A Study to Evaluate Vimseltinib in Adults With Active Chronic Graft-Versus-Host Disease (cGVHD)

December 12, 2025 updated by: Deciphera Pharmaceuticals, LLC

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

48

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • California
      • Duarte, California, United States, 91010
        • Recruiting
        • City of Hope National Medical Center
      • Los Angeles, California, United States, 90095
        • Recruiting
        • Ronald Regan UCLA Medical Center
      • Orange, California, United States, 92868-3201
        • Recruiting
        • University of California Irvine Health
    • Florida
      • Orlando, Florida, United States, 32804
        • Recruiting
        • AdventHealth Orlando
      • Tampa, Florida, United States, 33612
        • Recruiting
        • Moffitt Cancer Center
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Recruiting
        • Emory University Winship Cancer Institute
    • Illinois
      • Chicago, Illinois, United States, 60612
        • Recruiting
        • University of Illinois Medical Center - Hematology & Oncology
    • Kansas
      • Westwood, Kansas, United States, 66205
        • Recruiting
        • University of Kansas Cancer Center-Westwood
    • Kentucky
      • Lexington, Kentucky, United States, 40536
        • Recruiting
        • University of Kentucky Markey Cancer Center
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
        • Recruiting
        • Dana-Farber Cancer Institute
    • Michigan
      • Detroit, Michigan, United States, 48202
        • Recruiting
        • Henry Ford Cancer Institute
    • Missouri
      • St Louis, Missouri, United States, 63108
        • Recruiting
        • Washington University School of Medicine - Siteman Cancer Center
    • North Carolina
      • Charlotte, North Carolina, United States, 28204
        • Recruiting
        • Levine Cancer Institute
      • Durham, North Carolina, United States, 27705
        • Recruiting
        • Duke University Hospital
    • Ohio
      • Cincinnati, Ohio, United States, 45242
        • Recruiting
        • Oncology Hematology Care Clinical Trials, Llc
      • Cleveland, Ohio, United States, 44195
        • Recruiting
        • Cleveland Clinic
      • Columbus, Ohio, United States, 43210
        • Recruiting
        • The Ohio State University Comprehensive Cancer Center
    • Oregon
      • Portland, Oregon, United States, 97239
        • Recruiting
        • Oregon Health and Science University
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15232
        • Recruiting
        • UPMC Hillman Cancer Center
    • South Dakota
      • Sioux Falls, South Dakota, United States, 57105
        • Recruiting
        • Avera Cancer Institute
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Recruiting
        • Vanderbilt-Ingram Cancer Center
      • Nashville, Tennessee, United States, 37203
        • Recruiting
        • TriStar Bone Marrow Transplant
    • Texas
      • Austin, Texas, United States, 78704
        • Recruiting
        • St. David's South Austin Medical Center
      • Dallas, Texas, United States, 75390
        • Recruiting
        • UT Southwestern Medical Center
    • Utah
      • Salt Lake City, Utah, United States, 84143
        • Recruiting
        • Intermountain Health
    • Virginia
      • Richmond, Virginia, United States, 23298
        • Recruiting
        • Virginia Commonwealth University

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. 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).

  2. Participants with active cGVHD who have received and failed at least 2 prior lines of systemic therapy.
  3. 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.
  4. Adequate organ and bone marrow functions.
  5. Participants of reproductive potential agree to follow the contraception requirements.
  6. Karnofsky Performance Scale (KPS) of ≥60.

Exclusion Criteria:

  1. Has aGVHD without manifestations of cGVHD.
  2. Prior use of colony-stimulating factor 1 receptor (CSF1R) inhibitor for cGVHD.
  3. 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.

  4. History of malignancy except for:

    1. Underlying malignancy for which the transplant was performed
    2. 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.
  5. Malabsorption syndrome or other illness that could affect oral absorption.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Vimseltinib
Escalating doses of vimseltinib in 28 day cycles.
Drug: Vimseltinib
Administered orally
Other Names:
  • DCC-3014

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Participants with Dose-Limiting Toxicities (DLTs)
Time Frame: Cycle 1 (28 Days)
DLTs assessed for each dose level.
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)
AEs and SAEs assessed for each dose level.
Baseline to Study Completion (Estimated up to 24 months)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Objective Response Rate (ORR)
Time Frame: 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.
Baseline up to Cycle 7 Day 1 (Cycle = 28 days)
Organ-Specific Response
Time Frame: Baseline up to Cycle 7 Day 1 (Cycle = 28 days)
Organ-specific response per 2014 NIH cGVHD Criteria up to Cycle 7 Day 1
Baseline up to Cycle 7 Day 1 (Cycle = 28 days)
Pharmacokinetics (PK): Maximum Observed Plasma Concentration (Cmax)
Time Frame: Estimated up to 24 months
Cmax
Estimated up to 24 months
Duration of Response (DOR)
Time Frame: 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, initiation of new systemic therapy for cGVHD, or death due to any cause, whichever occurs first.
First CR or PR until PD or Death due to Any Cause (Estimated up to 24 months)
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)
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.
Baseline to, whichever occurs first of, PD, Addition of Systemic Immune Suppressive Therapy, or Death due to Any Cause (Estimated up to 24 months)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Deciphera Pharmaceuticals, LLC

Investigators

  • Study Director: Clinical Team, Deciphera Pharmaceuticals, LLC

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 21, 2024

Primary Completion (Estimated)

October 1, 2029

Study Completion (Estimated)

October 1, 2029

Study Registration Dates

First Submitted

September 27, 2024

First Submitted That Met QC Criteria

September 27, 2024

First Posted (Actual)

October 1, 2024

Study Record Updates

Last Update Posted (Actual)

December 15, 2025

Last Update Submitted That Met QC Criteria

December 12, 2025

Last Verified

December 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • DCC-3014-02-001

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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