Avapritinib (BLU-285) Clinical Trials

Size	Price	Qty
2mg	USD 147
5mg	USD 257

Clinical Trials

Avapritinib has been investigated across numerous clinical trials evaluating its efficacy, safety, and pharmacokinetics from Phase 1 through Phase 4. Ranging from active and recruiting to completed status, these studies focus on gastrointestinal stromal tumors, systemic mastocytosis, mast cell leukemia, acute myeloid leukemia, and unresectable advanced solid or central nervous system tumors. Clinical development is sponsored by pharmaceutical companies such as Blueprint Medicines Corporation and CStone Pharmaceuticals, alongside academic medical centers including Centre Leon Berard and Ruijin Hospital.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT06221683	RECRUITING	AML, Childhood; Acute Myeloid Leukemia	Children's Hospital of Soochow University	2024-01-01	PHASE2
NCT07131059	RECRUITING	AML, Adult	Institute of Hematology & Blood Diseases Hospital, China	2024-05-11
NCT06748001	RECRUITING	Mastocytosis, Systemic	Blueprint Medicines Corporation	2024-11-28	PHASE4
NCT03731260	ACTIVE_NOT_RECRUITING	Indolent Systemic Mastocytosis	Blueprint Medicines Corporation	2019-04-16	PHASE2
NCT07028073	RECRUITING	Acute Myeloid Leukemia With T(8;21)(Q22;Q22); Acute Myeloid Leukemia With T(16;16)(P13;Q22); KIT Gene Mutation; Avapritinib	The First Affiliated Hospital of Soochow University	2025-05-15	PHASE1; PHASE2
NCT06327685	RECRUITING	Systemic Mastocytosis With an Associated Hematologic Neoplasm	H. Lee Moffitt Cancer Center and Research Institute	2024-03-13	PHASE1
NCT04771520	RECRUITING	Anatomic Stage IV Breast Cancer AJCC v8; Clinical Stage IV Gastroesophageal Junction Adenocarcinoma AJCC v8; Clinical Stage IVA Gastroesophageal Junction Adenocarcinoma AJCC v8; Clinical Stage IVB Gastroesophageal Junction Adenocarcinoma AJCC v8; Locally Advanced Malignant Solid Neoplasm; Locally Advanced Melanoma; Locally Advanced Primary Malignant Central Nervous System Neoplasm; Locally Advanced Sarcoma; Metastatic Malignant Solid Neoplasm; Metastatic Melanoma; Metastatic Primary Malignant Central Nervous System Neoplasm; Metastatic Sarcoma; Pathologic Stage IV Gastroesophageal Junction Adenocarcinoma AJCC v8; Pathologic Stage IVA Gastroesophageal Junction Adenocarcinoma AJCC v8; Pathologic Stage IVB Gastroesophageal Junction Adenocarcinoma AJCC v8; Postneoadjuvant Therapy Stage IV Gastroesophageal Junction Adenocarcinoma AJCC v8; Postneoadjuvant Therapy Stage IVA Gastroesophageal Junction Adenocarcinoma AJCC v8; Postneoadjuvant Therapy Stage IVB Gastroesophageal Junction Adenocarcinoma AJCC v8; Prognostic Stage IIIC Breast Cancer AJCC v8; Prognostic Stage IV Breast Cancer AJCC v8; Stage IIIC Colorectal Cancer AJCC v8; Stage IIIC Lung Cancer AJCC v8; Stage IV Colorectal Cancer AJCC v8; Stage IV Lung Cancer AJCC v8; Stage IVA Colorectal Cancer AJCC v8; Stage IVA Lung Cancer AJCC v8; Stage IVB Colorectal Cancer AJCC v8; Stage IVB Lung Cancer AJCC v8; Stage IVC Colorectal Cancer AJCC v8	M.D. Anderson Cancer Center	2021-01-20	PHASE2
NCT06783790	RECRUITING	AML (Acute Myelogenous Leukemia)	Institute of Hematology & Blood Diseases Hospital, China	2025-01-20	PHASE2
NCT04116541	RECRUITING	Malignant Solid Tumor	Centre Leon Berard	2020-01-28	PHASE2
NCT06316960	RECRUITING	AML, Childhood; Relapse/Recurrence; Refractory AML; Core Binding Factor Acute Myeloid Leukemia; C-KIT Mutation	Children's Hospital of Soochow University	2024-03-01	PHASE2
NCT06765915	NOT_YET_RECRUITING	AML, Adult	Ruijin Hospital	2025-02-01	PHASE2
NCT04773782	COMPLETED	Solid Tumor, Unspecified, Child; Relapsed Solid Neoplasm; CNS Tumor	Blueprint Medicines Corporation	2022-02-24	PHASE1; PHASE2
NCT05821738	UNKNOWN	Core Binding Factor Acute Myeloid Leukemia; KIT Mutation-Related Tumors	The First Affiliated Hospital of Soochow University	2022-06-01	PHASE2
NCT03580655	COMPLETED	Advanced Systemic Mastocytosis; Aggressive Systemic Mastocytosis; Systemic Mastocytosis With an Associated Hematologic Neoplasm; Mast Cell Leukemia	Blueprint Medicines Corporation	2018-11-21	PHASE2
NCT04908176	COMPLETED	Gastrointestinal Stromal Tumors; GIST; Non-resectable Advanced Solid Tumors; Recurrent or Unresectable Central Nervous System (CNS) Tumors	Blueprint Medicines Corporation	2022-08-24	PHASE1
NCT04825574	COMPLETED	Gastrointestinal Stromal Tumors	Blueprint Medicines Corporation	2021-05-21	PHASE4
NCT02561988	COMPLETED	Aggressive Systemic Mastocytosis; Systemic Mastocytosis-associated Hematologic Non-mast Cell Disease; Mast Cell Leukemia; Relapsed or Refractory Myeloid Malignancies	Blueprint Medicines Corporation	2016-03-10	PHASE1
NCT04908176	Completed	Gastrointestinal Stromal Tumors\|GIST\|Non-resectable Advanced Solid Tumors\|Recurrent or Unresectable Central Nervous System (CNS) Tumors	Blueprint Medicines Corporation	2022-08-24	Phase 1
NCT04695431	Completed	Advanced Systemic Mastocytosis\|Aggressive Systemic Mastocytosis\|Systemic Mastocytosis With an Associated Hematological Neoplasm\|Mast Cell Leukemia	Blueprint Medicines Corporation\|Analysis Group Inc.	2020-12-02	--
NCT04254939	COMPLETED	Gastrointestinal Stromal Tumors	CStone Pharmaceuticals	2019-08-15	PHASE1; PHASE2
NCT03465722	COMPLETED	GIST	Blueprint Medicines Corporation	2018-03-26	PHASE3
NCT02508532	COMPLETED	Gastrointestinal Stromal Tumors (GIST); Other Relapsed or Refractory Solid Tumors	Blueprint Medicines Corporation	2015-08	PHASE1
NCT03731260	Active not recruiting	Indolent Systemic Mastocytosis	Blueprint Medicines Corporation	2019-04-16	Phase 2
NCT03580655	Active not recruiting	Advanced Systemic Mastocytosis\|Aggressive Systemic Mastocytosis\|Systemic Mastocytosis With an Associated Hematologic Neoplasm\|Mast Cell Leukemia	Blueprint Medicines Corporation	2018-11-21	Phase 2
NCT03465722	Completed	GIST	Blueprint Medicines Corporation	2018-03-26	Phase 3

(data from https://clinicaltrials.gov, updated on 2024-08-22)

Check the Avapritinib (BLU-285) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Avapritinib is a potent small-molecule inhibitor that selectively binds to and inhibits mutant forms of platelet-derived growth factor receptor alpha (PDGFRα D842V) and KIT (c-Kit D816V) receptor tyrosine kinases, blocking intracellular receptor autophosphorylation and downstream oncogenic signaling pathways. This inhibition reduces cellular proliferation and induces apoptosis in mutant-driven cancer cells, thereby suppressing tumor growth in clinical indications such as 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>).