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

An External Control, Observational, Retrospective Study Assessing the Effect of Avapritinib Compared With Best Available Therapy for Patients With Advanced Systemic Mastocytosis

Sponsors

Lead Sponsor: Blueprint Medicines Corporation

Collaborator: Analysis Group, Inc.

Source Blueprint Medicines Corporation
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.

Overall Status Enrolling by invitation
Start Date 2020-12-02
Completion Date 2021-10-01
Primary Completion Date 2021-05-01
Study Type Observational
Primary Outcome
Measure Time Frame
Comparative evaluation of overall survival (OS) between patients receiving best available therapy versus avapritinib in BU-285-2101 and BLU-285-2202 up to 12 years
Secondary Outcome
Measure Time Frame
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
Enrollment 250
Condition
Eligibility

Sampling Method:

Probability Sample

Criteria:

Inclusion Criteria for Patients in the External Control Arm: 1. Diagnosed with AdvSM, with known subtype including SM-AHN, ASM, or MCL 2. 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 3. Adult (≥18 years of age) at the initiation of first systemic line of therapy at the participating site 4. Had available performance status (e.g., ECOG score or Karnofsky score) 5. 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 6. 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 Exclusion Criteria for Patients in the External Control Arm 1. 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 2. 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 3. 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.

Gender:

All

Minimum Age:

18 Years

Maximum Age:

N/A

Healthy Volunteers:

No

Location
Facility:
Standford Cancer Center | Palo Alto, California, 94304, United States
Dana Farber Cancer Institute | Boston, Massachusetts, 02215, United States
Medizinische Universitat Wien | Vienna, Austria
Universitatmedizin Mannheim | Mannheim, Baden-Württemberg, Germany
Hospital Virgen del Valle | Toledo, Spain
Guy's and St. Thomas' NHS Foundation Trust | London, England, United Kingdom
Location Countries

Austria

Germany

Spain

United Kingdom

United States

Verification Date

2021-01-01

Responsible Party

Type: Sponsor

Has Expanded Access No
Condition Browse
Arm Group

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

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

Label: External Control Group

Description: Patients with advanced systemic mastocytosis that received best available therapy

Patient Data No
Study Design Info

Observational Model: Cohort

Time Perspective: Retrospective

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