Avapritinib

Generic Name: Avapritinib

Brand Names: Ayvakit, Ayvakyt

Drug Type: Small Molecule

Chemical Formula: C_₂₆H_₂₇FN_₁₀

CAS Number: 1703793-34-3

Unique Ingredient Identifier: 513P80B4YJ

Background: Avapritinib, or BLU-285, is a selective tyrosine kinase inhibitor of KIT and platelet derived growth factor receptor alpha indicated for the treatment of unresectable, metastatic gastrointestinal stromal tumors and advanced systemic mastocytosis. It is one of the first medications available for the treatment of multidrug resistant cancers. Avapritinib shares a similar mechanism with ripretinib.

Avapritinib was granted FDA approval on 9 January 2020 and EMA approval on 24 September 2020.

Indication: Avapritinib is indicated for the treatment of adults with unresectable or metastatic GIST harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations.

It is also used to treat adult patients with advanced systemic mastocytosis (AdvSM). AdvSM includes patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN), and mast cell leukemia. However, it is not recommended for the treatment of patients with AdvSM with platelet counts of less than 50 X 10 L.

Associated Conditions: Advanced Systemic Mastocytosis (AdvSM), Aggressive Systemic Mastocytosis, Indolent Systemic Mastocytosis, Mast Cell Leukemia (MCL), Metastatic Gastrointestinal Stromal Tumor (GIST), Systemic Mastocytosis With an Associated Hematological Neoplasm, Unresectable Gastrointestinal stromal tumor

Clinical Trials

Avapritinib Maintenance for AML With KIT Mutations

Phase 2

Not yet recruiting

Conditions: AML, Adult

Interventions: Drug: Avapritinib

First Posted Date: 2025-01-09

Last Posted Date: 2025-01-09

Lead Sponsor: Ruijin Hospital

Target Recruit Count: 47

Registration Number: NCT06765915

Locations: 🇨🇳
Ruijin Hospital of Shanghai Jiaotong University, Shanghai, Shanghai, China

Avapritinib Rollover Study

Phase 4

Recruiting

Conditions: Mastocytosis, Systemic

Interventions: Drug: Avapritinib

First Posted Date: 2024-12-24

Last Posted Date: 2025-04-10

Lead Sponsor: Blueprint Medicines Corporation

Target Recruit Count: 60

Registration Number: NCT06748001

Locations: 🇬🇧
Guys and St Thomas NHS Foundation Trust - St Thomas Hospital, London, United Kingdom
🇧🇪
Universitair Ziekenhuis Antwerpen (UZA), Edegem, Belgium
🇮🇹
A.O.U. San Giovanni di Dio e Ruggi d'Aragona di Salerno, Salerno, Italy

and more 2 locations

Avapritinib With Decitabine in Patients With SM-AHN

Phase 1

Recruiting

Conditions: Systemic Mastocytosis With an Associated Hematologic Neoplasm

Interventions: Drug: Avapritinib
Drug: Decitabine
Combination Product: Decitabine/Cedazuridine

First Posted Date: 2024-03-25

Last Posted Date: 2025-03-12

Lead Sponsor: H. Lee Moffitt Cancer Center and Research Institute

Target Recruit Count: 34

Registration Number: NCT06327685

Locations: 🇺🇸
Dana-Farber Cancer Institute, Boston, Massachusetts, United States
🇺🇸
Mayo Clinic - Arizona, Phoenix, Arizona, United States
🇺🇸
Moffitt Cancer Center, Tampa, Florida, United States

and more 4 locations

Safety and Efficacy of Avapritinib in Relapsed or Refractory Pediatric CBF-AML With KIT Mutation

Phase 2

Recruiting

Conditions: Relapse/Recurrence
C-KIT Mutation
Core Binding Factor Acute Myeloid Leukemia
AML, Childhood
Refractory AML

Interventions: Drug: Avapritinib
Drug: Azacitidine Injection
Drug: Decitabine Injection
Drug: Idarubicin Hydrochloride
Drug: Cytarabine
Drug: Granulocyte Colony-Stimulating Factor

First Posted Date: 2024-03-19

Last Posted Date: 2024-08-22

Lead Sponsor: Children's Hospital of Soochow University

Target Recruit Count: 50

Registration Number: NCT06316960

Locations: 🇨🇳
The First Affiliated Hospital of Guangxi Medical University, Nanning, Guangxi, China
🇨🇳
Kaifeng Children's Hospital, Kaifeng, Henan, China
🇨🇳
Third Xiangya Hospital of Central South University, Changsha, Hunan, China

and more 9 locations

Clinical Study of Induction Therapy Options Based on Molecular Subtyping and MRD in Children and Adolescents With AML

Phase 2

Recruiting

Conditions: AML, Childhood
Acute Myeloid Leukemia

Interventions: Drug: Homoharringtonine
Drug: Mitoxantrone hydrochloride liposome
Drug: Cytarabine
Drug: Recombinant Human Granulocyte Colony-Stimulating Factor
Drug: Etoposide
Drug: Idarubicin Hydrochloride
Drug: Venetoclax
Drug: Sorafenib
Drug: Gilteritinib
Drug: Avapritinib

First Posted Date: 2024-01-24

Last Posted Date: 2024-08-22

Lead Sponsor: Children's Hospital of Soochow University

Target Recruit Count: 500

Registration Number: NCT06221683

Locations: 🇨🇳
The First Affiliated Hospital of Guangxi Medical University, Nanning, Guangxi, China
🇨🇳
XiangYa Hospital Central South University, Changsha, Hunan, China
🇨🇳
Children's Hospital of Soochow University, Suzhou, Jiangsu, China

and more 10 locations

Avapritinib in CBF-AML With KIT Mutations

Phase 2

Recruiting

Conditions: Core Binding Factor Acute Myeloid Leukemia
KIT Mutation-Related Tumors

Interventions: Drug: Avapritinib

First Posted Date: 2023-04-20

Last Posted Date: 2023-05-09

Lead Sponsor: The First Affiliated Hospital of Soochow University

Target Recruit Count: 50

Registration Number: NCT05821738

Locations: 🇨🇳
First Affiliated Hospital of Soochow University, Suzhou, Jiangsu, China

Safety and Efficacy of Avapritinib in Chinese Patients With Gastrointestinal Stromal Tumor (GIST) in the Real World

Completed

Conditions: Gastrointestinal Stromal Tumors

Interventions: Drug: Avapritinib
Drug: Tyrosine kinase inhibitors other than avapritinib ( imatinib, sunitinib, et al )

First Posted Date: 2022-05-19

Last Posted Date: 2024-11-13

Lead Sponsor: CStone Pharmaceuticals

Target Recruit Count: 61

Registration Number: NCT05381753

Locations: 🇨🇳
Beijing Cancer Hospital, Beijing, Beijing, China

A Drug-drug Interaction Study of Avapritinib and Midazolam

Phase 1

Completed

Conditions: Recurrent or Unresectable Central Nervous System (CNS) Tumors
Gastrointestinal Stromal Tumors
GIST
Non-resectable Advanced Solid Tumors

Interventions: Drug: Avapritinib
Drug: midazolam

First Posted Date: 2021-06-01

Last Posted Date: 2024-12-13

Lead Sponsor: Blueprint Medicines Corporation

Target Recruit Count: 10

Registration Number: NCT04908176

Locations: 🇺🇸
Mayo Clinic Florida, Jacksonville, Florida, United States
🇺🇸
Thomas Jefferson University, Sidney Kimmel Cancer Center, Philadelphia, Pennsylvania, United States
🇺🇸
University of Michigan, Ann Arbor, Michigan, United States

Study for Patients Previously Treated in Avapritinib Clinical Trials

Phase 4

Completed

Conditions: Gastrointestinal Stromal Tumors

Interventions: Drug: Avapritinib

First Posted Date: 2021-04-01

Last Posted Date: 2024-11-26

Lead Sponsor: Blueprint Medicines Corporation

Target Recruit Count: 2

Registration Number: NCT04825574

Locations: 🇫🇷
Gustave Roussy Cancer Campus Grand Paris Institut de Cancerologie Gustave-Roussy, Villejuif, Val-de-Marne, France

A Study of Avapritinib in Pediatric Patients With Solid Tumors Dependent on KIT or PDGFRA Signaling

Phase 1

Recruiting

Conditions: Solid Tumor, Unspecified, Child
Relapsed Solid Neoplasm
CNS Tumor

Interventions: Drug: avapritinib

First Posted Date: 2021-02-26

Last Posted Date: 2023-06-15

Lead Sponsor: Blueprint Medicines Corporation

Target Recruit Count: 37

Registration Number: NCT04773782

Locations: 🇫🇷
Gustave Roussy, Villejuif, France
🇩🇪
Universitaetsmedizin Göttingen, Göttingen, Lower Saxony, Germany
🇩🇪
Hopp Children's Cancer Center, Heidelberg, Germany

and more 23 locations

Drug Development Updates

Stay informed with timely notifications on clinical trials, regulatory changes, and research advancements related to this medication.

Home Terms & Conditions Privacy Policy