A Study to Evaluate Vimseltinib in Adults With Active Chronic Graft-Versus-Host Disease (cGVHD)
- Registration Number
- NCT06619561
- Lead Sponsor
- Deciphera Pharmaceuticals, LLC
- Brief Summary
The purpose of this study is to determine if vimseltinib is safe, tolerable and works effectively to treat adults with active moderate to severe cGVHD. Participants will be treated with vimseltinib in 28-day treatment cycles for approximately 2 years.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 36
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Must be allogeneic hematopoietic stem cell transplant (HSCT) recipients with moderate to severe cGVHD requiring systemic immune suppression.
a. May have persistent active GVHD (aGVHD) and chronic GVHD (cGVHD) manifestations (overlap syndrome).
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Participants with active cGVHD who have received and failed at least 2 prior lines of systemic therapy.
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Stable dose of systemic corticosteroids is permitted but not required. If being taken, participants should be on a stable dose of corticosteroids for at least 2 weeks prior to starting study drug treatment.
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Adequate organ and bone marrow functions.
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Participants of reproductive potential agree to follow the contraception requirements.
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Karnofsky Performance Scale (KPS) of ≥60.
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Has aGVHD without manifestations of cGVHD.
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Prior use of colony-stimulating factor 1 receptor (CSF1R) inhibitor for cGVHD.
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History or other evidence of severe illness, uncontrolled infection, or any other conditions that would make the participant unsuitable for the study.
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History of malignancy except for:
- Underlying malignancy for which the transplant was performed
- Malignancy treated with curative intent and with no evidence of active disease present for more than 3 years prior to enrollment and felt to be at low risk for recurrence.
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Malabsorption syndrome or other illness that could affect oral absorption.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Vimseltinib Vimseltinib Escalating doses of vimseltinib in 28 day cycles.
- Primary Outcome Measures
Name Time Method Number of Participants with Dose-Limiting Toxicities (DLTs) Cycle 1 (28 Days) DLTs assessed for each dose level.
Number of Participants with Adverse Event(s) (AEs) and Serious Adverse Event(s) (SAEs) Baseline to Study Completion (Estimated up to 24 months) AEs and SAEs assessed for each dose level.
- Secondary Outcome Measures
Name Time Method Objective Response Rate (ORR) Baseline up to Cycle 7 Day 1 (Cycle = 28 days) ORR is percentage of participants achieving best overall response of complete response (CR) or partial response (PR) up to Cycle 7 Day 1 based on 2014 National Institutes of Health (NIH) cGVHD Criteria.
Duration of Response (DOR) First CR or PR until PD or Death due to Any Cause (Estimated up to 24 months) DOR for participants with CR or PR, defined as the time interval from the time of first CR or PR per 2014 NIH cGVHD Criteria, until PD or death due to any cause.
Organ-Specific Response Baseline up to Cycle 7 Day 1 (Cycle = 28 days) Organ-specific response per 2014 NIH cGVHD Criteria up to Cycle 7 Day 1
Failure-Free Survival (FFS) Baseline to, whichever occurs first of, PD, Addition of Systemic Immune Suppressive Therapy, or Death due to Any Cause (Estimated up to 24 months) FFS is the time from first dose to progressive disease (PD) per 2014 NIH cGVHD Criteria, addition of another systemic immune suppressive therapy, or death due to any cause, whichever occurs first.
Pharmacokinetics (PK): Maximum Observed Plasma Concentration (Cmax) Estimated up to 24 months Cmax
Trial Locations
- Locations (12)
City of Hope National Medical Center
🇺🇸Duarte, California, United States
Ronald Regan UCLA Medical Center
🇺🇸Los Angeles, California, United States
AdventHealth Orlando
🇺🇸Orlando, Florida, United States
Moffitt Cancer Center
🇺🇸Tampa, Florida, United States
Washington University School of Medicine - Siteman Cancer Center
🇺🇸Saint Louis, Missouri, United States
Oncology Hematology Care Clinical Trials, LLC
🇺🇸Cincinnati, Ohio, United States
St. David's South Austin Medical Center
🇺🇸Austin, Texas, United States
University of Kansas Cancer Center-Westwood
🇺🇸Westwood, Kansas, United States
Cleveland Clinic
🇺🇸Cleveland, Ohio, United States
Avera Cancer Institute
🇺🇸Sioux Falls, South Dakota, United States
University of Kentucky Markey Cancer Center
🇺🇸Lexington, Kentucky, United States
Tristar Bone Marrow Transplant
🇺🇸Nashville, Tennessee, United States