The purpose of this expanded access program (EAP) protocol is to provide investigationalbezuclastinib to patients with a diagnosis of nonadvanced systemic mastocytosis(NonAdvSM) or advanced systemic mastocytosis (AdvSM) who have received and failed or beenintolerant to at least one standard approved therapy and/or have no comparable orsatisfactory alternative therapy options.
Not Provided
Drug: Bezuclastinib
Drug: Bezuclastinib Tablets
Other Name: CGT9486
Key Inclusion Criteria:
- Able to provide written informed consent and commit to EAP assessments.
- ≥18 years of age.
- Able to swallow tablets.
- Diagnosed with ASM, SM-AHN, MCL, BMM, ISM, or SSM according to the 2022 WHO
Classification for SM
- Not receiving adequate disease control on current therapy(ies).
- Have clinically acceptable laboratory screening results.
Exclusion Criteria:
- Patients who are eligible for and/or enrolled in an on-going bezuclastinib clinical
trial.
- Patients who discontinued investigational use of bezuclastinib in previous clinical
trials due to toxicity or withdrawal of consent.
- Pregnant or currently breastfeeding.
- Prior or ongoing clinically significant illness or medical or physical condition
Other protocol-defined criteria apply.
Orso Health/Modena Asthma Allergy
La Jolla, California, United States
Dartmouth-Hitchcock Medical Center
Lebanon, New Hampshire, United States
Duke Blood Cancer Center
Durham, North Carolina, United States
AIR Care
Dallas, Texas, United States
Alexandra Malinowski, PharmD
1-877-633-8049
medinfo@cogentbio.com
Rachael Easton, MD, PhD, Study Director
Cogent Biosciences, Inc.