenile-Onset RRP (J-O RRP) as defined by age at first
diagnosis <12 years or with Adult- Onset RRP (A-O RRP) as defined by age at first diagnosis
≥12 years. A safety run-in will be performed with up to six participants with a one week
waiting period between each enrolled participant.
Call 214-648-5005 studyfinder@utsouthwestern.edu
I-Fan Mau
99252
All
18 Years and over
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT04398433
STU-2020-0247
Show full eligibility criteria
Hide eligibility criteria
Key
Inclusion Criteria:
• Histologically-documented HPV-6- or HPV-11-positive respiratory papilloma or
documentation of low-risk positive HPV using a Sponsor approved HPV-6/11 type-specific
assay
• Requirement for frequent RRP intervention to remove or resect respiratory papilloma,
as defined as at least two RRP surgical (including laser) interventions in the year
prior to and including Day 0
• Must be an appropriate candidate for upcoming surgical intervention as per
Investigator judgment and RRP Staging Assessment score
• Adequate bone marrow, hepatic, and renal function
• Participants must meet one of the below requirements:
• Be of non-child bearing potential (≥12 months of non-therapy-induced amenorrhea,
confirmed by follicle-stimulating hormone [FSH], if not on hormone replacement)
• Be surgically sterile (vasectomy in males or absence of ovaries and/or uterus in
females)
• Agree to use one highly effective or combined contraceptive methods that result
in a failure rate of <1% per year during the treatment period and at least
through week 12 after last dose
• Agree to abstinence from penile-vaginal intercourse, when this is the
participant's preferred and usual lifestyle
Key
Exclusion Criteria:
• Recipient of therapy directed towards RRP disease (other than surgery or ablation)
including but not limited to anti-virals (including cidofovir), radiation,
chemotherapy, anti-angiogenic therapy (including bevacizumab), prophylactic HPV
vaccination (including Gardasil) as therapeutic intervention, or therapy with an
experimental agent within 3 months prior to Day 0
• Ongoing or recent (within 1 year) evidence of autoimmune disease that required
treatment with systemic immunosuppressive treatments, with the exception of: vitiligo,
childhood asthma that has resolved, type 1 diabetes, residual hypothyroidism that
requires only hormone replacement, or psoriasis that does not require systemic
treatment
• Diagnosis of immunodeficiency or treatment with systemic immunosuppressive therapy
within 28 days prior to the first dose of trial treatment, including systemic
corticosteroids
• High risk of bleeding or require the use of anticoagulants for management of a known
bleeding diathesis
• Recipient of any live virus vaccine within 4 weeks prior to the first dose of trial
treatment or any non-live vaccine within two weeks prior to the first dose of trial
treatment
• History of clinically significant, medically unstable disease which, in the judgment
of the Investigator, would jeopardize the safety of the participant, interfere with
trial assessment or evaluation, or otherwise impact the validity of the trial results
• Fewer than two acceptable sites are available for IM injection considering the deltoid
and anterolateral quadriceps muscles. Study treatment should not be given within 2
centimeters (cm) of a tattoo, keloid or hypertrophic scar. If there is implanted
metal, implanted device, within the same limb the use of the deltoid muscle on the
same side of the body is excluded
• Prisoners or participants who are compulsory detained (involuntary incarceration) for
treatment of either a psychiatric or physical (i.e. infectious disease) illness
• Any medical or psychological or non-medical condition that might interfere with the
participation or safety of the participant, as determined by the investigator
The rationale of this clinical trial is to assess the feasibility of selective non-operative
management for locally advanced rectal cancer using dose-escalated ultra-fractionated short
course radiation therapy interdigitated with chemotherapy. We believe delivering short course
radiotherapy over a prolonged interval, at escalated doses and with concurrent chemotherapy
may be feasible and allow for improved clinical response.
1. At least 18 years of age. Both men and women and members of all races and ethnic
groups will be included.
2. Willing and able to provide written informed consent
3. Pathologic diagnosis of rectal adenocarcinoma
4. T3-4 and/or N+ disease per AJCC 8th edition
5. No prior treatment for rectal adenocarcinoma
6. Eastern Cooperative Group (ECOG) performance status of 0-2.
7. Laboratory values supporting acceptable organ and marrow function within 30 days of
eligibility confirmation. Defined as follows:
• WBC ≥ 3,000/mL;
• ANC WBC ≥ 1,000/mL;
• PLT ≥ 75,000/mL;
• T Bili ≤ 1.5 x upper limit of normal (ULN);
• AST/ALT ≤ 2.5 x ULN;
• Creatinine not above ULN, or creatinine clearance >50 mL/min/1.73 m2 for
participants with creatinine levels above institutional normal.
8. All men, as well as women of child-bearing potential must agree to use adequate
contraception (hormonal or barrier method of birth control; abstinence) starting with
the first dose of study therapy through 90 days after the last dose of study drugs.
Should a woman become pregnant or suspect she is pregnant while participating in this
study, she should inform her treating physician immediately.
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).
Exclusion Criteria:
1. Distant nodal disease (retroperitoneal nodes) including inguinal nodes, or any
metastatic disease by CT.
2. Prior RT to the pelvis.
3. Uncontrolled comorbid illness or condition including congestive heart failure,
unstable angina, cardiac arrhythmia, or psychiatric illness that would limit
compliance with the study requirements.
4. Psychiatric illness/social situations that would limit consenting and compliance with
study requirements.
5. Participants who are pregnant or nursing due to the potential for congenital
abnormalities and the potential of this regimen to harm nursing infants
Radiation: Ultrafractionated radiotherapy for rectal cancer
Rectal Cancer, Rectum
Rectal Cancer,T3-4 or N+
UT Southwestern; Parkland Health & Hospital System
Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD)
Double-blind, randomized, placebo-controlled, Phase 3 study to investigate the efficacy and
safety of odevixibat compared to placebo in children with biliary atresia who have undergone
a Kasai hepatoportoenterostomy.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Amal Aqul
103693
All
up to 111 Days old
Phase 3
This study is NOT accepting healthy volunteers
NCT04336722
STU-2020-0239
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:
• A male or female patient with a clinical diagnosis of BA
• Age at Kasai HPE ≤90 days
• Eligible to start study treatment within 3 weeks post-Kasai HPE
Key
Exclusion Criteria:
• Patients with intractable ascites
• Ileal resection surgery
• ALT ≥10× upper limit of normal (ULN) at screening
• Patients reliant only on total parenteral nutrition, or not able to take study
medication orally, at randomization
• Acute ascending cholangitis (patients may be randomized after resolution of acute
ascending cholangitis)
• Choledochal cystic disease
• INR >1.6 (the patient may be treated with Vitamin K intravenously; sample may be
redrawn and if INR is ≤1.6 at resampling the patient may be randomized)
• Any other conditions or abnormalities, including congenital abnormalities, major
cardiac surgery, hepatic, biliary, or GI disease which, in the opinion of the
Investigator or Medical Monitor, may compromise the safety of the patient, the
integrity of study results, or patient compliance with study requirements
• Weight <3.5kg at randomization
Trilaciclib, a CDK 4/6 Inhibitor, in Patients Receiving FOLFOXIRI/Bevacizumab for Metastatic Colorectal Cancer (mCRC): (PRESERVE1)
This is a randomized, double-blind, placebo-controlled, global, multicenter, Phase 3 trial
evaluating the impact of trilaciclib on myelopreservation and anti-tumor efficacy when
administered prior to FOLFOXIRI/bevacizumab in patients with pMMR/MSS mCRC who have not
received systemic therapy for metastatic disease.
1. Age ≥ 18 years of age at the time of signing the informed consent. Patients > 70 years
of age must have a G8 Health State Screening Tool (geriatric screening tool) score >.
2. Proficient mismatch repair/microsatellite stable (pMMR/MSS), histologically or
cytologically-confirmed adenocarcinoma of the colon or rectum. Patients with any BRAF
or KRAS mutation status are eligible.
3. Unresectable and measurable or evaluable disease per RECIST v1.1
4. ECOG performance status of 0 to 1
5. A formalin-fixed paraffin-embedded (FFPE) tumor specimen (from archival or fresh
biopsy) with an associated pathology report documenting pMMR/MSS mCRC must be
confirmed to be available to send to the Sponsor for planned retrospective biomarker
analyses.
6. Adequate organ function
Selected
Exclusion Criteria:
1. Prior systemic therapy for mCRC. Patients who received adjuvant/neoadjuvant therapy
(ie, treatment with curative intent) for colorectal cancer are eligible if it has been
≥ 6 months between the last dose of systemic chemotherapy and the date of informed
consent.
2. Any radiotherapy, chemotherapy, immunotherapy, biologic, investigational, or hormonal
therapy for cancer treatment (except for adjuvant hormonal therapy for breast cancer
or prostate cancer defined as M0 disease or PSA persistence/recurrence without
metastatic disease) within 3 weeks prior to the first dose of trilaciclib/placebo.
3. Receipt of any low-dose systemic chemotherapeutic agent (e.g., low-dose methotrexate
for rheumatoid arthritis) administered for a nononcologic purpose within 3 weeks prior
to the first dose of trilaciclib/placebo.
4. Presence of central nervous system (CNS) metastases/leptomeningeal disease requiring
immediate treatment with radiation therapy or steroids
5. QTcF interval > 450 msec (males) or > 470 msec (females) at screening. For patients
with ventricular pacemakers, QTcF > 500 msec.
6. Personal or family history of long QT syndrome
7. Symptomatic peripheral neuropathy
8. History of interstitial lung disease (ILD)
9. Prior allogeneic or autologous hematopoietic stem cell or bone marrow transplantation
Drug: Trilaciclib, Drug: Placebo
Colorectal Cancer Metastatic, Colon, Rectum, Myelosuppression-Adult, Chemotherapeutic Toxicity
Study of Elafibranor in Patients With Primary Biliary Cholangitis (PBC) (ELATIVE)
The main objective of the study is to evaluate the effect of daily oral administration of
80mg elafibranor on cholestasis (impairment of bile formation and/or bile accumulation) in
patients with PBC and inadequate response or intolerance to Ursodeoxycholic Acid (UCDA)
Call 214-648-5005 studyfinder@utsouthwestern.edu
Marlyn Mayo
14698
All
18 Years to 75 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT04526665
STU-2020-1159
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:
• Males or females age of 18 to 75 years (inclusive)
• Definite or probable PBC diagnosis
• ALP ≥ 1.67x upper limit of normal (ULN)
• Total bilirubin (TB) ≤ 2x ULN
• UDCA for at least 12 months (stable dose ≥ 3 months) prior to screening, or unable to
tolerate UDCA treatment (no UDCA for ≥ 3 months) prior to screening (per country
standard-of-care dosing)
• Females participating in this study must be of non-child bearing potential or must be
using highly efficient contraception for the full duration of the study and for 1
month after the last drug intake
Exclusion Criteria:
• History or presence of other concomitant liver disease
• Clinically significant hepatic decompensation, including patients with
cirrhosis/portal hypertension complications
• Medical conditions that may cause non-hepatic increases in ALP (e.g., Paget's disease)
or which may diminish life expectancy to < 2 years, including known cancers
• Patient has a positive test for Human Immunodeficiency Virus (HIV)
• Evidence of any other unstable or untreated clinically significant disease
• History of alcohol abuse
• For female patients: known pregnancy or lactating
• Use of fibrates and glitazones within 2 months prior to screening
• Use of azathioprine, cyclosporine, methotrexate, mycophenolate, pentoxifylline,
budesonide and other systemic corticosteroids; potentially hepatotoxic drugs
(including α-methyl-dopa, sodium valproic acid isoniazid, or nitrofurantoin) within 3
months prior to screening
• Use of antibodies or immunotherapy directed against interleukins (ILs) or other
cytokines or chemokines within 12 months prior to screening
• For patients with previous exposure to obeticholic acid (OCA), OCA should be
discontinued 3 months prior to screening
• Patients who are currently participating in, plan to participate in, or have
participated in an investigational drug study or medical device study containing
active substance within 30 days or five half-lives, whichever is longer, prior to
screening; for patients with previous exposure to seladelpar, seladelpar should be
discontinued 3 months prior to screening.
• Patients with previous exposure to elafibranor
• Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) > 5x ULN
• Albumin < 3.0 g/dL
• PBC Severely advanced patients according to Rotterdam criteria (TB > ULN and albumin <
LLN)
• International normalized ratio (INR) > 1.3 due to altered hepatic function
• Creatine phosphokinase CPK > 2X ULN
• Serum creatinine > 1.5 mg/dL
• Significant renal disease
Testicular Tissue Cryopreservation for Fertility Preservation
Testicular tissue cryopreservation is an experimental procedure where a young boy's
testicular tissue is retrieved and frozen. This technique is reserved for young male patients
who are not yet producing mature sperm, with the ultimate goal that their tissue may be used
in the future to restore fertility when experimental techniques emerge from the research
pipeline.
• Be male at any age.
• Be scheduled to undergo surgery, chemotherapy, drug treatment and/or radiation for the
treatment or prevention of a medical condition or malignancy with risk of causing
permanent and complete loss of subsequent testicular function.
• Or, have a medical condition or malignancy that requires removal of all or part of one
or both testicles.
• Have newly diagnosed or recurrent disease. Those who were not enrolled at the time of
initial diagnosis (i.e., patients with recurrent disease) are eligible if they have
not previously received therapy that is viewed as likely to result in complete and
permanent loss of testicular function.
• Have two testicles if undergoing elective removal of all or part of a testicle for
fertility preservation only. Note: removal of both testicles will limit fertility
preservation options.
• Sign an approved informed consent and authorization permitting the release of personal
health information. The patient and/or the patient's legally authorized guardian must
acknowledge in writing that consent for specimen collection has been obtained, in
accordance with institutional policies approved by the U.S. Department of Health and
• Consent for serum screening tests for infectious diseases [HIV-1, HIV-2, Hepatitis B,
Hepatitis C], to be performed at the time of testicular tissue harvesting.
• Undergo a full history and physical examination and obtain standard pre-operative
clearance (based on the most recent ACC/AHA Guideline for Perioperative Cardiovascular
Evaluation for Noncardiac Surgery) as determined by their primary surgeon.
• Participating in long term follow-up is a requirement of the protocol.
Exclusion Criteria:
• Diagnosed with psychological, psychiatric, or other conditions which prevent giving
fully informed consent.
• Diagnosed with an underlying medical condition that significantly increases their risk
of complications from anesthesia and surgery.
Procedure: Testicular biopsy
Lymphoma, Sarcoma, Cancer, Mycosis Fungoides, Brain and Nervous System, Other, Eye and Orbit, Anklylosing Spondylitis, Anus, Bones and Joints, Breast - Male, Carcinoid Tumor, Cardiovascular, Colon, Esophagus, Gall Bladder, Head and Neck, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung/Thoracic, Nose, Other Digestive Organ, Other Endocrine System, Other Male Genital, Other Respiratory and Intrathoracic Organs, Other Skin, Other Urinary, Pancreas, Prostate, Rectum, Stomach, Throat, Thyroid, Urinary Bladder, Leukemia, Other, Hodgkins Lymphoma, Heart, Leukemia, Not Otherwise Specified, Lymphoid Leukemia, Myeloid and Monocytic Leukemia, Non-Hodgkins Lymphoma, Other Hematopoietic, Small Intestine, Soft Tissue, Unknown Sites, Ill - Defined Sites, Autoimmune Disorders
Comparing Virtual Reality (VR) to Non-VR for Decreasing Preoperative/Procedural Anxiety
The primary objective of the study is to utilize the modified Yale Preoperative Anxiety scale
(mYPAS), a validated preoperative/procedural anxiety score, to measure preoperative anxiety
via distraction in pediatric oncology patients undergoing port access.
The hypothesis is that using Virtual Reality (VR) will objectively decrease anxiety scores
measured by mYPAS by five percent (primary outcome).
The secondary outcome will be the parents or the legally authorized representative (LAR)
subjective reports of anxiety with the use of VR.
The Kind VR device is used in house at Children's Health in the Dallas and Plano campuses.
The VR device used in this study qualifies as exempt from FDA IDE regulations. It is a
non-significant risk, non-invasive, interactive video device the user wears like goggles. The
study carries minimal risks to the subjects and is designed to minimize patient discomfort
from placement or motion sickness. Furthermore, the device has disposable covers for
protection against infection and can be sanitized between uses, once the disposable covers
are removed. Children's Health System of Texas (CHST) and this research group are not
partnering entities with the Kind VR, and the Kind VR device is not being studied. The effect
of virtual reality (VR) on preprocedural anxiety as measured by questionnaires and the
observations of the modified Yale Preoperative Anxiety Scale (mYPAS) is being studied
Most patients coming to the Clinic of Cancer and Blood Disorders (CCBD) are under chronic
care for their ongoing disease and are likely to be coming to the CCBD at least twice in a
6-month period. The CCBD schedule will be reviewed by the researchers for patients age 5-12,
requiring port access at least twice during the next six-month period. Patient families whose
child meets the basic screening criteria, and have no exclusion criteria, will be approached
privately as possible participants in the study. Up to 100 subjects will be enrolled over a
2-year period. Once the subject/parent or LAR agrees to participate, study staff will
randomize the subjects into which standard of care distraction method for anxiety management
they will receive first in this study.
• Any patient of the Children's Medical Center CCBD
• 5-12 years of age
• Patient requiring their port accessed twice or more within a 6 months period
• Ability to understand and the willingness to sign a written informed consent.
Exclusion Criteria
• Subjects younger than 5 and older than 12
• Patients requiring recovery in PICU or sites other than PACU
• If parents or subject is not willing to participate
• Subjects with severe developmental delays and subjects with developmental challenges
preventing them from keeping the VR device on are also excluded
• Patients who will not be in CCBD for port access at least twice in 6 months
Other: Virtual reality
Lymphoma, Sarcoma, Multiple Myeloma, Brain and Nervous System, Eye and Orbit, Anus, Bones and Joints, Breast - Female, Breast - Male, Carcinoid Tumor, Cervix, Colon, Corpus Uteri, Ear, Esophagus, Gall Bladder, Head and Neck, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung/Thoracic, Melanoma, skin, Nose, Ovary, Pancreas, Prostate, Rectum, Stomach, Throat, Thyroid, Urinary Bladder, Uterine (Endometrial), Vulva, Leukemia, Other, Hodgkins Lymphoma, Leukemia, Not Otherwise Specified, Lymphoid Leukemia, Myeloid and Monocytic Leukemia, Non-Hodgkins Lymphoma, Small Intestine, Soft Tissue, Preoperative Anxiety
Testing the Addition of an Anti-Cancer Immunotherapy Drug, Avelumab, to Gemcitabine and Carboplatin Chemotherapy Prior to Surgery in Muscle Invasive Urinary Tract Cancer vs. Surgery Alone in Patients Who Are Not Able to Receive Cisplatin Therapy (SWOG GAP TRIAL)
This phase II trial studies the effect of avelumab, gemcitabine and carboplatin before
surgery compared with surgery alone in treating patients with muscle invasive bladder or
upper urinary tract cancer who are not able to receive cisplatin therapy. Immunotherapy with
monoclonal antibodies, such as avelumab, may help the body's immune system attack the cancer,
and may interfere with the ability of tumor cells to grow and spread. Chemotherapy drugs,
such as gemcitabine and carboplatin, work in different ways to stop the growth of tumor
cells, either by killing the cells, by stopping them from dividing, or by stopping them from
spreading. Giving avelumab together with gemcitabine and carboplatin before surgery may work
better in lowering the chance of muscle invasive urinary tract cancer growing or spreading,
in patients who cannot receive cisplatin therapy compared to surgery alone.
• Participants must have one of the following:
• Histologically documented muscle-invasive bladder carcinoma (MIBC) from
transurethral resection of bladder tumor (TURBT) within 56 days prior to
registration
• Histologically confirmed high grade upper tract urothelial carcinoma (UTUC)
within 56 days prior to registration, with invasion confirmed by either a mass on
cross-sectional imaging or a tumor directly visualized during upper urinary tract
endoscopy within 56 days prior to registration
• Participants diagnosed with mixed urothelial carcinoma and variant histology
within 56 days prior to registration may be eligible if the majority (> 50%) of
the tumor consists of urothelial carcinoma. Participants with pure non-urothelial
variant histologies or any small cell histology are not eligible
• Participants must have clinical stage T2-T4aN0M0 bladder or upper tract cancer
confirmed by radiologic staging (computed tomography [CT] scan/magnetic resonance
imaging [MRI] abdomen and pelvis, and CT scan/x-ray of the chest) within 56 days prior
to registration
• Participants must have a bone scan within 56 days prior to registration if they have
bone pain or elevated serum alkaline phosphatase
• Participants must have a bimanual examination under anesthesia within 56 days prior to
registration
• Participants must not have received prior systemic chemotherapy, immunotherapy or
radiotherapy for the treatment of muscle invasive bladder cancer (MIBC) or upper tract
urothelial carcinoma (UTUC). Other prior pelvic radiotherapy is allowed if it does not
preclude surgery (radical cystectomy, nephroureterectomy or ureterectomy, based on
location of primary tumor). Prior intravesical therapy is allowed
• Participants must not have received immunosuppressive medication within 14 days prior
to registration, with the exception of intranasal, inhaled, topical steroids, or local
steroid injections (e.g., intra-articular injection) systemic corticosteroids at
physiologic doses not to exceed 10 mg/day of prednisone or its equivalent
• Participants must be >= 18 years of age
• Participants must have Zubrod performance status 0-2
• Participants must have history and physical examination within 28 days prior to
registration
• Participants must be surgical candidates as deemed by the local site oncologic surgeon
within 28 days prior to registration. This must be clearly documented
• Participants must have a serum creatinine =< the institutional upper limit of normal
(IULN) OR measured OR calculated creatinine clearance >= 30 mL/min using the
Crockroft-Gault Formula. This specimen must have been drawn and processed within 28
days prior to registration
• Participants must be deemed cisplatin-ineligible based on greater than or equal to 1
of the following:
• Zubrod performance status = 2
• Creatinine clearance (calculated by Crockroft-Gault formula or measured) 30 to <
60 ml/min,
• Neuropathy > grade 1
• Hearing loss > grade 1
• Congestive heart failure > grade 2
• Hemoglobin >= 9.0 g/dL (within 28 days prior to registration)
• Absolute neutrophil count >= 1,500/mcL (within 28 days prior to registration)
• Platelets >= 100,000/mcL (within 28 days prior to registration)
• Total bilirubin =< 1.5 x institutional upper limit of normal (ULN) (within 28 days
prior to registration)
• Aspartate aminotransferase (AST) =< 2.5 x institutional ULN (within 28 days prior to
registration)
• Alanine aminotransferase (ALT) =< 2.5 x institutional ULN (within 28 days prior to
registration)
• Participants with known history or current symptoms of cardiac disease, or history of
treatment with cardiotoxic agents, must have a clinical risk assessment of cardiac
function using the New York Heart Association Functional Classification and be class
2B or better
• Participants with known human immunodeficiency virus (HIV) must be on effective
anti-retroviral therapy and have undetectable viral load at their most recent viral
load test and within 6 months prior to registration
Exclusion Criteria:
• Participant must not have any other prior malignancy except for the following:
adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer,
prostate cancer Gleason score =< 3+4 in active surveillance, adequately treated stage
I or II cancer from which the participant is currently in complete remission, or any
other cancer from which the participant has been disease free for two years
• Participants must not be pregnant or nursing due to the risk of harm to a fetus or
nursing infant. Women/men of reproductive potential must have a negative serum or
urine pregnancy test within 28 days prior to registration and must have agreed to use
an effective contraceptive method. A woman is considered to be of "reproductive
potential" if she has had menses at any time in the preceding 12 consecutive months.
In addition to routine contraceptive methods, "effective contraception" also includes
heterosexual celibacy and surgery intended to prevent pregnancy (or with a side-effect
of pregnancy prevention) defined as a hysterectomy, bilateral oophorectomy or
bilateral tubal ligation. However, if at any point a previously celibate participant
chooses to become heterosexually active during the time period for use of
contraceptive measures outlined in the protocol, he/she is responsible for beginning
contraceptive measures
• Participants must not have a history of active primary immunodeficiency
• Participants must not have a history of or active autoimmune or inflammatory disorder,
with the exception of vitiligo, alopecia, hypothyroidism (stable on hormone
replacement), or chronic skin condition that does not require systemic therapy
Bladder Carcinoma Infiltrating the Muscle of the Bladder Wall, Stage II Bladder Cancer AJCC v8, Urinary Bladder, Infiltrating Renal Pelvis and Ureter Urothelial Carcinoma
The iCat2, GAIN (Genomic Assessment Informs Novel Therapy) Consortium Study
This research study is evaluating the use of specialized testing of solid tumors including
sequencing. The process of performing these specialized tests is called tumor profiling. The
tumor profiling may result in identifying changes in genes of the tumor that indicate that a
particular therapy may have activity. This is called an individualized cancer therapy (iCat)
recommendation. The results of the tumor profiling and, if applicable, the iCat
recommendation will be returned.
• Age -- Age ≤ 30 years at time of initial qualifying solid tumor diagnosis
• Diagnosis -- Histologic diagnosis of solid malignancy (excluding brain tumors and
lymphoma) that meets at least one of the following criteria:
• Refractory, defined as tumor progression after initiation of standard first line
therapy without having achieved a prior partial or complete remission OR Biopsy
proven residual disease at the completion of planned standard initial front-line
therapy.
• Recurrent, defined as tumor progression after achieving a prior partial or
complete remission
• Newly diagnosed high risk disease, defined as having an expected event free
survival of < 50% at 2 years.
• Lacks definitive diagnosis or classical genomic findings after histologic review
and standard molecular testing (rare tumor group).
• Examples include (eligibility not limited to these examples):
• Histology typically associated with a fusion in which fusion is not detected.
• Ewing-like sarcoma
• Undifferentiated sarcoma
• Inflammatory myofibroblastic tumor without ALK fusion
• Infantile fibrosarcoma without NTRK fusion
• Specimen Samples
• Sufficient tumor specimen available to meet the minimum requirements for
profiling from diagnosis or progression / recurrence
--- OR
• Surgery / biopsy planned as part of clinical care that is anticipated to yield
sufficient material to meet the minimum requirements for profiling; OR
• Patient has already had molecular profiling and patient has not yet started
matched targeted therapy based on the report .
Exclusion Criteria:
• No Therapy Planned
-- Patients who have declined further anticancer therapy will be excluded.
• Performance Status
-- Patients with Lansky (age < 16 years) or Karnofsky (age ≥16 years) score < 50 will
be excluded.
• Life Expectancy -- Patients with anticipated life expectancy < 3 months will be
excluded.
Genetic: Genetic testing and GAIN report
Sarcoma, Pediatric Solid Tumor, Brain and Nervous System, Other, Eye and Orbit, Anklylosing Spondylitis, Anus, Bones and Joints, Breast - Female, Breast - Male, Carcinoid Tumor, Cervix, Colon, Corpus Uteri, Ear, Esophagus, Gall Bladder, Head and Neck, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung/Thoracic, Melanoma, skin, Nose, Other Digestive Organ, Other Endocrine System, Other Female Genital, Other Male Genital, Other Respiratory and Intrathoracic Organs, Other Skin, Other Urinary, Ovary, Pancreas, Prostate, Rectum, Stomach, Throat, Thyroid, Urinary Bladder, Uterine (Endometrial), Vulva, Heart, Kaposis sarcoma, Small Intestine, Soft Tissue, Ill - Defined Sites
Cisplatin and Combination Chemotherapy in Treating Children and Young Adults With Hepatoblastoma or Liver Cancer After Surgery
This partially randomized phase II/III trial studies how well, in combination with surgery,
cisplatin and combination chemotherapy works in treating children and young adults with
hepatoblastoma or hepatocellular carcinoma. Drugs used in chemotherapy, such as cisplatin,
doxorubicin, fluorouracil, vincristine sulfate, carboplatin, etoposide, irinotecan,
sorafenib, gemcitabine and oxaliplatin, work in different ways to stop the growth of tumor
cells, either by killing the cells, by stopping them from dividing, or by stopping them from
spreading. Giving combination chemotherapy may kill more tumor cells than one type of
chemotherapy alone.
• Patients in Group F must have a body surface area (BSA) >= 0.6 m^2
• Patients must have a performance status corresponding to Eastern Cooperative Oncology
Group (ECOG) scores of 0, 1, or 2; use Karnofsky for patients > 16 years of age and
Lansky for patients =< 16 years of age; patients who are unable to walk because of
paralysis, but who are up in a wheelchair, will be considered ambulatory for the
purpose of assessing the performance score
• Patients must be newly diagnosed with histologically-proven primary pediatric hepatic
malignancies including hepatoblastoma or hepatocellular carcinoma, except as noted
below; patients with a diagnosis of hepatocellular neoplasm, not otherwise specified,
should be classified and treated per hepatoblastoma treatment arms; note that rapid
central pathology review is required in some cases; please note: all patients with
histology as assessed by the institutional pathologist consistent with pure small cell
undifferentiated (SCU) HB will be required to have testing for INI1/SMARCB1 by
immunohistochemistry (IHC) according to the practices at the institution
• Patients with histology consistent with pure SCU must have positive INI1/SMARCB1
staining
• For all Group A patients, WDF status as determined by rapid review will be used to
further stratify patients to Group A1 or A2
• For Groups B, C and D, rapid review is required if patients are either >= 8 years
of age or have an alphafetoprotein (AFP) =< 100 at diagnosis
• For all Groups E and F patients, rapid central pathology review is required
• In emergency situations when a patient meets all other eligibility criteria and has
had baseline required observations, but is too ill to undergo a biopsy safely, the
patient may be enrolled without a biopsy
• Clinical situations in which emergent treatment may be indicated include, but are
not limited to, the following circumstances:
• Anatomic or mechanical compromise of critical organ function by tumor (e.g.,
respiratory distress/failure, abdominal compartment syndrome, urinary
obstruction, etc.)
• Uncorrectable coagulopathy
• For a patient to maintain eligibility for AHEP1531 when emergent treatment is
given, the following must occur:
• The patient must have a clinical diagnosis of hepatoblastoma, including an
elevated alphafetoprotein (AFP), and must meet all AHEP1531 eligibility
criteria at the time of emergent treatment
• Patient must be enrolled on AHEP1531 prior to initiating protocol therapy; a
patient will be ineligible if any chemotherapy is administered prior to
AHEP1531 enrollment
• Note: If the patient receives AHEP1531 chemotherapy emergently PRIOR to
undergoing a diagnostic biopsy, pathologic review of material obtained in the
future during either biopsy or surgical resection must either confirm the
diagnosis of hepatoblastoma or not reveal another pathological diagnosis to be
included in the analysis of the study aims
• Patients may have had surgical resection of the hepatic malignancy prior to
enrollment; all other anti-cancer therapy for the current liver lesion is prohibited
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 60
mL/min/1.73 m^2 or
• A serum creatinine based on age/gender as follows:
• Age: maximum serum creatinine (mg/dL)
• 1 month to < 6 months: 0.4 (male and female)
• 6 months to < 1 year: 0.5 (male and female)
• 1 to < 2 years: 06 (male and female)
• 2 to < 6 years: 0.8 (male and female)
• 6 to < 10 years: 1 (male and female)
• 10 to < 13 years: 1.2 (male and female)
• 13 to < 16 years: 1.5 (male), 1.4 (female)
• >= 16 years: 1.7 (male), 1.4 (female)
• Total bilirubin =< 5 x upper limit of normal (ULN) for age
• Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase
[SGOT])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT])
< 10 x upper limit of normal (ULN) for age
• Shortening fraction of >= 28% by echocardiogram (for patients on
doxorubicin-containing regimens [Groups C, D, E2, and F] assessed within 8 weeks prior
to study enrollment) or
• Ejection fraction of >= 47% by echocardiogram or radionuclide angiogram (for patients
on doxorubicin-containing regimens [Groups C, D, E2, and F] assessed within 8 weeks
prior to study enrollment)
• Group F patients only: QT/corrected QT (QTc) interval =< 450 milliseconds for males
and =< 470 milliseconds for females (assessed within 8 weeks prior to study
enrollment)
• Normal pulmonary function tests (including diffusion capacity of the lung for carbon
monoxide [DLCO]) if there is clinical indication for determination (e.g. dyspnea at
rest, known requirement for supplemental oxygen) (for patients receiving chemotherapy
[Groups A, B, C, D, E2, F]); for patients who do not have respiratory symptoms or
requirement for supplemental oxygen, pulmonary function tests (PFTs) are NOT required
• All patients and/or their parents or legal guardians must sign a written informed
consent
• All institutional, Food and Drug Administration (FDA), and National Cancer Institute
(NCI) requirements for human studies must be met
Exclusion Criteria:
• Prior chemotherapy or tumor directed therapy (i.e. radiation therapy, biologic agents,
local therapy (embolization, radiofrequency ablation, and laser); therefore, patients
with a pre-disposition syndrome who have a prior malignancy are not eligible
• Patients who are currently receiving another investigational drug
• Patients who are currently receiving other anticancer agents
• Patients with uncontrolled infection
• Patients who previously received a solid organ transplant, other than those who
previously received an orthotopic liver transplantation (OLT) as primary treatment of
their hepatocellular carcinoma
• Patients with hypersensitivity to any drugs on their expected treatment arm
• Group C: Patients who have known deficiency of dihydropyrimidine dehydrogenase (DPD)
• Group D:
• Patients with chronic inflammatory bowel disease and/or bowel obstruction
• Patients with concomitant use of St. John's wort, which cannot be stopped prior
to the start of trial treatment
• Group F:
• Patients with peripheral sensitive neuropathy with functional impairment
• Patients with a personal or family history of congenital long QT syndrome
• These criteria apply ONLY to patients who may receive chemotherapy (all groups other
than Group E1):
• 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
• Note for Group F: patients of childbearing potential should use effective
birth control during treatment with sorafenib and for at least 2 weeks after
stopping treatment
Derazantinib in Subjects With FGFR2 Gene Fusion-, Mutation- or Amplification- Positive Inoperable or Advanced Intrahepatic Cholangiocarcinoma (FIDES-01)
This pivotal, open-label, single-arm study will evaluate the anti-cancer activity of
derazantinib by Objective Response Rate (ORR) by central radiology review as per RECIST v1.1
in subjects with inoperable or advanced intrahepatic cholangiocarcinoma (iCCA) whose tumors
harbor FGFR2 gene fusions (by FISH performed by the central laboratory) or FGFR2 gene
mutations or amplifications (based on NGS testing performed or commissioned by the respective
study center) and who received at least one prior regimen of systemic therapy. Subjects will
be dosed orally once per day at 300 mg of derazantinib capsules.
1. Signed written informed consent granted prior to initiation of any study-specific
procedures
2. 18 years of age or older
3. Histologically or cytologically confirmed locally advanced, inoperable (where surgery
is not indicated due to disease extension, co-morbidities, or other technical
reasons), or metastatic iCCA or mixed histology tumors (combined
hepatocellular-cholangiocarcinoma [cHCC-CCA])
4. Substudy 1: FGFR2 fusion status based on the following assessments:
a) If central laboratory designated by Sponsor: Positive FISH test; and/or b) If
non-central laboratory: i) Positive FISH or NGS test: patients may be enrolled and may
start dosing, but central confirmation is required* ii) Negative FISH or NGS test:
tissue may be submitted to the central laboratory designated by the Sponsor, and
patients may only be enrolled if the central test is positive
*Using standard protocols and approved by local IRB/EC, by CLIA or other similar
agency.
Substudy 2: FGFR2 mutation/amplification status based on local NGS testing performed
or commissioned by the respective study site.
5. Received at least one regimen of prior systemic therapy and then experienced
documented radiographic progression
6. Measurable disease by RECIST version 1.1 criteria
7. ECOG performance status ≤ 1
8. Adequate organ functions as indicated by the following laboratory values (based on
screening visit values from the central laboratory).
• Hematological
• Hemoglobin (Hgb) ≥ 9.0 g/dL
• Absolute neutrophil count (ANC) ≥ 1.5 x 10^9/L
• Platelet count ≥ 75 x 109/L
• International normalized ratio (INR) 0.8 to upper limit of normal (ULN) or ≤
3 for subjects receiving anticoagulant therapy such as Coumadin or heparin
• Hepatic
• Total bilirubin ≤ 2 x ULN
• AST and ALT ≤ 3 ULN (≤ 5 x ULN for subjects with liver metastases)
• Albumin ≥ 2.8 g/dL
• Renal
• Serum creatinine ≤ 1.5 x ULN
• Creatinine clearance of ≥ 30 mL/min as estimated by the Cockcroft-Gault
equation
9. Female and male patients of child-producing potential must agree to avoid becoming
pregnant or impregnating a partner, respectively, use double-barrier contraceptive
measures, oral contraception, or avoidance of intercourse, during the study*, and
until at least 120 for 90 days after the last dose of derazantinib.
*From the day of first study medication, or for oral contraception from 14 days before
first study medication.
Male patients are considered not to be of child-producing potential if they have
azoospermia (whether due to vasectomy or an underlying medical condition). Female
patients are considered not to be of child-producing potential if they are:
• postmenopausal* , or
• have had a hysterectomy and/or bilateral oophorectomy, bilateral salpingectomy or
bilateral tubal ligation/occlusion, at least 6 weeks prior to screening, or
• have a congenital or acquired condition that prevents childbearing.
Male or female patients of child-producing potential must agree to comply with one of
the following until at least 120 days after the last dose of derazantinib:
1. Abstinence from heterosexual activity**
2. Using (or having their partner use) an acceptable method of contraception during
heterosexual activity. Acceptable methods of contraception are***:
• any ONE of:
•an intrauterine device (IUD)
•vasectomy of a female patient's male partner
•a contraceptive rod implanted into the skin.
• any TWO in combination of:
•diaphragm with spermicide (cannot be used in conjunction with cervical
cap/spermicide)
•cervical cap with spermicide (nulliparous women only)
•contraceptive sponge (nulliparous women only)
•male condom or female condom (cannot be used together)
•hormonal contraceptive (oral contraceptive pill [estrogen/progestin pill
or progestin-only pill], contraceptive skin patch, vaginal contraceptive
ring, or subcutaneous contraceptive injection)
*Postmenopausal is defined as at least 12 months with no menses without an
alternative medical cause; in women < 45 years of age a high follicle
stimulating hormone (FSH) level in the postmenopausal range may be used to
confirm a post -menopausal state in women not using hormonal contraception
or hormonal replacement therapy. In the absence of 12 months of amenorrhea,
a single FSH measurement is not sufficient.
• Abstinence (relative to heterosexual activity) can be used as the sole
method of contraception if it is consistently employed as the subject's
preferred and usual lifestyle and if considered acceptable by local
regulatory agencies and ERCs/IRBs. Periodic abstinence (e.g., calendar,
ovulation, sympto-thermal, post-ovulation methods, etc.) and withdrawal
are not acceptable methods of contraception.
• If a contraceptive method listed above is restricted by local
regulations/guidelines, then it does not qualify as an acceptable
method of contraception for subjects participating at sites in
this country/region.
Exclusion Criteria:
1. Receipt of treatment before the first dose of study drug (Cycle 1 Day 1) within an
interval shorter than the following, as applicable:
• One chemotherapy or biological (e.g., antibody) cycle interval
• Five half-lives of any small-molecule investigational or licensed medicinal
product
• Two weeks, for any investigational medicinal product with an unknown half-life
• Four weeks of curative radiotherapy
• Seven days of palliative radiotherapy
• 28 days of radiotherapy
2. Major surgery, locoregional therapy, or radiation therapy within four weeks of the
first dose of derazantinib
3. Previous treatment with any FGFR inhibitor (e.g., Balversa® [erdafitinib], Pemazyre®
[pemigatinib], infigratinib, rogaratinib, futibatinib, lenvatinib, ponatinib,
dovitinib, nintedanib, AZD4547, LY2784455).
•Subjects who received less than four weeks of therapy and were unable to continue
therapy due to toxicity will be allowed to participate
4. Unable or unwilling to swallow the complete daily dose of derazantinib capsules
5. Clinically unstable central nervous system (CNS) metastases (to be eligible, subjects
must have stable disease ≥ 3 months, confirmed by magnetic resonance imaging (MRI) or
computed tomography (CT) scan, and/or have CNS metastases well controlled by low-dose
steroids, anti-epileptics, or other symptom-relieving medications)
6. Current evidence of clinically significant corneal or retinal disorder likely to
increase the risk of eye toxicity, including but not limited to bullous/band
keratopathy, keratoconjunctivitis (unless keratoconjunctivitis sicca), corneal
abrasion, inflammation/ulceration, confirmed by ophthalmologic examination.
7. Concurrent uncontrolled or active hepatobiliary disorders, untreated or ongoing
complications after laparoscopic procedures or stent placement, including but not
limited to active cholangitis, biloma or abscess (to be eligible, the subjects have to
be treated and disorders/complications should be resolved within 2 weeks prior to the
first dose of derazantinib)
8. History of significant cardiac disorders:
• Myocardial infarction (MI) or congestive heart failure defined as Class II to IV
per the New York Heart Association (NYHA) classification within 6 months of the
first dose of derazantinib (MI that occurred > 6 months prior to the first dose
of derazantinib will be permitted)
• QTcF >450 msec (males or females)
9. Serum electrolyte abnormalities defined as follows:
•Hyperphosphataemia: Serum phosphate > institutional ULN
•Hyperkalemia: > 6.0 mmol/L
•Hypokalemia: < 3.0 mmol/L
•Hypercalcemia: corrected serum calcium < 1.75 mmol/L (< 7.0 mg/dL)
• Hypocalcemia: corrected serum calcium > 3.1 mmol/L (> 12.5 mg/dL)
• Hypomagnesemia: < 0.4 mmol/L (< 0.9 mg/dL)
10. Significant gastrointestinal disorder(s) that could, in the opinion of the
Investigator, interfere with the absorption, metabolism, or excretion of derazantinib
(e.g., Crohn's disease, ulcerative colitis, extensive gastric resection)
11. History of additional malignancy that is progressing or requires active treatment.
Exceptions include basal cell carcinoma of the skin, squamous cell carcinoma of the
skin that has undergone potentially curative therapy, and in situ cervical cancer.
12. Concurrent uncontrolled illness not related to cancer, including but not limited to:
• Psychiatric illness/substance abuse/social situation that would limit compliance
with study requirements
• Known uncontrolled human immunodeficiency virus (HIV) infection
• Severe bacterial, fungal, viral and/or parasitic infections on therapeutic oral
or IV medication at the time of first dose of study drug administration
13. Blood or albumin transfusion within 5 days of the blood draw being used to confirm
eligibility
14. Pregnant or breast feeding
15. Known hypsersensitivity to derazantinib, or to any of the study drug excipients
(starch, lactose, crospovidone, magnesium stearate)
Drug: derazantinib
Intrahepatic Cholangiocarcinoma, Combined Hepatocellular and Cholangiocarcinoma, Liver
iCCA, intrahepatic cholangiocarcinoma, FGFR2 gene fusion or FGFR2 gene mutation or amplification, biliary cancer, bile duct cancer, FGFR2 gene rearrangement, liver cancer, targeted therapy, combined hepatocellular and cholangiocarcinoma, cHCC-CCA, derazantinib
A Study of Tivozanib in Combination With Durvalumab in Subjects With Advanced Hepatocellular Carcinoma (DEDUCTIVE)
This study will evaluate the safety, tolerability, DLTs, MTD, and preliminary anti tumor
activity of tivozanib in combination with durvalumab in subjects with advanced HCC.
1. ≥ 18 years old
2. Signed and dated written informed consent
3. Histologically or cytologically confirmed unresectable locally advanced or metastatic
hepatocellular carcinoma. Measurable or evaluable disease by RECIST 1.1 criteria.
Patients can be either untreated or have progressed on both bevacizumab and
atezolizumab.
4. Child-Pugh Class A.
5. ECOG performance status ≤ 1 (see Appendix A) and life expectancy ≥ 3 months.
6. Body weight > 30 kg.
7. Measured creatinine clearance (crCL) >40 mL/min or calculated crCL >40 mL/min as
determined by Cockcroft-Gault (using actual body weight).
8. Sexually active pre-menopausal female subjects (and female partners of male subjects)
must use highly effective contraceptive measures, while on study and for at least 90
days after the last dose of study drug. Sexually active male subjects must use
adequate contraceptive measures, while on study and for at least 90 days after the
last dose of study drug. All fertile male and female subjects and their partners must
agree to use a highly effective method of contraception.
Exclusion Criteria:
1. Subjects who have received prior systemic treatment for HCC except for both
bevacizumab and atezolizumab.
2. Female subjects who are pregnant or breastfeeding or male or female subjects of
reproductive potential who are not willing to employ effective birth control from
screening to 90 days after the last dose of study drug.
3. Brain metastases or spinal cord compression. Subjects with suspected brain metastases
at screening should have an MRI (preferred) or CT scan each preferable with IV
contrast of the brain prior to study entry. Brain metastases will not be recorded on
RECIST Target Lesions at baseline.
4. Any of the following hematologic abnormalities:
• Hemoglobin < 9.0 g/dL
• Absolute neutrophil count (ANC) < 1500 per mm3
• Platelet count < 75,000 per mm3
5. Any of the following serum chemistry or urinalysis abnormalities:
• Total bilirubin > 2 × ULN (>2.5 mg/dL in subjects with Gilbert's syndrome)
• AST or ALT > 5 × ULN
• Alkaline phosphatase > 2.5 × ULN (or > 5 × ULN for subjects with liver or bone
metastasis)
• Serum creatinine > 1.5 × ULN •> 2+ proteinuria
6. History of hepatic encephalopathy within past 12 months or requirement for medications
to prevent or control encephalopathy (eg, no lactulose, rifaximin, etc if used for
purposes of hepatic encephalopathy).
7. GI Bleeding (eg, esophageal varices or ulcer bleeding) within 12 months. (Note: For
patients with a history of GI bleeding for more than 12 months or assessed as high
risk for esophageal variceal bleed by the Investigator, adequate endoscopic therapy
according to institutional standards is required).
8. Clinically meaningful ascites defined as ascites requiring non-pharmacologic
intervention (eg, paracentesis) to maintain symptomatic control, within 6 months prior
to the first scheduled dose. Subjects on stable doses of diuretics for ascites for ≥ 2
months are eligible.
9. Main portal vein thrombosis (Vp4) as documented on imaging. (VP4 is defined as portal
vein thrombosis in the main trunk of the portal vein or a portal vein branch
contralateral to the primarily involved lobe (or both).
10. For subjects who require ongoing therapeutic anti-coagulation or anti-platelet
therapy; the subject must be off either therapy for at least 7 days prior to the first
dose of investigational product. Low-dose aspirin for cardiac prophylaxis/protection
is permitted per local institutional standards.
11. Patients co-infected with HBV and HCVHBV positive [presence of hepatitis B surface
antigen (HBsAg) and/or hepatitis B core antibodies (anti-HBcAb) with detectable HBV
DNA (≥10IU/ml)]; HCV positive (presence of anti-HCV antibodies).
12. Major surgery (as defined by the investigator) within 28 days prior to first dose of
IP or still recovering from prior surgery. Local procedures (eg, core needle biopsy,
and prostate biopsy) are allowed if completed at least 3 days prior to the
administration of the first dose of study treatment.
13. Significant cardiovascular disease, including:
• Clinically symptomatic heart failure. Subjects with a history of heart failure
must have an ECHO or MUGA scan to document left ventricular ejection fraction
(LVEF) > 45% prior to start of protocol therapy
• Any New York Heart Association classification ≥ Class 2 (prefer Class 0 or 1)
• Any stenting procedure within the last 3 months
• Venous thromboembolism or arterial thromboembolism within the last 3 months
• Any IVC tumor thrombosis
• History of a hemorrhagic event (i.e., GI bleed within 6 months)
• Uncontrolled hypertension: blood pressure >150/95 mmHg on more than 2
antihypertensive medications, on two consecutive measurements obtained at least
24 hours apart. Subjects with a history of hypertension must have been on stable
doses of anti-hypertensive drugs for ≥ 2 weeks prior to start of protocol
therapy.
• Myocardial infarction within 3 months prior to start of protocol therapy
14. Subjects with delayed healing of wounds, ulcers, and/or bone fractures
15. Serious/active infection or infection requiring parenteral antibiotics
16. Inadequate recovery from any prior surgical procedure; major surgical procedure within
4 weeks prior to start of protocol therapy.
17. Inability to comply with protocol requirements
18. History of another primary malignancy except for:
• Malignancy treated with curative intent and with no known active disease ≥ 5
years before the first dose of study drug and low potential risk for recurrence
• Adequately treated non-melanoma skin cancer of lentigo maligna without evidence
of disease
19. Active infection including tuberculosis (clinical evaluation that includes clinical
history, physical examination and radiographic findings, and tuberculosis testing in
line with local practice), hepatitis C, or human immunodeficiency virus (positive HIV
1/2 antibodies). Subjects positive for hepatitis C (HCV) antibody are eligible only if
polymerase chain reaction is negative for HCV RNA.
20. Patients with a history or current HBV infection (detectable HBV DNA), should be
placed on anti-viral treatment and tested at every cycle for HBV DNA viral load.
21. Palliative radiotherapy with a limited field of radiation within 2 weeks or with wide
field of radiation or to more than 30% of the bone marrow within 4 weeks before the
first dose of study intervention.
22. Treatment with systemic hormonal therapy within 3 weeks prior to start of protocol
therapy, with the exception of:
• Hormonal therapy for appetite stimulation or contraception
• Nasal, ophthalmic, inhaled and topical steroid preparations
• Oral replacement therapy for adrenal insufficiency
• Low-dose maintenance steroid therapy (equivalent of prednisone 10mg/day) for
other conditions
• Hormone replacement therapy such as testosterone
23. Strong CYP3A4 inducers (see Appendix B) within 2 weeks prior to start of, or during,
protocol therapy.
24. Prior exposure to tivozanib or durvalumab. For subjects who have received prior
atezolizumab:
• Must not have experienced a toxicity that led to permanent discontinuation of
prior immunotherapy.
• All AEs while receiving prior immunotherapy must have completely resolved or
resolved to baseline prior to screening for this study.
• Must not have experienced a Grade ≥ 3 immune-related AE or an immune-related
neurologic or ocular AE of any grade while receiving prior immunotherapy. Note:
Participants with an endocrine AE of Grade ≤ 2 are permitted to enroll if they
are stably maintained on appropriate replacement therapy and are asymptomatic.
• Must not have required the use of additional immunosuppression other than
corticosteroids for the management of an AE, not have experienced recurrence of
an AE if re-challenged, and not currently require maintenance doses of > 10 mg
prednisone or equivalent per day.
25. History of allogeneic organ transplantation
26. Active or prior documented autoimmune or inflammatory disorders (including
inflammatory bowel disease [e.g., colitis or Crohn's disease], diverticulitis [with
the exception of diverticulosis], systemic lupus erythematosus, Sarcoidosis syndrome,
or Wegener syndrome [granulomatosis with polyangiitis, Graves' disease, rheumatoid
arthritis, hypophysitis, uveitis, etc.]). The following are exceptions to this
criterion:
• Subjects with vitiligo or alopecia
• Subjects with hypothyroidism (e.g., following Hashimoto syndrome) stable on
hormone replacement
• Any chronic skin condition that does not require systemic therapy
• Subjects without active disease in the last 5 years may be included but only
after consultation with Medical Monitor
• Subjects with celiac disease controlled by diet alone
27. Uncontrolled intercurrent illness, including but not limited to, ongoing or active
infection, symptomatic congestive heart failure, uncontrolled hypertension, unstable
angina pectoris, uncontrolled cardiac arrhythmia, active interstitial lung disease,
serious chronic gastrointestinal conditions associated with diarrhea, or psychiatric
illness/social situations that would limit compliance with study requirement,
substantially increase risk of incurring AEs or compromise the ability of the subject
to give written informed consent
28. History of leptomeningeal carcinomatosis
29. History of active primary immunodeficiency
30. Mean QT interval corrected for heart rate using Fridericia's formula (QTcF) ≥470 ms
calculated from 3 ECGs (within 15 minutes at 5 minutes apart)
31. Known allergy or hypersensitivity to any of the study drugs or any of the study drug
excipients
32. Concurrent enrollment in another clinical study, unless it is an observational (non
interventional) clinical study or during the follow-up period of an interventional
study
33. Receipt of live attenuated vaccine within 30 days prior to the first dose of study
drug. Note: Subjects, if enrolled, should not receive live vaccine whilst receiving
study drug and up to 30 days after the last dose of study drug.
34. Current or prior use of immunosuppressive medication within 14 days before the first
dose of durvalumab. The following are exceptions to this criterion:
• Intranasal, inhaled, topical steroids, or local steroid injections (e.g., intra
articular injection)
• Systemic corticosteroids at physiologic doses not to exceed 10 mg/day of
prednisone or its equivalent
• Steroids as premedication for hypersensitivity reactions (e.g., CT scan
premedication)
35. Previous study drug assignment in the present study.
Drug: Tivozanib, Drug: Durvalumab
Hepatocellular Carcinoma, Liver
UT Southwestern; Parkland Health & Hospital System
Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840
The main objective of the study is to evaluate the change in liver copper (Cu) concentration
following 48 weeks of treatment with ALXN1840 in adult participants with Wilson Disease (WD)
who have been previously treated for at least 1 year with standard of care (that is,
trientine, penicillamine, or zinc). In the Treatment Period, efficacy and safety of ALXN1840
will be assessed at Week 48.
Call 214-648-5005 studyfinder@utsouthwestern.edu
William Lee
14217
All
18 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT04422431
STU-2020-0703
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:
1. Diagnosis of WD by Leipzig Criteria > 4.
2. Continuous treatment for WD with penicillamine, trientine or zinc for at least 1 year
prior to screening.
3. Body mass index < 30 kilograms/meter squared.
4. Able to cooperate with a percutaneous liver biopsy.
5. Willing and able to follow protocol-specified contraception requirements.
6. Capable of giving signed informed consent.
Exclusion Criteria:
1. Decompensated cirrhosis or Model for End Stage Liver Disease score > 13.
2. Modified Nazer score > 7.
3. Clinically significant gastrointestinal bleed within past 3 months.
4. Alanine aminotransferase > 2 × upper limit of normal.
5. History of bleeding abnormality or known coagulopathy, including platelet count <
100,000, and international normalized ratio for prothrombin time ≥ 1.5.
6. Participant unwilling to accept blood products, if required.
7. Marked neurological disease requiring either nasogastric feeding tube or intensive
inpatient medical care.
8. Hemoglobin less than lower limit of the reference range for age and sex.
9. Participants in renal failure, defined as in end-stage renal disease on dialysis
(chronic kidney disease 5) or creatinine clearance < 30 milliliters/minute.
10. Lymphoma, leukemia, or any malignancy within the past 5 years.
11. Current or chronic history of liver disease not associated with WD.
Comparing the Outcome of Immunotherapy-Based Drug Combination Therapy With or Without Surgery to Remove the Kidney in Metastatic Kidney Cancer, the PROBE Trial (PROBE)
This phase III trial compares the effect of adding surgery to a standard of care
immunotherapy-based drug combination versus a standard of care immunotherapy-based drug
combination alone in treating patients with kidney cancer that has spread to other places in
the body (metastatic). Immunotherapy with monoclonal antibodies, such as nivolumab,
ipilimumab, pembrolizumab, and avelumab, may help the body's immune system attack the cancer,
and may interfere with the ability of tumor cells to grow and spread. Axitinib may stop the
growth of tumor cells by blocking some of the enzymes needed for cell growth. Surgery to
remove the kidney, called a nephrectomy, is also considered standard of care; however,
doctors who treat kidney cancer do not agree on its benefits. It is not yet known if the
addition of surgery to an immunotherapy-based drug combination works better than an
immunotherapy-based drug combination alone in treating patients with kidney cancer.
• STEP 1 REGISTRATION: Participants must have a histologically proven diagnosis of clear
cell or non-clear cell renal cell carcinoma. Participants with collecting duct
carcinoma histology are not eligible. Participants with multifocal or bilateral tumors
are eligible
• STEP 1 REGISTRATION: Participants must have primary tumor in place
• STEP 1 REGISTRATION: Participants must have the following scans performed, showing
clinical evidence of measurable or non-measurable metastatic disease:
• Computed tomography (CT) scan of the chest (can be performed without contrast if
CT contrast cannot be given)
• CT of abdomen and pelvis with contrast OR magnetic resonance imaging (MRI) of the
abdomen and pelvis with or without contrast
Scans must be performed within the following timeframes:
• Treatment naive participants must have scans documenting metastatic disease completed
within 90 days prior to study registration
• Previously treated participants must have scans documenting metastatic disease
completed within 90 days prior to first dose of systemic treatment
• STEP 1 REGISTRATION: Participants with symptomatic metastases may have received
palliative radiotherapy or receive palliative radiotherapy after registration
• STEP 1 REGISTRATION: Participants must have no clear contraindications to
nephrectomy
• STEP 1 REGISTRATION: Participants must be offered the opportunity to participate
in specimen bank. With participant consent, specimens must be collected and
submitted via the Southwest Oncology Group (SWOG) Specimen Tracking System
• STEP 1 REGISTRATION: Participants must be informed of the investigational nature
of this study and must sign and give informed consent in accordance with
institutional and federal guidelines
• STEP 1 REGISTRATION: As part of the Oncology Patient Enrollment Network (OPEN)
registration process the treating institution's identity is provided in order to
ensure that the current (within 365 days) date of institutional review board
approval for this study has been entered in the system
• STEP 2 REGISTRATION: Participants must have at least one of the following scans
performed 12 weeks (+/- 2 weeks) after starting pre-randomization treatment
• CT scan of the chest (can be performed without contrast if CT contrast cannot be
given)
• CT of abdomen and pelvis with contrast OR MRI of the abdomen and pelvis with or
without contrast Scans must be performed within 28 days prior to randomization.
Response should be assessed by comparing with a CT or MRI of the chest, abdomen and
pelvis obtained prior to starting pre-randomization treatment. Participants with
complete response in all metastatic sites are not eligible to randomize to Step 2
• STEP 2 REGISTRATION: Participants must have one of the following objective statuses
after 12 weeks of pre-randomization treatment
• Stable disease
• Partial response
• The treating investigator believes the patient is deriving clinical benefit from
systemic therapy AND have Zubrod performance status 0-1
• STEP 2 REGISTRATION: Participants must plan to continue the immune-based therapy
received during pre-randomization treatment
• STEP 2 REGISTRATION: Participants must be randomized on or between the 11th and
14th week of protocol-directed pre-randomization treatment therapy
• STEP 2 REGISTRATION: Participants must have received at least one of the minimum
amounts of immunotherapy:
• 2 infusions of nivolumab + 1 infusion of ipilimumab
• 2 infusions of pembrolizumab
• 2 infusions of avelumab
• STEP 2 REGISTRATION: Participants must have a planned surgery date within 42 days
of randomization
• STEP 2 REGISTRATION: Participants must be a surgical candidate as determined by
study urologist. The urology consult should be done within 42 days prior to
randomization
• STEP 2 REGISTRATION: Participants must have a complete physical examination and
medical history within 28 days prior to randomization
• STEP 2 REGISTRATION: Participants must have a Zubrod performance status of 0-1
within 28 days prior to randomization
• STEP 2 REGISTRATION: Total bilirubin =< institutional upper limit of normal (ULN)
(within 28 days prior to randomization)
• STEP 2 REGISTRATION: Aspartate aminotransferase (AST)/alanine aminotransferase
(ALT) =< 3 x institutional upper limit of normal (ULN) (within 28 days prior to
randomization)
• STEP 2 REGISTRATION: Serum creatinine =< 1.5 x the institutional upper limit of
normal (IULN) OR measured OR calculated creatinine clearance >= 50 mL/min using
the Cockcroft-Gault Formula) (must have been drawn and processed within 28 days
prior to randomization)
Exclusion Criteria:
• STEP 1 REGISTRATION: Participants must not have known active brain metastases.
Participants with previously treated brain metastases are eligible if participant has
no neurologic signs or symptoms suggestive of brain metastasis. Brain imaging studies
are not required. If brain imaging studies are performed, they must be negative for
disease
• STEP 1 REGISTRATION: Participants must not have received the following prior treatment
of metastatic renal cell carcinoma:
• Treatment naive participants must not have received any prior lines of systemic
therapy for metastatic renal cell carcinoma beyond the line intended as part of
protocol therapy
• Previously treated participants must not have received any systemic therapy for
metastatic renal cell carcinoma beyond the one regimen received off protocol as
specified in Step 1 pre-randomization treatment
• STEP 1 REGISTRATION: Participants must not have received more than the following
amounts protocol-directed pre-randomization treatment:
• Treatment naive participants must not have received any pre-randomization
treatment.
• Previously treated participants must not be planning to receive any additional
treatment prior to Step 2 randomization, and must not have received more than the
following amounts of pre-randomization treatment:
• 4 infusions of nivolumab
• 4 infusions of ipilimumab
• 4 infusions of pembrolizumab
• 7 infusions of avelumab
• STEP 1 REGISTRATION: Participants must not have received immunotherapy for any cancer
within the following timeframes:
• Treatment naive participants must not have received any immunotherapy within a
year of registration
• Previously treated participants must not have received any other immunotherapy
within a year of the start of off protocol specified pre-randomization treatment
• STEP 1 REGISTRATION: Participants must not have a solitary kidney and not have a
transplanted kidney
• STEP 1 REGISTRATION: No other prior malignancy is allowed except for the following:
adequately treated basal cell or squamous cell skin cancer, any in situ or T1 cancer,
adequately treated stage I or II cancer from which the participant is currently in
complete remission, or any other cancer from which the participant has been disease
free for at least two years
• STEP 1 REGISTRATION: Participants must not have been previously diagnosed with a
medical condition that makes them ineligible for immune based combination therapy or
nephrectomy
• STEP 2 REGISTRATION: Participants must not show progression in the primary tumor.
Participants who are considered to have pseudo progression are allowed
• STEP 2 REGISTRATION: Participants must not have known active brain metastases.
Participants with previously treated brain metastases are eligible if participant has
no neurologic signs or symptoms suggestive of brain metastasis. Brain imaging studies
are not required. If brain imaging studies are performed, they must be negative for
disease
• STEP 2 REGISTRATION: No other prior malignancy is allowed except for the following:
adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer,
adequately treated stage I or II cancer from which the participant is currently in
complete remission, or any other cancer from which the participant has been disease
free for two years
Procedure: Cytoreductive Nephrectomy, Drug: Active Comparator
Metastatic Renal Cell Carcinoma, Metastatic Clear Cell Renal Cell Carcinoma, Stage IV Renal Cell Cancer AJCC v8, Kidney
Study to Investigate the Efficacy and Safety of Dupilumab in Pediatric Patients With Active Eosinophilic Esophagitis (EoE) (EoE KIDS)
The Primary objective is to demonstrate the efficacy of dupilumab treatment compared with
placebo in pediatric patients with active eosinophilic esophagitis (EoE) based on histologic
improvement meeting validated histologic criteria.
The Secondary objectives are:
- To demonstrate the efficacy of dupilumab compared to placebo in pediatric patients with
active EoE after 16 weeks of treatment as assessed by endoscopic visual measurements of
disease activity using the Eosinophilic Esophagitis-Endoscopic Reference Score
(EoE-EREFS) and histologic abnormalities as measured by the EoE Histology Scoring System
(EoE-HSS)
- To evaluate the safety, tolerability, and immunogenicity of dupilumab treatment for up
to 16 weeks in pediatric patients with active EoE
- To evaluate the effects of dupilumab on transcriptomic signatures associated with EoE
and type 2 inflammation
- To study the effects of dupilumab on the type 2 inflammation gene expression signature
- To evaluate the concentration-time profile of functional dupilumab in serum in this
population
- To assess efficacy of long-term (52 weeks) dupilumab treatment
- To assess safety, tolerability, and immunogenicity of long-term (52 weeks) dupilumab
treatment
- To evaluate the impact of dupilumab treatment on EoE signs and symptoms
Call 214-648-5005 studyfinder@utsouthwestern.edu
Christopher Parrish
168280
All
1 Year to 11 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT04394351
STU-2020-1291
Show full eligibility criteria
Hide eligibility criteria
Key
Inclusion Criteria:
• A documented diagnosis of eosinophilic esophagitis (EoE)
• Baseline endoscopic biopsies with a demonstration on central reading of
intraepithelial eosinophilic infiltration
Key
Exclusion Criteria:
• Body weight <5 kg or ≥60 kg at screening
• Other causes of esophageal eosinophilia
• Active Helicobacter pylori
• History of Crohn's disease, ulcerative colitis, celiac disease, or prior esophageal
surgery
• Any esophageal stricture unable to be passed with a standard, diagnostic, upper
endoscope or any critical esophageal stricture that requires dilation at screening
• Treatment with swallowed topical corticosteroids within 8 weeks prior to baseline
standard of care endoscopy
• History of bleeding disorders or esophageal varices that, in the opinion of the
investigator, would put the patient at undue risk for significant complications from
an endoscopy procedure
• Active parasitic infection or suspected parasitic infection
• Known or suspected immunodeficiency disorder
NOTE: Other protocol defined inclusion/exclusion criteria apply.
Assessing Durvalumab and FLOT Chemotherapy in Resectable Gastric and Gastroesophageal Junction Cancer
This is a Global Study of Neoadjuvant-Adjuvant Durvalumab or Placebo and FLOT Chemotherapy
Followed by Adjuvant Durvalumab or Placebo in Patients with Resectable Gastric and
Gastroesophageal Cancer (GC/GEJC) (MATTERHORN).
• Patients with histologically documented gastric or gastroesophageal junction
adenocarcinoma with resectable disease (Stage II or higher per AJCC 8th edition).
• Patients must undergo radical surgery.
• No prior anti-cancer therapy for the current malignancy.
• World Health Organization (WHO)/ECOG performance status of 0 or 1 at enrollment.
• Adequate organ and marrow function.
• Availability of tumor sample prior to study entry.
• Must have a life expectancy of at least 24 weeks.
Key
Exclusion Criteria:
• Patients with peritoneal dissemination or distant metastasis.
• Patients with adenosquamous cell carcinoma, squamous cell carcinoma, or GI stromal
tumor.
• Current or prior use of immunosuppressive medication within 14 days before the first
dose of durvalumab.
• Contra-indication to any of the study drugs.
• History of allogeneic organ transplantation.
Effects of Hypoglossal Nerve Stimulation on Cognition and Language in Down Syndrome and Obstructive Sleep Apnea
This study is a prospective, single-arm study conducted under a common implant and follow-up
protocol. The objective will be to follow fifty-seven (57) adolescents and young adults
(10-21 years of age), with Down syndrome, moderate to severe sleep apnea, and
post-adenotonsillectomy, for 12 months after undergoing implant of the Inspire Upper Airway
Stimulation (UAS) System. The study is being conducted in order to evaluate objective change
in cognition and expressive language after implant and therapy with the Inspire UAS System.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Ron Mitchell
124198
All
10 Years to 21 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT04801771
STU-2021-0286
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:
• Diagnosis of Down syndrome
• Age 10-21 years
• Prior adenotonsillectomy
• Severe OSA (AHI > 10, AHI < 50, no more than 25% AHI attributable to central events)
based on prior in-lab PSG performed after adenotonsillectomy and within 18 months of
enrollment
• Approval from at least two of the three physician reviewers based upon the results of
a routine drug-induced sleep endoscopy (DISE) having occurred within 12 months of
enrollment
• Subjects must have either tracheotomy or be ineffectively treated with CPAP due to
non-compliance, discomfort, un-desirable side effects, persistent symptoms despite
compliance use, or refusal to use the device
• Children and their parents/guardians must be willing to have stimulation hardware
permanently implanted, and be willing to participate in follow-up visits,
postoperative PSG, and questionnaire completion
• Children's parents/guardians must complete a questionnaire confirming that their child
is capable of communicating feelings of pain or discomfort. They must also confirm
they are able to assess their child for adverse effects related to device implantation
• Children and their parents/guardians must be proficient in English
Exclusion Criteria:
• Body mass index (BMI) above the 95th percentile for subject's age
• Circumferential airway collapse at the level of the velopharynx observed during DISE
• Other medical conditions resulting in medical instability (eg. congestive heart
failure, recent open heart surgery, immunosuppression, or chronic lung disease or
aspiration)
• Presence of another medical condition requiring future magnetic resonance imaging
(MRI) of the chest
• Patients with another implantable device which could interact unintentionally with the
Inspire system
• Any contraindication for general anesthesia
• History of bleeding or clotting disorders and those on blood thinning or NSAID
medications for the week prior to implantation surgery. Subjects will be asked to
refrain from the use of NSAIDS for two weeks after implantation or any revision
surgeries
• Subject is currently taking muscle relaxant medication
• Life expectancy less than 12 months
• Subject's inability to communicate pain or discomfort to their caretaker/parent, based
on parental or investigator assessment
• Nonverbal candidates will be excluded due to an inability to complete testing
procedures including expressive language sampling
• Subjects with a co-occurring diagnosis of autism spectrum disorder
• Subjects that have a positive β-HCG
• Subjects deemed unfit for participation by the investigator for any other reason
Device: Inspire Upper Airway Stimulation (UAS) System
Obstructive Sleep Apnea, Down Syndrome, Ear, Nose, Throat
Apical Suspension Repair for Vault Prolapse In a Three-Arm Randomized Trial Design (ASPIRe)
The study is a multi-center, randomized, surgical trial of women with symptomatic
post-hysterectomy apical (cuff) prolapse desiring surgical treatment. This study will compare
the three available surgical treatments performed in usual practice. The purpose of this
study is to compare two commonly performed mesh apical repair (sacral colpopexy vs. Apical
Transvaginal Mesh) and vaginal native tissue apical repairs with mesh reinforced repairs. The
primary outcome is measured over time (up to 60 months) using a survival analysis approach.
The investigators hypothesize that treatment failure will not differ between vaginally and
abdominally placed mesh for vault vaginal prolapse, and mesh repairs (regardless of route of
implantation) will be superior to native tissue apical suspension.
Call 214-648-5005 studyfinder@utsouthwestern.edu
David Rahn
49553
Female
21 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT02676973
STU 042016-010
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:
1. Women age 21 or older
2. Prior total hysterectomy (no cervix present)
3. Prolapse beyond the hymen (defined as Ba, C, or Bp > 0 cm)
4. Vaginal cuff descent into at least the lower two thirds of the vagina (defined as
point C> -2/3 TVL)
5. Bothersome bulge symptoms as indicated on question 3 of the PFDI-20 form relating to
'sensation of bulging' or 'something falling out'
6. Desires surgical treatment for post-hysterectomy vaginal prolapse
7. Available for up to 60 month follow-up
Exclusion Criteria:
1. Previous synthetic material or biologic grafts (placed vaginally or abdominally) to
augment POP repair including anterior, posterior and/or apical compartments
2. Known previous formal SSLS performed for either uterovaginal or post-hysterectomy
vaginal vault prolapse *
3. Known adverse reaction to synthetic mesh or biological grafts; these complications
include but are not limited to erosion, fistula, or abscess
4. Unresolved chronic pelvic pain-active
5. Prior abdominal or pelvic radiation
6. Contraindication to any of the index surgical procedures
• Known Horseshoe Kidney or Pelvic Mass overlying the sacrum
• Active diverticular abscess or active diverticulitis
• Shortened vaginal length (<6 cm TVL)
• NOTE:
• Only documented SSLS will be an exclusion.
• Mesh used for only mid-urethral sling will NOT be an exclusion
• If prior POP repair is unknown and unable to be documented, subjects
will be eligible based on clinician judgment. The investigator will
examine and assess for evidence of mesh or graft if no evidence of mesh
or graft is present on examination subject remains eligible.
Procedure: Open, Robotic, or Laparoscopic, Procedure: Transvaginal Native Tissue Repair, Procedure: Uphold™ LITE
Open-Label Extension and Safety Study for Participants With Crohn's Disease Previously Enrolled in the Etrolizumab Phase III Study GA29144 (JUNIPER)
This open-label extension and safety monitoring study is composed of two parts: Part 1 will
evaluate the long-term safety and efficacy of continued etrolizumab treatment in participants
with moderately to severely active Crohn's disease who were previously enrolled in the
etrolizumab Phase III Study GA29144 (NCT02394028) and who meet eligibility criteria for
enrollment into Part 1. In Part 2, participants who have stopped etrolizumab treatment
(either by exiting Part 1 of this study or by entering directly from Study GA29144
[NCT02394028]) will be monitored for 92 weeks for progressive multifocal leukoencephalopathy
(PML) and other safety events.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Moheb Boktor
184157
All
18 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT02403323
STU 102017-058
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:
Part 1 Open-Label Extension:
• Patients previously enrolled in etrolizumab Phase III study GA29144 (NCT02394028) who
meet the eligibility criteria for open-label etrolizumab as described in the protocol
Part 2 Safety Monitoring:
• Patients who participated in etrolizumab Phase III study GA29144 (NCT02394028) and are
not eligible or choose not to enter Part 1
• Patients who transfer from Part 1
• Completion of the 12-week safety follow-up period prior to entering
Exclusion Criteria:
Part 1 Open-Label Extension:
• Any new, significant, uncontrolled condition
Part 2 Safety Monitoring:
• No exclusion criteria
Drug: Etrolizumab
Crohn Disease, Colon, Small Intestine
UT Southwestern; Parkland Health & Hospital System
PREA, PK And Safety PASS Study Of IV Pantoprazole In Pediatric Subjects
The purpose of this study is to characterize the pharmacokinetics (PK) and safety of
intravenous (IV) pantoprazole in patients 1 to 16 years old who are candidates for acid
suppression therapy.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Aakash Goyal
117056
All
1 Year to 16 Years old
Phase 4
This study is NOT accepting healthy volunteers
NCT02401035
STU 032017-112
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:
• Subjects aged 1 to 16 years who in the judgment of the investigator are candidates for
gastric acid suppression therapy (ie, those with a presumptive diagnosis of GERD, a
clinical diagnosis of suspected GERD, symptomatic GERD, or endoscopically proven GERD)
and whom the investigator judges would need to receive IV PPI therapy for at least 4
days.
• Body weight > 5th percentile for age.
• Y-site or dedicated IV line for administration of pantoprazole sodium.
• Expected survival for at least 30 days.
• Fertile male and female subjects of childbearing potential at risk for pregnancy must
agree to use a highly effective method of contraception throughout the study and for
at least 28 days after the last dose of assigned treatment. Female subjects of
non-childbearing potential must be premenarchal, have undergone hysterectomy with
bilateral oophorectomy, have medically confirmed ovarian failure, or achieved
post-menopausal status.
Exclusion Criteria:
• Participation in other studies involving investigational drug(s) or treatment with an
investigational drug within 30 days or 5 half lives prior to study entry and/or during
study participation.
• Other severe acute or chronic medical or psychiatric condition or laboratory
abnormality that may increase the risk associated with study participation or
investigational product administration or may interfere with the interpretation of
study results and, in the judgment of the investigator, would make the subject
inappropriate for entry into this study.
• Pregnant females; breastfeeding females; fertile male subjects, and female subjects of
childbearing potential who are unwilling or unable to use a highly effective method of
contraception for the duration of the study and for at least 28 days after last dose
of investigational product.
• Serum CK levels >3x ULN.
• Known history of HIV or clinical manifestations of AIDS.
• Known hypersensitivity to PPIs or to any substituted benzimidazole or to any of the
excipients.
• History of treatment with any PPI within 2 days (48 hours) before investigational
product dosing on Day 1.
• Use of H2RAs, sucralfate, misoprostol, or prokinetic agents, and bismuth preparations
within 1 day (24 hours) before investigational product dosing on Day 1.
• Any disorder requiring chronic (every day) use of warfarin, carbamazepine, or
phenytoin, methotrexate, atazanavir or nelfinavir, clopidogrel, and potent inhibitors
and inducers of CYP2C19.
• Chronic (daily) use of glucocorticoids. Steroid inhalers and topical steroids may be
used.
• Active malignancy of any type, or history of a malignancy (Subject with a history of
malignancies that have been surgically removed or eradicated by irradiation or
chemotherapy and who have no evidence of recurrence for at least 5 years before
Screening are acceptable).
• ALT or BUN >2.0 ULN or estimated creatinine >1.5 X ULN for age or any other laboratory
abnormality considered by the Investigator to be clinically significant within 14 days
before Screening.
• In the Investigator's opinion, a chronic condition (eg, diabetes, epilepsy), which is
either not stable or well controlled and may interfere with the conduct of the study.
• History of sensitivity to heparin or heparin induced thrombocytopenia.
Drug: IV pantoprazole
Gastroesophageal Reflux Disease
candidate for acid suppression therapy, presumptive diagnosis of GERD, clinical diagnosis of suspected GERD, symptomatic GERD, endoscopically proven GERD