A Study to Evaluate Vimseltinib in Adults With Active Chronic Graft-Versus-Host Disease (cGVHD)
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorDeciphera Pharmaceuticals, LLC
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Must be allogeneic hematopoietic stem cell transplant (HSCT) recipients with moderate to severe cGVHD requiring systemic immune suppression.
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.
Disqualifiers
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.
Underlying malignancy for which the transplant was performed
Trial design
Design model
Single group
Treatments tested in this trial
Vimseltinib
DrugAdministered orally
Treatment groups
48 Participants
are divided into 1 treatment groupGroup A: VimseltinibExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Number of Participants with Dose-Limiting Toxicities (DLTs)
DLTs assessed for each dose level.
Time frame
Cycle 1 (28 Days)2
Number of Participants with Adverse Event(s) (AEs) and Serious Adverse Event(s) (SAEs)
AEs and SAEs assessed for each dose level.
Time frame
Baseline to Study Completion (Estimated up to 24 months)Secondary outcomes
1
Objective Response Rate (ORR)
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.
Time frame
Baseline up to Cycle 7 Day 1 (Cycle = 28 days)2
Duration of Response (DOR)
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, initiation of new systemic therapy for cGVHD, or death due to any cause, whichever occurs first.
Time frame
First CR or PR until PD or Death due to Any Cause (Estimated up to 24 months)3
Organ-Specific Response
Organ-specific response per 2014 NIH cGVHD Criteria up to Cycle 7 Day 1
Time frame
Baseline up to Cycle 7 Day 1 (Cycle = 28 days)4
Failure-Free Survival (FFS)
FFS is the time from first dose to progressive disease (PD) per 2014 NIH cGVHD Criteria, initiation of new systemic therapy for cGVHD, or death due to any cause, whichever occurs first.
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)Sponsors and contacts
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Source ClinicalTrials.gov