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, a Sanofi company

Now Enrolling: The HARBOR study.

Living with indolent systemic mastocytosis (ISM)? You may qualify for the HARBOR Study evaluating an investigational drug. This study is being conducted by Blueprint Medicines, a Sanofi Company.

What is the purpose of the HARBOR Study?

HARBOR is a phase 2/3 study that is exploring whether a study drug, called elenestinib, could be safe and effective in treating indolent systemic mastocytosis (ISM).

Elenestinib is an investigational compound under ongoing research. It has not been approved by the U.S. FDA or any regulatory authority in any jurisdiction as safe or effective for use in ISM or any other indication.

Is study participation right for me?

You may be eligible if you:

• Are 18 years or older

• Have been diagnosed with ISM

• Have symptoms associated with your ISM diagnosis

Talk to your healthcare provider if you are interested in learning more about this clinical study.

A study doctor and staff can explain to you in greater detail other study requirements and eligibility criteria.

To learn more about the HARBOR study, visit www.HarborClinicalTrial.com or contact 877-93-HARBOR (877-934-2726).

Cogent Biosciences

Cogent Biosciences’ clinical trials have completed enrollment and are no longer recruiting. However, there is an Expanded Access Program available for patients in US.

Expanded Access Program for Bezuclastinib (United States Only): 

• The purpose of this Expanded Access Program (EAP) protocol is to provide investigational bezuclastinib to patients with a diagnosis of Nonadvanced Systemic Mastocytosis (NonAdvSM) or Advanced Systemic Mastocytosis (AdvSM) who have received and failed or been intolerant to at least one standard approved therapy and/or have no comparable or satisfactory alternative therapy options 
• ClinicalTrials.gov ID: NCT06915766 (Hyperlink to: https://clinicaltrials.gov/study/NCT06915766)  
• Status: A patient’s physician can submit a request to Cogent for Expanded Access to their investigational therapy for Systemic Mastocytosis. These requests must come from the treating physician and cannot be made directly by the patient, their parent/legal guardian, or caregiver. Requests are reviewed on a case-by-case basis, and submitting a request does not guarantee access. More information about Cogent’s Expanded Access Program can be found at https://www.cogentbio.com/Bezuclastinib/#our-expanded-access-policy  

SM-AHN sub-study still enrolling

Apex Clinical Trial: Bezuclastinib in Patients with Advanced Systemic Mastocytosis

Status: Main trial has completed enrollment, no longer recruiting

SM-AHN sub-study: RECRUITING, enrolling patients with Systemic Mastocytosis with an Associated Hematologic Neoplasm (SM-AHN) who are receiving or who are appropriate to receive treatment with azacitidine. Patients will receive Cogent’s investigational therapy, bezuclastinib, while taking azacitidine for their AHN.

Locations: United States (Arizona, California, Georgia, Massachusetts, Ohio, South Carolina, Texas, Utah), Australia, Canada, Switzerland, United Kingdom

ClinicalTrials.gov ID NCT04996875 https://clinicaltrials.gov/study/NCT04996875