Reparixin for Myelofibrosis
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores the effectiveness and safety of the drug Reparixin for individuals with myelofibrosis, a condition affecting bone marrow and blood cell production. The study targets those with a specific type of myelofibrosis that has not responded well to other treatments, particularly those involving Janus kinase inhibitors, which block certain enzymes. Participants will take Reparixin in pill form over several cycles to determine its impact on disease management. Suitable candidates have myelofibrosis with persistent symptoms, such as an enlarged spleen, and have not found success with previous treatments. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group of people.
Do I have to stop taking my current medications for the trial?
The trial requires that at least two weeks must have passed since the last dose of any myelofibrosis-directed drug treatments, except for hydroxyurea, before enrolling. This means you may need to stop certain medications before starting the trial.
Is there any evidence suggesting that Reparixin is likely to be safe for humans?
Research has shown that Reparixin is under study for its safety and effectiveness in treating myelofibrosis, a type of blood cancer. Patient studies have tested Reparixin to assess its tolerability and potential side effects. Previous patients demonstrated that the treatment is generally well-tolerated, meaning most people can take the medicine without major issues.
While specific side effects are not detailed in the sources, the fact that Reparixin is in a phase 2 trial suggests it has passed initial safety tests. Earlier studies likely showed it is safe enough for testing in more people. However, like any treatment, some side effects might occur, but these are usually manageable.
For those considering joining this trial, remember that being in a phase 2 trial means researchers are still learning about the treatment, including any possible side effects. Always consult a healthcare provider to understand the risks and benefits before participating.12345Why do researchers think this study treatment might be promising?
Reparixin is unique because it targets the inflammatory pathways involved in myelofibrosis by inhibiting a specific protein called CXCR1/2, which is different from the typical approach of targeting the JAK2 pathway. Most current treatments for myelofibrosis, like ruxolitinib, focus on the JAK2 pathway to manage symptoms and slow disease progression. Researchers are excited about reparixin because it offers a novel mechanism of action that could potentially provide benefits for patients who do not respond well to existing therapies. Additionally, its oral administration makes it a convenient option for patients, potentially improving adherence and quality of life.
What evidence suggests that Reparixin might be an effective treatment for myelofibrosis?
Research has shown that reparixin, the treatment under study in this trial, might help treat myelofibrosis. In studies with mice, reparixin reduced the thickening and stiffening of tissue in the bone marrow and spleen. This thickening, known as fibrosis, can cause health problems. Reparixin blocks certain signals, potentially leading to the death of cancer stem cells and slowing the disease. Although limited information exists from human studies, these early findings suggest that reparixin could be promising for people with myelofibrosis.34678
Who Is on the Research Team?
Marina Kremyanskaya, PhD, MD
Principal Investigator
Icahn School of Medicine at Mount Sinai
Aaron Gerds, MD, MS
Principal Investigator
Cleveland Clinic Taussig Cancer Institute
Are You a Good Fit for This Trial?
This trial is for adults with intermediate-2 or high-risk primary myelofibrosis, post-ET/PV MF who have not responded well to JAK inhibitor treatment or can't take it. Participants should be in good enough health to perform daily activities with minimal assistance (ECOG ≤ 2), have proper organ function, and a life expectancy of at least six months. Pregnant women and those with recent severe heart issues, active serious infections, or other unstable conditions are excluded.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive oral reparixin three times daily on a 4-week cycle for a core study period of 6 cycles (24 weeks)
Extension
Participants may continue receiving reparixin once daily on a 4-week cycle if at least stable disease is met by IWG-MRT criteria
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Reparixin
Find a Clinic Near You
Who Is Running the Clinical Trial?
Icahn School of Medicine at Mount Sinai
Lead Sponsor
Dompé Farmaceutici S.p.A
Industry Sponsor
Novartis
Industry Sponsor
Vasant Narasimhan
Novartis
Chief Executive Officer since 2018
MD from Harvard Medical School, Bachelor's in Biological Sciences from University of Chicago, Master's in Public Policy from John F. Kennedy School of Government
Shreeram Aradhye
Novartis
Chief Medical Officer since 2022
MD from Yale University, MSc in Clinical Epidemiology from University of Pennsylvania