Chronic Graft-Versus-Host Disease (cGVHD)
Conditions
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
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.
Interventions
DRUGVimseltinib
Administered orally
Sponsors
Deciphera Pharmaceuticals, LLC
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants with Dose-Limiting Toxicities (DLTs) | Cycle 1 (28 Days) | DLTs assessed for each dose level. |
| Number of Participants with Adverse Event(s) (AEs) and Serious Adverse Event(s) (SAEs) | Baseline to Study Completion (Estimated up to 24 months) | AEs and SAEs assessed for each dose level. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Organ-Specific Response | Baseline up to Cycle 7 Day 1 (Cycle = 28 days) | Organ-specific response per 2014 NIH cGVHD Criteria up to Cycle 7 Day 1 |
| Objective Response Rate (ORR) | 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. |
| Pharmacokinetics (PK): Maximum Observed Plasma Concentration (Cmax) | Estimated up to 24 months | Cmax |
| Failure-Free Survival (FFS) | 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. |
| Duration of Response (DOR) | 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. |
Countries
United States
Contacts
Primary ContactClinical Team
Outcome results
None listed