A Study to Evaluate Vimseltinib in Adults With Active Chronic Graft-Versus-Host Disease (cGVHD)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Vimseltinib.
- Who it may be relevant to
- Registry conditions: Chronic Graft-Versus-Host Disease (cGVHD). Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Phase 2, Open-label, Multicenter Study to Evaluate the Safety, Tolerability, PK, and Efficacy of Vimseltinib in Adults With Active Chronic GVHD After Failure of Prior Systemic Therapy
Overview
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.
Interventions
- Drug Vimseltinib
Administered orally
Primary outcome measures
- Number of Participants with Dose-Limiting Toxicities (DLTs) [Time frame: Cycle 1 (28 Days)]
- Number of Participants with Adverse Event(s) (AEs) and Serious Adverse Event(s) (SAEs) [Time frame: Baseline to Study Completion (Estimated up to 24 months)]
Secondary outcome measures (5)
- Objective Response Rate (ORR) [Time frame: Baseline up to Cycle 7 Day 1 (Cycle = 28 days)]
- Duration of Response (DOR) [Time frame: First CR or PR until PD or Death due to Any Cause (Estimated up to 24 months)]
- Organ-Specific Response [Time frame: Baseline up to Cycle 7 Day 1 (Cycle = 28 days)]
- Failure-Free Survival (FFS) [Time frame: Baseline to, whichever occurs first of, PD, Addition of Systemic Immune Suppressive Therapy, or Death due to Any Cause (Estimated up to 24 months)]
- Pharmacokinetics (PK): Maximum Observed Plasma Concentration (Cmax) [Time frame: Estimated up to 24 months]
Eligibility criteria
Inclusion criteria
- Must be allogeneic hematopoietic stem cell transplant (HSCT) recipients with moderate to severe cGVHD requiring systemic immune suppression.
a. May have persistent active acute GVHD (aGVHD) and chronic GVHD (cGVHD) manifestations (overlap syndrome).
- Participants with active cGVHD who have received and failed at least 2 prior lines of systemic therapy.
- 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.
- Adequate organ and bone marrow functions.
- Participants of reproductive potential agree to follow the contraception requirements.
- Karnofsky Performance Scale (KPS) of ≥60.
Exclusion criteria
- Has aGVHD without manifestations of cGVHD.
- Prior use of colony-stimulating factor 1 receptor (CSF1R) inhibitor for cGVHD.
- History or other evidence of severe illness, uncontrolled infection, or any other conditions that would make the participant unsuitable for the study. All wounds must be healed and free of infection or dehiscence.
- 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.
- Malabsorption syndrome or other illness that could affect oral absorption.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 26 centers
- City of Hope National Medical Center — Duarte
- Ronald Regan UCLA Medical Center — Los Angeles
- University of California Irvine Health — Orange
- AdventHealth Orlando — Orlando
- Moffitt Cancer Center — Tampa
- Emory University Winship Cancer Institute — Atlanta
- University of Illinois Medical Center - Hematology & Oncology — Chicago
- University of Kansas Cancer Center-Westwood — Westwood
- … and 18 more centers
Identifiers
NCT: NCT06619561 · DCC-3014-02-001