Clinical Trial Title(Apex) CGT9486 in Patients with Advanced Systemic Mastocytosis
National Clinical Trial Number:NCT04996875
Clinical Trial Protocol Description:
Mastocytosis is an abnormal accumulation of mast cells in one or more organ systems. Advanced systemic mastocytosis (AdvSM) is a form of mastocytosis in which mast cells accumulate in internal tissues and organs such as the liver, spleen, bone marrow, and small intestines. Signs and symptoms vary based on which parts of the body are affected. There are three types of AdvSM: aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematologic neoplasm (SM-AHN), and mast cell leukemia (MCL).
CGT9486 is an investigational tyrosine kinase inhibitor that targets the mutated KIT gene. It is an oral drug (pills). Which part of the study a person participates in depends on which part of the study is enrolling, or “open,” when they join the study.
Part 1 participants will be assigned at random to a dose group. Each group will have a different dose of study drug, and the participant will take study drug either once or twice per day. Participants have an equal chance to be assigned to any of the open groups. The participant and the study doctor and staff will know to which group the participant is assigned. Once all of the participants in Part 1 have been in the study for 2 months, the Sponsor review study data and select a dose to use in Part 2. Participants in Part 2 will all receive the same dose that was selected in Part 1.
During Cycle 1, study visits are on days 1, 2, 8, 15, and 22. During Cycle 2, study visits are on days 1, 2, and 15. During Cycles 3-15 study visits are on Day 1. For every other cycle starting after Cycle 15, until the end of treatment, the study visit will be on Day 1. If a participant is randomized into Part 1, they will also return to the study site on days 4 and 11 of Cycle 1 and days 8 and 22 of Cycle 2. Each study visit may be completed in up to1 full day, or be scheduled across multiple days depending on the study doctor and staff’s decision.
Long-term Follow-up for the study is every 3-months after the participant’s Safety Follow-up Visit, for at least 2 years. Participants will be contacted and they may be asked about their health, their AdvSM, and
Clinical Trial Eligibility Criteria:
In order to participate you must meet the following criteria:
- Are 18 years of age or older.
- Have been diagnosed with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematologic neoplasm (SM-AHN), or mast cell leukemia (MCL).
- Have clinically acceptable local laboratory screening results.
- Are able to swallow pills.
You will be excluded from the study if any of the following criteria apply to you:
- Have persistent side effects from previous treatment for your AdvSM that has gone away.
- Have an associated hematologic cancer that requires immediate anticancer treatment.
- Have clinically significant cardiac disease.
- Have an active, uncontrolled, systemic bacterial, fungal, or viral infection at Screening.
This is a partial list of eligibility requirements. To inquire about your eligibility, please call the contact number provided. If you wish to inquire via email, please include the title of the study in your message.