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:
- 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.