Vimseltinib for Chronic Graft-Versus-Host Disease

Recruiting at 25 trial locations

Overseen By Clinical Team

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Deciphera Pharmaceuticals, LLC

Must not be taking: CSF1R inhibitors

Disqualifiers: Severe illness, Uncontrolled infection, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 24 months

48 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests whether vimseltinib can safely and effectively treat chronic graft-versus-host disease (cGVHD), a condition that can occur after a bone marrow transplant when donated cells attack the recipient's body. Participants will take vimseltinib in 28-day cycles for about two years. The trial seeks individuals with moderate to severe cGVHD who have tried at least two other treatments without success and may still need certain medications for their condition. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering a chance to benefit from a potentially effective new therapy.

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.

Is there any evidence suggesting that vimseltinib is likely to be safe for humans?

Research shows that vimseltinib is being tested for safety and tolerability in people with moderate to severe chronic graft-versus-host disease (cGVHD). While specific side effects are not yet available, its progression to a phase 2 trial indicates some safety in earlier tests. Phase 2 trials aim to further assess a treatment's safety and effectiveness. This suggests that participants in initial studies tolerated vimseltinib well enough to proceed with testing. However, as an experimental treatment, unknown risks may still exist. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Vimseltinib is unique because it targets chronic graft-versus-host disease (cGVHD) by inhibiting a specific protein called CSF1R, which plays a crucial role in the survival and function of certain immune cells that contribute to the disease. This approach is different from current treatments like corticosteroids and immunosuppressants, which broadly dampen the immune system and can have significant side effects. By focusing specifically on CSF1R, vimseltinib aims to reduce harmful immune responses while possibly minimizing side effects associated with broader immune suppression. Researchers are excited because this targeted action could offer a more precise and potentially safer treatment option for cGVHD.

What evidence suggests that vimseltinib might be an effective treatment for chronic graft-versus-host disease?

Research shows that vimseltinib is under study as a treatment for chronic graft-versus-host disease (cGVHD), a condition where donor cells attack the recipient's body after a transplant. Although specific information about vimseltinib for cGVHD in humans remains limited, the drug is designed to block a protein potentially involved in the disease. This approach could help reduce symptoms. Earlier studies found that vimseltinib was generally well-tolerated, which is encouraging for those seeking new treatment options. As more research progresses, the effectiveness of the drug will become clearer. ^{1 2 3 4 5}

Who Is on the Research Team?

Clinical Team

Principal Investigator

Deciphera Pharmaceuticals, LLC

Are You a Good Fit for This Trial?

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 agree to follow the required birth control measures.

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

Interventions

• Vimseltinib

Trial Overview 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.

How Is the Trial Designed?

Group I: Vimseltinib Experimental Treatment 1 Intervention

Escalating doses of vimseltinib in 28 day cycles.

Who Is Running the Clinical Trial?

Deciphera Pharmaceuticals, LLC

Lead Sponsor

Trials

19

Recruited

2,100+

Published Research Related to This Trial

In a study of 8 patients with severe sclerotic chronic graft-vs-host disease (scGVHD), dasatinib showed a higher response rate of 60% compared to 25% for imatinib, indicating it may be a more effective treatment option.

Dasatinib was better tolerated and had a lower incidence of serious side effects, making it a safer and steroid-sparing alternative for patients who are refractory to imatinib.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Imatinib and dasatinib as salvage therapy for sclerotic chronic graft-vs-host disease. Sánchez-Ortega, I., Parody, R., Servitje, O., et al.[2019]

The study by Flynn et al. supports the potential of spleen tyrosine kinase (Syk) inhibition as a promising treatment strategy for chronic graft-versus-host disease (cGVHD).

These findings provide a strong rationale for advancing clinical trials to evaluate the efficacy and safety of Syk inhibitors in cGVHD patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Syk and tired of current chronic GVHD therapies. Fowler, DH., Pavletic, SZ.[2021]

In a study of 43 patients with chronic graft-versus-host disease (cGVHD) treated with tyrosine kinase inhibitors (TKIs), overall response rates improved over time, with 58.6% responding by 12 months, and 80% of patients experienced clinical benefits allowing for corticosteroid dose reductions.

Ruxolitinib was found to be as effective as imatinib in treating sclerotic GVHD, with similar overall survival rates of 96%-100% across all TKI treatments, indicating that these drugs are both effective and feasible options for managing cGVHD.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Single-center, Real-world Experience of Chronic GVHD Treatment Using Ibrutinib, Imatinib, and Ruxolitinib and its Treatment Outcomes. Linn, SM., Novitzky-Basso, I., Abduljalil, O., et al.[2023]

Steroid refractory chronic graft-versus-host disease (cGVHD) has limited treatment options, primarily based on phase II trials and retrospective analyses, highlighting the need for more systematic evaluations of second-line therapies.

Current treatment modalities include a variety of agents such as steroids, calcineurin inhibitors, and newer options like tyrosine kinase inhibitors, but the effectiveness of these treatments often relies on a trial-and-error approach, emphasizing the need for valid biomarkers to predict patient responses.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Consensus Conference on Clinical Practice in Chronic GVHD: Second-Line Treatment of Chronic Graft-versus-Host Disease. Wolff, D., Schleuning, M., von Harsdorf, S., et al.[2015]

Imatinib, a tyrosine kinase inhibitor, was used to treat a patient with cutaneous graft versus host disease (GvHD) resistant to steroids, but it led to complications including multiorgan failure due to low cardiac output.

The study highlights the diagnostic challenge of pericardial effusion and suggests that monitoring lactate levels, central venous pressure, venous saturation, and brain natriuretic peptide can aid in early detection of cardiac issues in such patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cardiac tamponade and graft versus host disease: one more reason to remember. Vetrugno, L., Tomasino, S., Battezzi, A., et al.[2022]

Ruxolitinib (Jakafi) has received approval for the treatment of chronic graft-vs-host disease, indicating its efficacy in managing this condition.

The approval of ruxolitinib highlights its role as a kinase inhibitor, which works by targeting specific pathways involved in the immune response, potentially improving patient outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

New Indication for Ruxolitinib. Aschenbrenner, DS.[2023]

In a pilot study of 19 patients with refractory chronic graft-versus-host disease (cGVHD), imatinib showed a high overall response rate of 79% at 6 months, with 7 complete remissions and 8 partial remissions, indicating its efficacy as a treatment option.

Despite some patients experiencing significant side effects leading to discontinuation of the drug, the 18-month overall survival probability was 84%, suggesting that imatinib can be a promising therapy for patients with difficult-to-treat fibrotic cGVHD.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Imatinib for refractory chronic graft-versus-host disease with fibrotic features. Olivieri, A., Locatelli, F., Zecca, M., et al.[2021]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06619561

NCT06619561 | A Study to Evaluate Vimseltinib in Adults ... 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.

2. ashpublications.org ashpublications.org/ashclinicalnews/news/8608/A-Study-to-Evaluate-Vimseltinib-in-Adults-With

A Study to Evaluate Vimseltinib in Adults With Active Chronic ... The purpose of this study is to determine if vimseltinib is safe and tolerable and works effectively to treat adults with moderate to severe active cGVHD.

3. duke-research.dukehealth.org duke-research.dukehealth.org/studies/pro00116796

Deciphera DCC-3014-02-001: Vimseltinib in Adults With ... We are doing this study to find out if an investigational drug called vimseltinib (the study drug) is a safe and effective option for people with active ...

4. clinicaltrials.icts.uci.edu clinicaltrials.icts.uci.edu/trial/NCT06619561

Vimseltinib in Adults With Active Chronic Graft-Versus-Host ... 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.

5. masseycancercenter.org masseycancercenter.org/clinical-trials/details/phase-2-openlabel-randomized-multicenter-study-to-evaluate-the-efficacy-safety-and-tolerability-of-v

Phase 2, Open-Label, Randomized, Multicenter Study to ... Phase 2, Open-Label, Randomized, Multicenter Study to Evaluate the Efficacy, Safety, and Tolerability of Vimseltinib in Participants with Recurrent or ...