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

December 17, 2021 updated by: Blueprint Medicines Corporation

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

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.

Study Overview

Status

Completed

Conditions

Study Type

Observational

Enrollment (Actual)

317

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

14 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Probability Sample

Study Population

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

Description

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 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
  5. 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Retrospective

Cohorts and Interventions

Group / Cohort
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Comparative evaluation of overall survival (OS) between patients receiving best available therapy versus avapritinib in BU-285-2101 and BLU-285-2202
Time Frame: up to 12 years
Overall Survival defined as time from initiation of systemic treatment to death from any cause
up to 12 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Comparative evaluation between patients receiving best available therapy versus avapritinib of duration of treatment (DOT)
Time Frame: Up to 12 years
DOT, defined as the duration from initiation of line of systemic treatment to discontinuation of same line of treatment for any reason and;
Up to 12 years
Comparative evaluation between patients receiving best available therapy versus avapritinib and time to next treatment line (TtNTL)
Time Frame: Up to 12 years
TtNTL, defined as the time from initiation of the line of systemic treatment to the initiation of the next line of treatment
Up to 12 years
Comparative evaluation of change in serum tryptase concentration in patients receiving best available therapy versus avapritinib
Time Frame: Up to 12 years
Change in serum tryptase concentration, defined as change in serum tryptase from baseline, for each line of therapy
Up to 12 years
To characterize the safety profile and conduct comparative evaluation of safety between patients receiving best available therapy vs. avapritinib
Time Frame: Up to 12 years
AEs that result in treatment modification or discontinuation, hospitalization, or death according to evaluation of responsible physician
Up to 12 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Blueprint Medicines Corporation

Collaborators

Analysis Group, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 2, 2020

Primary Completion (Actual)

October 4, 2021

Study Completion (Actual)

October 4, 2021

Study Registration Dates

First Submitted

December 15, 2020

First Submitted That Met QC Criteria

January 4, 2021

First Posted (Actual)

January 5, 2021

Study Record Updates

Last Update Posted (Actual)

January 10, 2022

Last Update Submitted That Met QC Criteria

December 17, 2021

Last Verified

December 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BLU-285-2405

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

IPD Plan Description

IPD will not be shared

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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