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11 Mastocytosis Trials

Power is an online platform that helps thousands of Mastocytosis patients discover FDA-reviewed trials every day. Every trial we feature meets safety and ethical standards, giving patients an easy way to discover promising new treatments in the research stage.

Systemic mastocytosis with an associated hematologic neoplasm (SM-AHN) is a challenging disease to treat. Targeted KIT inhibitors have been approved for this indication based on their ability to control the mastocytosis portion of the disease, but patients frequently experience progression of the concomitant myeloid malignancy (i.e. the AHN). Using a combination approach to treat both aspects of the disease has the potential to provide enhanced disease control; however, overlapping toxicity is a concern. In this study, investigators aim to study the safety and tolerability of combined avapritinib and decitabine for the treatment of SM-AHN.
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Age:18+
Sex:All
34 Participants Needed
The goal of this clinical trial is to evaluate elenestinib (BLU-263) in participants with Advanced Systemic Mastocytosis (AdvSM), SM with an associated hematologic neoplasm (SM-AHN), and other hematologic malignancies. The main questions it aims to answer are: * Determine Recommended Dose of elenestinib (BLU-263) monotherapy for participants with AdvSM * Safety and tolerability of elenestinib (BLU-263) monotherapy * Efficacy of elenestinib (BLU-263) monotherapy in participants with AdvSM * Determine Recommended Dose of elenestinib (BLU-263) in combination with azacitidine in participants with AdvSM * Safety and tolerability of elenestinib (BLU-263) in combination with azacitidine * Efficacy of elenestinib (BLU-263) in combination with azacitidine in participants with AdvSM The estimated study duration for each participant will be approximately 4 years: 2 years of treatment followed by 2 years of follow-up. Participants may be required to attend monthly visits for the first six months, followed by quarterly visits for the remainder of the study.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1
Age:18+
Sex:All
67 Participants Needed
Image of trial facility.

MGD024 for Blood Cancers

Grand Rapids, Michigan
This trial is testing a new drug called MGD024 in patients with certain blood cancers that haven't responded to other treatments. Researchers want to see if MGD024 is safe, how it works in the body, and if it helps fight cancer. Patients will receive the drug periodically, and their response will be monitored regularly.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Age:18+
Sex:All
130 Participants Needed
This trial is testing a new drug called bezuclastinib, given with usual care, in patients with nonadvanced systemic mastocytosis whose symptoms are not well-managed. The drug aims to reduce the activity of cells that cause symptoms, potentially leading to better symptom control.
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:18+
Sex:All
207 Participants Needed
This trial tests BLU-263 combined with the best possible care for patients with specific conditions whose symptoms are not well-controlled. The medication aims to reduce symptoms by blocking overactive cells.
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2, 3
Age:16+
Sex:All
463 Participants Needed
This is an open-label, two-part Phase 2 study investigating CGT9486 for the treatment of patients with Advanced Systemic Mastocytosis (AdvSM), including patients with Aggressive SM (ASM), SM with Associated Hematologic Neoplasm (SM-AHN), and Mast Cell Leukemia (MCL).
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:18+
Sex:All
140 Participants Needed
This phase I trial studies the best dose and side effects of flotetuzumab for the treatment of patients with blood cancers (hematological malignancies) that have spread to other places in the body (advanced) and have come back after a period of improvement (relapsed) or does not respond to treatment (refractory). Flotetuzumab is a monoclonal antibody that may interfere with the ability of cancer cells to grow and spread.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1
Age:12+
Sex:All
13 Participants Needed
This trial tests TL-895, a strong oral medication that targets specific proteins to treat Myelofibrosis and Indolent Systemic Mastocytosis. It is for patients who haven't responded to other treatments or can't take certain therapies.
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:18+
Sex:All
121 Participants Needed
The purpose of this study is to evaluate the efficacy and safety of oral masitinib versus placebo in the treatment of patients suffering from smouldering or indolent systemic mastocytosis with severe symptoms of mast cell mediator release, unresponsive to optimal symptomatic treatment.
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:18 - 75
Sex:All
140 Participants Needed
This trial is testing a new medication called avapritinib combined with the best possible care for patients with a condition called indolent systemic mastocytosis (ISM). These patients have symptoms that are not well managed by their current treatments. Avapritinib works by blocking certain proteins that cause abnormal cell growth and activity, aiming to improve symptom control.
Prior Safety Data
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:18+
Sex:All
251 Participants Needed
This is an open-label, single arm, Phase 2 study evaluating the efficacy and safety of avapritinib (BLU-285) in patients with advanced systemic mastocytosis (AdvSM), including patients with aggressive SM (ASM), SM with associated hematologic neoplasm (SM-AHN), and mast cell leukemia (MCL)
No Placebo Group
Prior Safety Data
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:18+
Sex:All
103 Participants Needed
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Frequently Asked Questions

How much do Mastocytosis clinical trials pay?

Each trial will compensate patients a different amount, but $50-100 for each visit is a fairly common range for Phase 2–4 trials (Phase 1 trials often pay substantially more). Further, most trials will cover the costs of a travel to-and-from the clinic.

How do Mastocytosis clinical trials work?

After a researcher reviews your profile, they may choose to invite you in to a screening appointment, where they'll determine if you meet 100% of the eligibility requirements. If you do, you'll be sorted into one of the treatment groups, and receive your study drug. For some trials, there is a chance you'll receive a placebo. Across Mastocytosis trials 30% of clinical trials have a placebo. Typically, you'll be required to check-in with the clinic every month or so. The average trial length for Mastocytosis is 12 months.

How do I participate in a study as a "healthy volunteer"?

Not all studies recruit healthy volunteers: usually, Phase 1 studies do. Participating as a healthy volunteer means you will go to a research facility several times over a few days or weeks to receive a dose of either the test treatment or a "placebo," which is a harmless substance that helps researchers compare results. You will have routine tests during these visits, and you'll be compensated for your time and travel, with the number of appointments and details varying by study.

What does the "phase" of a clinical trial mean?

The phase of a trial reveals what stage the drug is in to get approval for a specific condition. Phase 1 trials are the trials to collect safety data in humans. Phase 2 trials are those where the drug has some data showing safety in humans, but where further human data is needed on drug effectiveness. Phase 3 trials are in the final step before approval. The drug already has data showing both safety and effectiveness. As a general rule, Phase 3 trials are more promising than Phase 2, and Phase 2 trials are more promising than phase 1.

Do I need to be insured to participate in a Mastocytosis medical study ?

Clinical trials are almost always free to participants, and so do not require insurance. The only exception here are trials focused on cancer, because only a small part of the typical treatment plan is actually experimental. For these cancer trials, participants typically need insurance to cover all the non-experimental components.

What are the newest Mastocytosis clinical trials ?

Most recently, we added Avapritinib + Decitabine for Systemic Mastocytosis, Elenestinib for Blood Cancers and MGD024 for Blood Cancers to the Power online platform.