Systemic mastocytosis Clinical Trials

8 recruitingLast updated: June 17, 2026

There are 8 actively recruiting systemic mastocytosis clinical trials across 26 countries. Studies span Phase 2, Phase 1, Phase 3. Top locations include Madrid, Spain, New York, New York, United States, Boston, Massachusetts, United States. Updated daily from ClinicalTrials.gov.


Systemic mastocytosis Trials at a Glance

8 actively recruiting trials for systemic mastocytosis are listed on ClinicalTrialsFinder across 6 cities in 26 countries. The largest study group is Phase 2 with 3 trials, with the heaviest enrollment activity in Madrid, New York, and Boston. Lead sponsors running systemic mastocytosis studies include Blueprint Medicines Corporation, Cogent Biosciences, Inc., and Fondazione IRCCS Policlinico San Matteo di Pavia.

Browse systemic mastocytosis trials by phase

Treatments under study

About Systemic mastocytosis Clinical Trials

Looking for clinical trials for Systemic mastocytosis? There are currently 3 studies actively recruiting participants. Clinical trials offer access to new treatments before they are widely available, and every approved therapy in use today was first tested through a clinical trial.

Below you can browse trials, sign up for alerts when new Systemic mastocytosis trials open, and view eligibility criteria for each study. Each listing includes the study phase, locations, and enrollment details.

Frequently Asked Questions

Common questions about Systemic mastocytosis clinical trials

A clinical trial is a carefully designed research study that tests new medical treatments, drugs, devices, or approaches in human volunteers. Every approved medication and treatment available today was proven safe and effective through clinical trials.

All clinical trials are reviewed and approved by Institutional Review Boards (IRBs) — independent committees that evaluate patient safety. Trials follow strict protocols, and your health is monitored closely throughout. You can withdraw at any time.

Not necessarily. Many trials compare the new treatment against the current standard of care, meaning all participants receive active treatment. When placebos are used, they are typically combined with standard treatment, not given alone. The trial description will always specify the design.

Under the Affordable Care Act, most private insurers are required to cover routine patient care costs during a clinical trial. The sponsor typically covers the investigational treatment itself. Medicare also covers routine costs for qualifying trials.

Yes. Participation is completely voluntary. You can withdraw at any time, for any reason, without it affecting your access to standard medical care.

Each trial has specific eligibility criteria — including age, diagnosis, disease stage, prior treatments, and general health. Browse the trials listed above and check their eligibility sections. You can also contact the trial site directly to discuss your situation.

Showing 18 of 8 trials

Recruiting
Phase 2Phase 3

(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis

Indolent Systemic MastocytosisSmoldering Systemic Mastocytosis
Blueprint Medicines Corporation534 enrolled68 locationsNCT04910685
Recruiting
Phase 2

(Apex) Bezuclastinib in Patients With Advanced Systemic Mastocytosis

Advanced Systemic Mastocytosis (AdvSM)SM With an Associated Hematologic Neoplasm (SM-AHN)Mast Cell Leukemia (MCL)+1 more
Cogent Biosciences, Inc.140 enrolled42 locationsNCT04996875
Recruiting

Peripheral Blood KIT-D816V Mutation in Adult Systemic Mastocytosis

Systemic mastocytosisKIT D816V Mutation
The First Affiliated Hospital of Soochow University50 enrolled1 locationNCT07562542
Recruiting

A Non-Interventional Study in Participants With Indolent Systemic Mastocytosis (ISM) in Germany

Indolent Systemic Mastocytosis
Blueprint Medicines Corporation80 enrolled8 locationsNCT07255638
Recruiting

An Observational Study in Participants With Indolent Systemic Mastocytosis (ISM)

Indolent Systemic Mastocytosis
Blueprint Medicines Corporation150 enrolled1 locationNCT07264959
Recruiting
Phase 1

Avapritinib With Decitabine in Patients With SM-AHN

Systemic Mastocytosis With an Associated Hematologic Neoplasm
H. Lee Moffitt Cancer Center and Research Institute34 enrolled7 locationsNCT06327685
Recruiting

Mastocytosis From Pediatric Age to Adulthood: Local Registry of Cutaneous and Systemic Mastocytosis

Systemic mastocytosisCutaneous Mastocytosis
Fondazione IRCCS Policlinico San Matteo di Pavia600 enrolled1 locationNCT07142473
Recruiting
Phase 2

Study of TL-895 in Subjects With Myelofibrosis or Indolent Systemic Mastocytosis

MyelofibrosisIndolent Systemic Mastocytosis
Telios Pharma, Inc.121 enrolled52 locationsNCT04655118