Chronic Graft-Versus-Host Disease (cGVHD)Phase 2NCT06619561
A Study to Evaluate Vimseltinib in Adults With Active Chronic Graft-Versus-Host Disease (cGVHD)
A Phase 2 study testing Vimseltinib in people with Chronic Graft-Versus-Host Disease (cGVHD), run by Deciphera Pharmaceuticals, LLC at 26 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Chronic Graft-Versus-Host Disease (cGVHD)
- What is being tested?
- Vimseltinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 26 sitesUnited States
- Study dates
- 21 Nov 2024to Oct 2029
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Must be allogeneic hematopoietic stem cell transplant (HSCT) recipients with moderate to severe cGVHD requiring systemic immune suppression.
- 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.
Who cannot join
- 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.
Trial locations and contacts
26 sites in 1 country. Contact the study team or a site near you to ask about taking part.
26 sites in 1 country
United States26 sites, 26 recruiting
- Recruiting
Emory University Winship Cancer Institute
Atlanta, Georgia, 30322
- Recruiting
St. David's South Austin Medical Center
Austin, Texas, 78704
- Recruiting
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
- Recruiting
Levine Cancer Institute
Charlotte, North Carolina, 28204
- Recruiting
University of Illinois Medical Center - Hematology & Oncology
Chicago, Illinois, 60612
- Recruiting
Oncology Hematology Care Clinical Trials, LLC
Cincinnati, Ohio, 45242
- Recruiting
Cleveland Clinic
Cleveland, Ohio, 44195
- Recruiting
The Ohio State University Comprehensive Cancer Center
Columbus, Ohio, 43210
- Recruiting
UT Southwestern Medical Center
Dallas, Texas, 75390
- Recruiting
Henry Ford Cancer Institute
Detroit, Michigan, 48202
- Recruiting
City of Hope National Medical Center
Duarte, California, 91010
- Recruiting
Duke University Hospital
Durham, North Carolina, 27705
- Recruiting
University of Kentucky Markey Cancer Center
Lexington, Kentucky, 40536
- Recruiting
Ronald Regan UCLA Medical Center
Los Angeles, California, 90095
- Recruiting
Tristar Bone Marrow Transplant
Nashville, Tennessee, 37203
- Recruiting
Vanderbilt-Ingram Cancer Center
Nashville, Tennessee, 37232
- Recruiting
University of California Irvine Health
Orange, California, 92868-3201
- Recruiting
AdventHealth Orlando
Orlando, Florida, 32804
- Recruiting
UPMC Hillman Cancer Center
Pittsburgh, Pennsylvania, 15232
- Recruiting
Oregon Health and Science University
Portland, Oregon, 97239
- Recruiting
Virginia Commonwealth University
Richmond, Virginia, 23298
- Recruiting
Intermountain Health
Salt Lake City, Utah, 84143
- Recruiting
Avera Cancer Institute
Sioux Falls, South Dakota, 57105
- Recruiting
Washington University School of Medicine - Siteman Cancer Center
St Louis, Missouri, 63108
- Recruiting
Moffitt Cancer Center
Tampa, Florida, 33612
- Recruiting
University of Kansas Cancer Center-Westwood
Westwood, Kansas, 66205
About this study
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
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.
- Being tested
- Vimseltinib
- Keywords
- Allogeneic hematopoietic stem cell transplant (HSCT), Graft-Versus-Host Disease, GVHD, cGVHD, Graft versus host disease, Immune System Diseases, Organizing Pneumonia, Bronchiolitis Obliterans, Bronchiolitis, Bronchial Diseases, Respiratory Tract Diseases, Lung Diseases, Obstructive, Lung Diseases, Bronchiolitis Obliterans Syndrome, Graft vs Host Disease, Sclerosis, Fibrosis, Hematopoietic stem cell transplantation, Liver diseases
- Medical subject headings
- Bronchiolitis Obliterans Syndrome, Graft vs Host Disease, Immune System Diseases, Organizing Pneumonia, Bronchiolitis Obliterans, Bronchiolitis, Bronchial Diseases, Respiratory Tract Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VimseltinibDrug
Also called DCC-3014
Administered orally
| Study group | Role | What they receive |
|---|---|---|
| Vimseltinib | Experimental | Escalating doses of vimseltinib in 28 day cycles. Receives: Drug: Vimseltinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Dose-Limiting Toxicities (DLTs)
DLTs assessed for each dose level.
Measured: Cycle 1 (28 Days)
Number of Participants with Adverse Event(s) (AEs) and Serious Adverse Event(s) (SAEs)
AEs and SAEs assessed for each dose level.
Measured: Baseline to Study Completion (Estimated up to 24 months)
Secondary outcomes (5)
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.
Measured: Baseline up to Cycle 7 Day 1 (Cycle = 28 days)
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.
Measured: First CR or PR until PD or Death due to Any Cause (Estimated up to 24 months)
Organ-Specific Response
Organ-specific response per 2014 NIH cGVHD Criteria up to Cycle 7 Day 1
Measured: Baseline up to Cycle 7 Day 1 (Cycle = 28 days)
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.
Measured: 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)
Cmax
Measured: Estimated up to 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Oct 2024
- Study start21 Nov 2024
- Primary completionOct 2029 (estimated)
- Study completionOct 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Graft-Versus-Host Disease (cGVHD)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 48 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Deciphera Pharmaceuticals, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Team, Study director, Deciphera Pharmaceuticals, LLC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06619561
- Sponsor's protocol ID
- DCC-3014-02-001
- Status last verified
- Dec 2025
- First submitted
- 27 Sep 2024
- Last updated
- 15 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No