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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Vimseltinib | Experimental | Escalating doses of vimseltinib in 28 day cycles. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Vimseltinib | Drug | Administered orally |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants with Dose-Limiting Toxicities (DLTs) | DLTs assessed for each dose level. | 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. | Baseline to Study Completion (Estimated up to 24 months) |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Baseline up to Cycle 7 Day 1 (Cycle = 28 days) |
| Duration of Response (DOR) |
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Inclusion Criteria:
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).
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.
Exclusion Criteria:
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:
Malabsorption syndrome or other illness that could affect oral absorption.
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Clinical Team | Contact | 888-724-3274 | clinicaltrials@deciphera.com |
| Name | Affiliation | Role |
|---|---|---|
| Clinical Team | Deciphera Pharmaceuticals, LLC | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| City of Hope National Medical Center | Recruiting | Duarte | California | 91010 | United States | |
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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) |
| Organ-Specific Response | Organ-specific response per 2014 NIH cGVHD Criteria up to Cycle 7 Day 1 | 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. | 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 | Estimated up to 24 months |
| Ronald Regan UCLA Medical Center |
| Recruiting |
| Los Angeles |
| California |
| 90095 |
| United States |
| University of California Irvine Health | Recruiting | Orange | California | 92868-3201 | United States |
| AdventHealth Orlando | Recruiting | Orlando | Florida | 32804 | United States |
| Moffitt Cancer Center | Recruiting | Tampa | Florida | 33612 | United States |
| Emory University Winship Cancer Institute | Recruiting | Atlanta | Georgia | 30322 | United States |
| University of Illinois Medical Center - Hematology & Oncology | Recruiting | Chicago | Illinois | 60612 | United States |
| University of Kansas Cancer Center-Westwood | Recruiting | Westwood | Kansas | 66205 | United States |
| University of Kentucky Markey Cancer Center | Recruiting | Lexington | Kentucky | 40536 | United States |
| Dana-Farber Cancer Institute | Recruiting | Boston | Massachusetts | 02215 | United States |
| Henry Ford Cancer Institute | Recruiting | Detroit | Michigan | 48202 | United States |
| Washington University School of Medicine - Siteman Cancer Center | Recruiting | St Louis | Missouri | 63108 | United States |
| Levine Cancer Institute | Recruiting | Charlotte | North Carolina | 28204 | United States |
| Duke University Hospital | Recruiting | Durham | North Carolina | 27705 | United States |
| Oncology Hematology Care Clinical Trials, LLC | Recruiting | Cincinnati | Ohio | 45242 | United States |
| Cleveland Clinic | Recruiting | Cleveland | Ohio | 44195 | United States |
| The Ohio State University Comprehensive Cancer Center | Recruiting | Columbus | Ohio | 43210 | United States |
| Oregon Health and Science University | Recruiting | Portland | Oregon | 97239 | United States |
| UPMC Hillman Cancer Center | Recruiting | Pittsburgh | Pennsylvania | 15232 | United States |
| Avera Cancer Institute | Recruiting | Sioux Falls | South Dakota | 57105 | United States |
| Tristar Bone Marrow Transplant | Recruiting | Nashville | Tennessee | 37203 | United States |
| Vanderbilt-Ingram Cancer Center | Recruiting | Nashville | Tennessee | 37232 | United States |
| St. David's South Austin Medical Center | Recruiting | Austin | Texas | 78704 | United States |
| UT Southwestern Medical Center | Recruiting | Dallas | Texas | 75390 | United States |
| Intermountain Health | Recruiting | Salt Lake City | Utah | 84143 | United States |
| Virginia Commonwealth University | Recruiting | Richmond | Virginia | 23298 | United States |
| ID | Term |
|---|---|
| D000092122 | Bronchiolitis Obliterans Syndrome |
| D006086 | Graft vs Host Disease |
| D007154 | Immune System Diseases |
| D000092124 | Organizing Pneumonia |
| D001989 | Bronchiolitis Obliterans |
| D001988 | Bronchiolitis |
| D001982 | Bronchial Diseases |
| D012140 | Respiratory Tract Diseases |
| D008173 | Lung Diseases, Obstructive |
| D008171 | Lung Diseases |
| D012598 | Sclerosis |
| D005355 | Fibrosis |
| D008107 | Liver Diseases |
| ID | Term |
|---|---|
| D001991 | Bronchitis |
| D012141 | Respiratory Tract Infections |
| D007239 | Infections |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D004066 | Digestive System Diseases |
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