Clinical Trials

Multiple clinical trials are evaluating bezuclastinib in early- to mid-stage development, encompassing Phase 1, Phase 2, Phase 1/2, and expanded access protocols for gastrointestinal stromal tumors and systemic mastocytosis subtypes (indolent, aggressive, smoldering, advanced, and mast cell leukemia). Ranging from completed to actively recruiting and non-recruiting statuses, these studies assess monotherapy and sunitinib combination regimens. Industry sponsors including Cogent Biosciences Inc. and Plexxikon, alongside collaborators such as the Sarcoma Alliance for Research through Collaboration and Dana-Farber Cancer Institute, direct these investigations.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT06208748 ACTIVE_NOT_RECRUITING
Gastrointestinal Stromal Tumors; GIST
Sarcoma Alliance for Research through Collaboration
 2024-08-01 PHASE2
NCT04996875 RECRUITING
Advanced Systemic Mastocytosis (AdvSM); SM With an Associated Hematologic Neoplasm (SM-AHN); Mast Cell Leukemia (MCL); Aggressive Systemic Mastocytosis (ASM)
Cogent Biosciences, Inc.
 2021-11-09 PHASE2
NCT05186753 ACTIVE_NOT_RECRUITING
SSM; Mastocytosis, Indolent; Mastocytosis, Systemic; Mastocytosis; ISM; BMM; Smoldering Systemic Mastocytosis; Bone Marrow Mastocytosis
Cogent Biosciences, Inc.
 2022-06-27 PHASE2
NCT06208748 Not yet recruiting
Gastrointestinal Stromal Tumors|GIST
Sarcoma Alliance for Research through Collaboration|Cogent Biosciences Inc.|Dana-Farber Cancer Institute|The Life Raft Group
 2024-03 Phase 2
NCT04996875 Recruiting
Advanced Systemic Mastocytosis (AdvSM)|SM With an Associated Hematologic Neoplasm (SM-AHN)|Mast Cell Leukemia (MCL)|Aggressive Systemic Mastocytosis (ASM)
Cogent Biosciences Inc.
 2021-11-09 Phase 2
NCT02401815 Completed
Gastrointestinal Stromal Tumors
Cogent Biosciences Inc.|Plexxikon
 2015-03-06 Phase 1|Phase 2

(data from https://clinicaltrials.gov, updated on 2025-10-31)

Check the Bezuclastinib product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Bezuclastinib selectively binds to and inhibits wild-type and oncogenic mutant forms of the c-KIT receptor tyrosine kinase, including the resistance-conferring D816V mutation, which blocks downstream autophosphorylation and survival signaling. This targeted inhibition suppresses neoplastic cell proliferation, providing the mechanistic rationale for its clinical evaluation in gastrointestinal stromal tumors and systemic mastocytosis.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.