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

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Brief Title

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

Official Title

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

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.
    



Study Type

Observational


Primary Outcome

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

Secondary Outcome

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

Condition

Advanced Systemic Mastocytosis


Study Arms / Comparison Groups

 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

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information



Estimated Enrollment

250

Start Date

December 2, 2020

Completion Date

September 2021

Primary Completion Date

September 2021

Eligibility 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

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

Austria

Location Countries

Austria

Administrative Informations


NCT ID

NCT04695431

Organization ID

BLU-285-2405


Responsible Party

Sponsor

Study Sponsor

Blueprint Medicines Corporation

Collaborators

 Analysis Group, Inc.

Study Sponsor

, , 


Verification Date

January 2021