Retrospective Study Assessing the Effect of Avapritinib Versus Best Available Therapy in Patients With AdvSM

NCT04695431 | Completed

• 1 other identifier: BLU-285-2405
• Study Type: observational
• Target: 317
• Locations: 5 countries
• Sites: 6

Brief Summary

BLU-285-2405 is a multi-center, synthetic control, observational and retrospective study designed to compare clinical outcomes for avapritinib compared with best available therapy for patients with AdvSM.

Conditions

Advanced Systemic Mastocytosis; Aggressive Systemic Mastocytosis; Systemic Mastocytosis With an Associated Hematological Neoplasm; Mast Cell Leukemia

Outcome Measures

Primary Outcomes (1)

• Comparative evaluation of overall survival (OS) between patients receiving best available therapy versus avapritinib in BU-285-2101 and BLU-285-2202

  Overall Survival defined as time from initiation of systemic treatment to death from any cause

  up to 12 years

Secondary Outcomes (4)

• Comparative evaluation between patients receiving best available therapy versus avapritinib of duration of treatment (DOT)

  Up to 12 years

• Comparative evaluation between patients receiving best available therapy versus avapritinib and time to next treatment line (TtNTL)

  Up to 12 years

• Comparative evaluation of change in serum tryptase concentration in patients receiving best available therapy versus avapritinib

  Up to 12 years

• To characterize the safety profile and conduct comparative evaluation of safety between patients receiving best available therapy vs. avapritinib

  Up to 12 years

Study Arms (2)

Patients from the BLU-285-2101 and BLU-285-2202 studies

Patients with advanced systemic mastocytosis who received treatment with avapritinib as part of the BLU-285-2101 and BLU-285-2202 studies

External Control Group

Patients with advanced systemic mastocytosis that received best available therapy

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Patients age 18 or older with a diagnosis of advanced SM.

You may qualify if:

• Diagnosed with AdvSM, with known subtype including SM-AHN, ASM, or MCL
• Received at least one line of systemic therapy for AdvSM, which may include but not limited to regimens containing:
• Midostaurin Cytoreductive therapy: cladribine, interferon alpha, azacitidine, decitabine Selective TKIs: imatinib, nilotinib, dasatinib Hydroxyurea Antibody therapy: brentuximab vedotin
• Adult (≥18 years of age) at the initiation of first systemic line of therapy at the participating site
• Had an index date at least 3 months prior to the start of data collection (in order to include patients with at least 3 months of follow-up after index date), unless date of death occurred less than three months from index date
• Had an approved waiver of informed consent or signed informed consent for participation in the retrospective chart review study, if no institutional waiver from the site was granted

You may not qualify if:

• Malignancy that is not in remission at time of SM diagnosis, or new non-hematological malignancy diagnosed after SM diagnosis, except for: completely resected basal cell and squamous cell skin cancer, curatively treated localized prostate cancer, and completely resected carcinoma in situ of any site
• Among patients with SM-AHN, presence of either of the following:
• Patients in whom the SM component is consistent with an indolent systemic mastocytosis (ISM) or SSM or,
• the AHN component is a lymphoid malignancy, or one of the following myeloid malignancies: acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) that is very high-or high-risk as defined by the IPSS-R, or a Philadelphia chromosome positive malignancy or,
• there is a known FIP1L1/PDGFRA fusion gene (including those with CHIC-2 deletion and partial deletion of PDGFRA), independent of KIT mutational status
• Received avapritinib as the first line of systemic therapy for AdvSM at participating site, or prior to initiation of first systemic therapy at participating site.

Sponsors & Collaborators

• Blueprint Medicines Corporation: lead
• Analysis Group, Inc.: collaborator

Study Sites (6)

Standford Cancer Center

Palo Alto, California, 94304, United States

Location

Dana Farber Cancer Institute

Boston, Massachusetts, 02215, United States

Location

Medizinische Universitat Wien

Vienna, Austria

Location

Universitatmedizin Mannheim

Mannheim, Baden-Wurttemberg, Germany

Location

Hospital Virgen del Valle

Toledo, Spain

Location

Guy's and St. Thomas' NHS Foundation Trust

London, England, United Kingdom

Location

Related Publications (1)

• Reiter A, Gotlib J, Alvarez-Twose I, Radia DH, Lubke J, Bobbili PJ, Wang A, Norregaard C, Dimitrijevic S, Sullivan E, Louie-Gao M, Schwaab J, Galinsky IA, Perkins C, Sperr WR, Sriskandarajah P, Chin A, Sendhil SR, Duh MS, Valent P, DeAngelo DJ. Efficacy of avapritinib versus best available therapy in the treatment of advanced systemic mastocytosis. Leukemia. 2022 Aug;36(8):2108-2120. doi: 10.1038/s41375-022-01615-z. Epub 2022 Jul 5.

  PMID: 35790816 DERIVED

MeSH Terms

Conditions

Mastocytosis, Systemic; Leukemia, Mast-Cell

Condition Hierarchy (Ancestors)

Mastocytosis; Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Mast Cell Activation Disorders; Immune System Diseases; Leukemia; Leukemia, Myeloid, Acute; Leukemia, Myeloid; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 15, 2020
• First Posted: January 5, 2021
• Study Start: December 2, 2020
• Primary Completion: October 4, 2021
• Study Completion: October 4, 2021
• Last Updated: January 10, 2022

Record last verified: 2021-12

Data Sharing

• IPD Sharing: Will not share

Locations

DE (1); US (2); AU (1); ES (1); GB (1)