Clinical trial NCT06619561: A Study to Evaluate Vimseltinib in Adults With Active Chronic Graft-Versus-Host Disease (cGVHD)
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Chronic Graft-Versus-Host Disease (cGVHD)
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
Lead sponsor: Deciphera Pharmaceuticals, LLC
Sponsor class: INDUSTRY
First posted: 2024-10-01
Enrollment: 36
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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 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.
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.

Primary outcomes:
measure: Number of Participants with Dose-Limiting Toxicities (DLTs)
timeFrame: Cycle 1 (28 Days)
measure: Number of Participants with Adverse Event(s) (AEs) and Serious Adverse Event(s) (SAEs)
timeFrame: Baseline to Study Completion (Estimated up to 24 months)
Secondary outcomes:
measure: Objective Response Rate (ORR)
timeFrame: Baseline up to Cycle 7 Day 1 (Cycle = 28 days)
measure: Duration of Response (DOR)
timeFrame: First CR or PR until PD or Death due to Any Cause (Estimated up to 24 months)
measure: Organ-Specific Response
timeFrame: Baseline up to Cycle 7 Day 1 (Cycle = 28 days)
measure: Failure-Free Survival (FFS)
timeFrame: Baseline to, whichever occurs first of, PD, Addition of Systemic Immune Suppressive Therapy, or Death due to Any Cause (Estimated up to 24 months)
measure: Pharmacokinetics (PK): Maximum Observed Plasma Concentration (Cmax)
timeFrame: Estimated up to 24 months
Other outcomes:
Locations:
city: Duarte
country: United States
facility: City of Hope National Medical Center
state: California
city: Los Angeles
country: United States
facility: Ronald Regan UCLA Medical Center
state: California
city: Orlando
country: United States
facility: AdventHealth Orlando
state: Florida
city: Tampa
country: United States
facility: Moffitt Cancer Center
state: Florida
city: Westwood
country: United States
facility: University of Kansas Cancer Center-Westwood
state: Kansas
city: Lexington
country: United States
facility: University of Kentucky Markey Cancer Center
state: Kentucky
city: Saint Louis
country: United States
facility: Washington University School of Medicine - Siteman Cancer Center
state: Missouri
city: Cincinnati
country: United States
facility: Oncology Hematology Care Clinical Trials, LLC
state: Ohio
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Sioux Falls
country: United States
facility: Avera Cancer Institute
state: South Dakota
city: Nashville
country: United States
facility: Tristar Bone Marrow Transplant
state: Tennessee
city: Austin
country: United States
facility: St. David's South Austin Medical Center
state: Texas
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
Study officials:
affiliation: Deciphera Pharmaceuticals, LLC
name: Clinical Team
role: STUDY_DIRECTOR
References: