Clinical Trials

Mast cell disease patients can play a crucial role in developing new treatments. We are grateful to share clinical trial information from our partners that adhere to international regulatory and clinical standards and conduct ethical and transparent research.

Clinical Trial Participation

The decision to participate in a clinical trial is one that you need to consider carefully. Educate yourself about the investigational drug as well as the clinical trial to assess:

• Effectiveness and safety profile of the investigational drug,
• Risks and benefits of trial participation,
• Trial participation requirements (e.g. how many times you need to visit the clinical site.

One of the best sources of information is your physician(s) as well as the trial site investigator.

It’s important to know that clinical trials are not treatments; they are research studies designed to answer very specific questions about a drug or procedure. When deciding to participate in a clinical trial you will need to weigh the potential trial benefits along with the associated risks.

Active Clinical Trials

Clinical trials are an integral part of drug development and are required before a medicine can be available by prescription. Information on active trials, trials that are currently recruiting and those that will soon be recruiting can be found at the link below.

We have pulled together some searches from clinicaltrials.gov for some common mast cell disease related search terms. You can find links to these searches here: Mast Cell Searches of Active Clinical Trials

For more information visit clinicaltrials.gov

Active Trials from our Partners in Hope

Blueprint Medicines

Blueprint Medicines has two actively enrolling studies.

HARBOR: Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis https://clinicaltrials.gov/ct2/show/NCT04910685

AZURE: Study of Elenestinib (BLU-263) in Advanced Systemic Mastocytosis (AdvSM) and Other KIT Altered Hematologic Malignancies https://www.clinicaltrials.gov/ct2/show/NCT05609942

Cogent Biosciences

Cogent Biosciences is currently enrolling two clinical trials to evaluate an investigational medication called bezuclastinib (CGT9486) for people with Systemic Mastocytosis.

For more information about Cogent’s clinical trials and your potential eligibility for the studies, please visit: www.mastocytosistrials.com/TMS

• Apex is a Global Phase 2 clinical trial for people with Advanced Systemic Mastocytosis including SM with an Associated Hematologic Neoplasm (SM-AHN), Mast Cell Leukemia (MCL), and Aggressive Systemic Mastocytosis (ASM)
• Summit is a Global Phase 2 clinical trial for people with Nonadvanced Systemic Mastocytosis, including Indolent Systemic Mastocytosis (ISM), Smoldering Systemic Mastocytosis (SSM), and Bone Marrow Mastocytosis (BMM)