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

Mast Cell Leukemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04695431
• Other study ID #: BLU-285-2405
• Status: Completed
• Start date: December 2, 2020
• Est. completion date: October 4, 2021

Study information

• Verified date: December 2021
• Source: Blueprint Medicines Corporation
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 317
• Est. completion date: October 4, 2021
• Est. primary completion date: October 4, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Advanced Systemic Mastocytosis
• Aggressive Systemic Mastocytosis
• Hematologic Neoplasms
• Leukemia, Mast-Cell
• Mast Cell Leukemia
• Mastocytosis
• Systemic Mastocytosis With an Associated Hematological Neoplasm

Locations

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

Sponsors (2)

Lead Sponsor	Collaborator
Blueprint Medicines Corporation	Analysis Group, Inc.

Countries where clinical trial is conducted

United States,  Austria,  Germany,  Spain,  United Kingdom, 

Outcome

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