(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis
This is a randomized, double-blind, placebo-controlled, Phase 2/3 study comparing the efficacy and safety of elenestinib (BLU-263) + symptom directed therapy (SDT) with placebo + SDT in participants w...
(Apex) Bezuclastinib in Patients With Advanced Systemic Mastocytosis
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 Asso...
Study of TL-895 in Subjects With Myelofibrosis or Indolent Systemic Mastocytosis
This study evaluates TL-895, a potent, orally-available and highly selective irreversible tyrosine kinase inhibitor for the treatment of Myelofibrosis (Cohorts 1-3) or Indolent Systemic Mastocytosis (...
Top Cities for Systemic Mastocytosis Clinical Trials
Systemic Mastocytosis clinical trials are recruiting across 24 cities. Here are the cities with the most active studies:
About Systemic Mastocytosis
Systemic mastocytosis is a rare condition caused by accumulation of abnormal mast cells in the bone marrow, skin, liver, spleen, and GI tract. It can cause allergic reactions, bone pain, and organ damage. Treatment includes targeted therapies, antihistamines, and mast cell stabilizers.
Clinical trials are advancing new treatments for systemic mastocytosis. Currently, 3 studies are recruiting a combined 795 participants across the United States. Research is being conducted by 3 organizations including Blueprint Medicines Corporation, Cogent Biosciences, Inc., Telios Pharma, Inc..
2026 Systemic Mastocytosis Research Landscape
As of March 2026, the systemic mastocytosis clinical trial landscape includes 3 actively recruiting studies across 24 cities in the United States. These studies are collectively seeking 795 participants, with an average enrollment target of 265 per study.
Research is being led by 3 different organizations, including Blueprint Medicines Corporation, Cogent Biosciences, Inc., Telios Pharma, Inc..
Geographically, systemic mastocytosis trials are most concentrated in Birmingham, Alabama (2 trials); Boston, Massachusetts (2 trials); Rochester, Minnesota (2 trials); New York, New York (2 trials); Cincinnati, Ohio (2 trials) and 7 other cities.
Featured Systemic Mastocytosis Studies
Highlighted recruiting studies for systemic mastocytosis, selected by enrollment size and research scope.
(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis
This is a randomized, double-blind, placebo-controlled, Phase 2/3 study comparing the efficacy and safety of elenestinib (BLU-263) + symptom directed therapy (SDT) with placebo + SDT in participants with indolent systemic mastocytosis (ISM) whose symptoms are not adequately controlled by SDT. Parts 1 and 2 will enroll participants with ISM. Participants enrolled in Part 2 will roll over onto Part ...
(Apex) Bezuclastinib in Patients With Advanced Systemic Mastocytosis
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).
Study of TL-895 in Subjects With Myelofibrosis or Indolent Systemic Mastocytosis
This study evaluates TL-895, a potent, orally-available and highly selective irreversible tyrosine kinase inhibitor for the treatment of Myelofibrosis (Cohorts 1-3) or Indolent Systemic Mastocytosis (Cohort 5). Participants must be diagnosed with Myelofibrosis and be relapsed/refractory (e.g., having failed prior therapy), intolerant, or ineligible to receive JAKi treatment, or be diagnosed with I...
Frequently Asked Questions About Systemic Mastocytosis Clinical Trials
Are there systemic mastocytosis clinical trials near me?
Yes, there are 3 systemic mastocytosis clinical trials currently recruiting across 24+ cities in the United States, including Birmingham, Alabama; Boston, Massachusetts; Rochester, Minnesota. Browse the studies above to find one at a location convenient for you.
How do I join a systemic mastocytosis clinical trial?
To join a systemic mastocytosis clinical trial: 1) Browse the available studies on this page, 2) Click on a study that interests you, 3) Check the study locations to find a site near you, 4) Review the eligibility criteria, and 5) Contact the study site or complete the eligibility form. The process is free and you can withdraw at any time.
Are systemic mastocytosis clinical trials free?
Yes, participation in systemic mastocytosis clinical trials is free. Study-related treatments, medical tests, and doctor visits are provided at no cost to participants. Many studies also offer compensation for your time and travel expenses.
What types of systemic mastocytosis treatments are being studied?
Current systemic mastocytosis clinical trials are testing a range of approaches. These include new drugs, combination therapies, medical devices, and other interventions sponsored by 3 research organizations.
Is it safe to participate in systemic mastocytosis clinical trials?
Clinical trials are carefully regulated by the FDA and institutional review boards (IRBs). All trials must follow strict safety protocols, and participants receive close medical monitoring throughout the study. You can withdraw from a trial at any time without penalty.
Data updated March 1, 2026 from ClinicalTrials.gov
About This Data
Clinical trial information on this page is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health (NIH) and National Library of Medicine (NLM). Study data is refreshed every hour to ensure accuracy.
Medical Disclaimer: The information provided on this page is for informational purposes only and does not constitute medical advice. Always consult with a qualified healthcare provider before making decisions about clinical trial participation or changes to your treatment plan.
Page reviewed by the HelloStudys Research Team · Last updated March 1, 2026 · Data from ClinicalTrials.gov