Vimseltinib for Chronic Graft-Versus-Host Disease
Recruiting in Palo Alto (17 mi)
+3 other locations
Age: 18+
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 2
Recruiting
Sponsor: Deciphera Pharmaceuticals, LLC
Must not be taking: CSF1R inhibitors
Disqualifiers: Severe illness, Uncontrolled infection, others
No Placebo Group
Prior Safety Data
Trial Summary
What is the purpose of this trial?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.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but you can stay on a stable dose of corticosteroids if needed. It's best to discuss your specific medications with the trial team.
What data supports the effectiveness of the drug Vimseltinib for chronic graft-versus-host disease?
While there is no direct data on Vimseltinib for chronic graft-versus-host disease, similar drugs like imatinib and ruxolitinib, which are also tyrosine kinase inhibitors, have shown promising results in treating this condition. For example, imatinib has been effective in patients with fibrotic chronic graft-versus-host disease, achieving a 79% overall response rate in a study.
12345How is the drug Vimseltinib different from other drugs for chronic graft-versus-host disease?
Vimseltinib is unique because it targets specific pathways involved in chronic graft-versus-host disease, potentially offering a new approach compared to existing treatments like steroids and tyrosine kinase inhibitors, which have varying effectiveness and side effects. This drug may provide an alternative for patients who do not respond well to current therapies.
13567Eligibility Criteria
This trial is for adults with active moderate to severe chronic Graft-Versus-Host Disease (cGVHD) after stem cell transplant. They must have tried at least two other treatments without success, be able to do most activities (KPS ≥60), and may still have some acute GVHD symptoms. Stable use of steroids is okay, and they need good organ/bone marrow function.Inclusion Criteria
I received a stem cell transplant and need treatment for a severe reaction.
I may have ongoing symptoms of both acute and chronic graft-versus-host disease.
I have chronic GVHD and two treatments haven't worked for me.
+4 more
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Treatment
Participants receive escalating doses of vimseltinib in 28-day cycles for approximately 2 years
24 months
Monthly visits for dose escalation and monitoring
Follow-up
Participants are monitored for safety and effectiveness after treatment
4 weeks
Participant Groups
The study tests Vimseltinib's safety and effectiveness in treating cGVHD over approximately 2 years. Participants will undergo treatment cycles lasting 28 days each with the goal of reducing the severity of their condition.
1Treatment groups
Experimental Treatment
Group I: VimseltinibExperimental Treatment1 Intervention
Escalating doses of vimseltinib in 28 day cycles.
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
Cleveland ClinicCleveland, OH
Avera Cancer InstituteSioux Falls, SD
City of Hope National Medical CenterDuarte, CA
Washington University School of Medicine - Siteman Cancer CenterSaint Louis, MO
Loading ...
Who Is Running the Clinical Trial?
Deciphera Pharmaceuticals, LLCLead Sponsor
References
Imatinib and dasatinib as salvage therapy for sclerotic chronic graft-vs-host disease. [2019]To assess the toxicity, tolerance, steroid-sparing capacity, effectiveness, and response rate to imatinib and dasatinib for the treatment of severe sclerotic chronic graft-vs-host disease (scGVHD).
New Indication for Ruxolitinib. [2023]The kinase inhibitor ruxolitinib (Jakafi) is now approved to treat chronic graft-vs-host disease.
Syk and tired of current chronic GVHD therapies. [2021]In this issue of Blood, Flynn et al1 provide key data that lend further support to the development of clinical trials of spleen tyrosine kinase (Syk) inhibition for more effective chronic graft-versus-host disease (cGVHD) treatment.
A Single-center, Real-world Experience of Chronic GVHD Treatment Using Ibrutinib, Imatinib, and Ruxolitinib and its Treatment Outcomes. [2023]Chronic graft-versus-host disease (cGVHD) is a common cause of morbidity and mortality following allogeneic hematopoietic stem cell transplantation. Tyrosine kinase inhibitors (TKIs), including ruxolitinib, imatinib, and ibrutinib, have shown promising efficacy in cGVHD treatment.
Imatinib for refractory chronic graft-versus-host disease with fibrotic features. [2021]We previously reported that patients with fibrotic, chronic graft-versus-host disease (cGVHD) have antibodies activating the platelet-derived growth factor receptor pathway. Because this pathway can be inhibited by imatinib, we performed a pilot study including 19 patients with refractory cGVHD, given imatinib at a starting dose of 100 mg per day. All patients had active cGVHD with measurable involvement of skin or other districts and had previously failed at least 2 treatment lines. Patient median age was 29 years (range, 10-62 years), and median duration of cGvHD was 37 months (range, 4-107 months). The organs involved were skin (n = 17), lung (n = 11), and bowel (n = 5); 15 patients had sicca syndrome. Imatinib-related, grade 3 to 4 toxicity included fluid retention, infections, and anemia. Imatinib was discontinued in 8 patients: in 3 because of toxicity and in 5 because of lack of response (n = 3) or relapse of malignancy (n = 2). Overall response rate at 6 months was 79%, with 7 complete remissions (CRs) and 8 partial remissions (PRs). With a median follow-up of 17 months, 16 patients are alive, 14 still in CR or PR. The 18-month probability of overall survival is 84%. This study suggests that imatinib is a promising treatment for patients with refractory fibrotic cGVHD.
Consensus Conference on Clinical Practice in Chronic GVHD: Second-Line Treatment of Chronic Graft-versus-Host Disease. [2015]Steroid refractory chronic graft-versus-host disease (cGVHD) is associated with a significant morbidity and mortality. Although first-line treatment of cGVHD is based on controlled trials, second-line treatment is almost solely based on phase II trials or retrospective analyses. The consensus conference on clinical practice in cGVHD held in Regensburg aimed to achieve a consensus on the current evidence of treatment options as well as to provide guidelines for daily clinical practice. Treatment modalities are the use of steroids and calcineurin inhibitors as well as immunomodulating modalities (photopheresis, mTOR-inhibitors, thalidomide, hydroxychloroquine, vitamin A analogs, clofazimine), and cytostatic agents (mycophenolate mofetil, methotrexate, cyclophosphamide, pentostatin). Recent reports showed some efficacy of rituximab, alemtuzumab, and etanercept in selected patients. Moreover, tyrosine kinase inihibitors such as imatinib came into the field because of their ability to interfere with the platelet-derived growth factor (PDGF-R) pathway involved in fibrosis. An other treatment option is low-dose thoracoabdominal irradiation. Although different treatment options are available, the "trial-and-error system" remains the only way to identify the drug effective in the individual patient, and valid biomarkers are eagerly needed to identify the likelihood of response to a drug in advance. Moreover, the sparse evidence for most treatment entities indicates the urgent need for systematic evaluation of second-line treatment options in cGVHD.
Cardiac tamponade and graft versus host disease: one more reason to remember. [2022]In patients with cutaneous graft versus host disease (GvHD) that is resistant to traditional steroid therapy, imatinib is a first-generation tyrosine kinase inhibitor that seems to be a viable option. However, its antifibrotic activity can be associated with serosal inflammation and fluid retention.