Reviewed by Michael Gill, B. Sc.
12 Mastocytosis Clinical Trials Near Me
Top Hospitals for Mastocytosis Clinical Trials
Image of Dana Farber Cancer Institute in Massachusetts.
Dana Farber Cancer Institute
Boston
4Active Trials
7All Time Trials for Mastocytosis
2005First Mastocytosis Trial
Image of Stanford Cancer Institute in California.
Stanford Cancer Institute
Stanford
4Active Trials
4All Time Trials for Mastocytosis
2016First Mastocytosis Trial
Top Cities for Mastocytosis Clinical Trials
Image of Boston in Massachusetts.
Boston
8Active Trials
Dana Farber Cancer InstituteTop Active Site
Image of Ann Arbor in Michigan.
Ann Arbor
5Active Trials
University of MichiganTop Active Site
Mastocytosis Clinical Trials by Phase of Trial
Most Recent Mastocytosis Clinical Trials
Top Treatments for Mastocytosis Clinical Trials
Treatment Name
Active Mastocytosis Clinical Trials
All Time Trials for Mastocytosis
First Recorded Mastocytosis Trial
Avapritinib
3
3
2016
Best supportive care
1
1
2022
MGD024
1
1
2022
CGT9486 Tablets
1
1
2021
CGT9486 tablets
1
1
2021
Recently Completed Studies with FDA Approved Treatments for Mastocytosis
Treatment
Year
Sponsor
DCC-2618
2015
Deciphera Pharmaceuticals LLC

What Are Mastocytosis Clinical Trials?

Mastocytosis is a rare disorder in which abnormal mast cells accumulate and become active in the internal organs, skin, and bone marrow. Mast cells are specific white blood cells that release chemicals to create inflammatory responses and protect the body from infection. Mastocytosis can affect children and adults and is typically caused by variations in the KIT gene.

Symptoms of mastocytosis include hives, flushing, anaphylaxis, throat swelling, itching, shortness of breath, palpitations, BP changes, dizziness, uterus cramps, vomiting, muscle or bone pain, brain fog, etc. Doctors diagnose the disorder with bone marrow or skin biopsy. Mastocytosis cases that begin in adulthood are often chronic and involve the skin and bone marrow, while skin manifestations are characteristic of childhood cases.

Since mastocytosis is a rare disorder, it requires in-depth studying. People afflicted by it can partake in mastocytosis clinical trials to access new drugs, medical treatments, and the latest treatments for their condition. These clinical trials offer people the chance to contribute to the developing understanding of this condition.

Why Is Mastocytosis Being Studied Through Clinical Trials?

The life expectancy of people with mastocytosis is generally shorter than the life expectancy of the general population. Typically, most individuals die within 3 to 5 years after the initial diagnosis. Depending on its severity, mastocytosis can significantly impact an individual’s lifestyle.

Moreover, there is no cure for mastocytosis, but we have several treatment options. Mastocytosis clinical trials will help medical experts learn more about this disorder, discover possible cures and more effective treatment plans, and test new drugs and surgical approaches. Clinical trials can also help develop new screening and prevention methods.

What Are The Types of Treatments Available for Mastocytosis?

While there is no known cure for mastocytosis, many supportive treatments can help control its symptoms. Doctors use antihistamines to inhibit the effects of mast cell histamine. Mast cell stabilizers and leukotriene-inhibiting agents are also helpful. Symptoms like anaphylaxis and low BP are treated with epinephrine. Aggressive systemic mastocytosis requires kinase inhibitors, immune modulators, chemotherapeutic agents, or interferon.

What Are Some Recent Breakthrough Clinical Trials for Mastocytosis?

There have been multiple mastocytosis clinical trials in recent times. Here are the most notable ones:

2018: KIT Inhibition Clinical Validation in Advanced SM – KIT D816V is found in almost 90% of systemic mastocytosis patients. Detecting it is vital for making diagnostic and therapeutic decisions. The KIT inhibitor midostaurin was approved for advanced systemic mastocytosis; it can reverse organ damage and symptoms related to AdvSM.

2018-ongoing: PATHFINDER Clinical Trial to Evaluate the Safety & Efficacy of Avapritinib in Patients with AdvSM – This ongoing study and its earlier phase 1 study EXPLORER led to the FDA approving avapritinib in the treatment of adult patients with all types of AdvSM in June 2021.

Who Are Some of the Key Opinion Leaders / Researchers / Institutions Conducting Mastocytosis Trial Research?

Blueprint Medicines

Blueprint Medicines has worked on the PIONEER clinical trial of avapritinib in patients with non-advanced SM. The trial has shown successful results based on which Blueprint Medicine plans on submitting this drug’s application to the FDA and EMA.

The Mast Cell Disease Society

The Mast Cell Disease Society is the pioneer in mastocytosis clinical trials, research, resources, and more. This organization is dedicated to offering patients and medical professionals fighting mastocytosis the knowledge and research they need to discover new treatment therapies and create an effective management plan for mast cell diseases.

About The Author

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 20th, 2021

Last Reviewed: November 4th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.

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