About Mastocytosis Trials

About the Apex Clinical Trial

What is Advanced Systemic Mastocytosis?

Advanced systemic mastocytosis (AdvSM) is a rare, aggressive, life-threatening disease that occurs when mast cells accumulate in organs and tissues of the body.^g Mast cells are a type of white blood cell that are part of your immune system. Mast cells are important in allergic reactions and fighting infections. In most cases, the abnormal accumulation of mast cells is caused by a genetic mutation in the KIT tyrosine receptor kinase gene, specifically KIT D816V.^h Diagnosis is difficult and current treatments approved by the FDA and other healthcare authorities have side effects which have limited dosing and availability of these treatments.^i,j,k

Patients with AdvSM may suffer from a number of debilitating symptoms such as:

Fatigue
Nausea, Abdominal Pain, and other gastrointestinal symptoms
Low blood counts
Bone pain and/or fractures
Abnormal liver function and/or enlargement of the liver
Enlargement of the spleen and/or lymph nodes
Muscle or joint pain
Headaches and/or difficulty concentrating
Itching, Skin redness/swelling
Flush episodes (reddening of the face/sudden feeling of warmth)

What is the Apex Clinical Trial?

The Apex clinical trial is a Phase 2 clinical trial evaluating an investigational medication bezuclastinib (CGT9486) in people with AdvSM. Bezuclastinib is designed to selectively and potently target the gene, KIT D816V, known to drive AdvSM in most cases. The investigational medication is in the form of a pill to be taken orally every day. The study aims to identify an optimal, safe dose of bezuclastinib and to determine the efficacy of bezuclastinib.

About the Summit Clinical Trial

What is Nonadvanced Systemic Mastocytosis?

Nonadvanced systemic mastocytosis (NonAdvSM) is a group of rare, life-long diseases caused by an accumulation of mast cells in organs and tissues, causing severely debilitating and potentially life-threatening symptoms. Mast cells are a type of immune cell that are important in fighting infections and cause many of the symptoms of allergic reactions. Indolent Systemic Mastocytosis (ISM) and Smoldering Systemic Mastocytosis (SSM) are both considered forms of NonAdvSM and in most cases, are caused by a genetic mutation in the KIT tyrosine receptor kinase gene, specifically KIT D816V. Diagnosis for NonAdvSM is difficult and currently there are no available disease specific treatments for patients.

Patients with NonAdvSM may suffer from a number of chronic symptoms such as:^l,m,n

Itching, skin lesions, skin redness and warmth (flushing)
Wheezing, shortness of breath
Fatigue
Anaphylaxis
Abdominal pain, bloating, diarrhea, nausea, vomiting
Brain fog, dizziness, memory impairment, mood changes, or trouble concentrating
Bone and joint pain

What is the Summit Clinical Trial?

The Summit clinical trial is a placebo-controlled Phase 2 clinical study evaluating an investigational medication bezuclastinib (CGT9486) in people with NonAdvSM. Bezuclastinib is designed to selectively and potently target KIT D816V, the gene which is known to drive NonAdvSM in most cases. The investigational medication is in the form of a pill to be taken orally each day. Patients will continue to receive their current medication(s) to control the symptoms of their NonAdvSM, known as their best supportive care, while they participate in this clinical trial. The study aims to identify an optimal, safe dose of bezuclastinib and to determine the efficacy of bezuclastinib.