- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04695431
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
Study Overview
Status
Study Type
Enrollment (Actual)
Contacts and Locations
Study Locations
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Vienna, Austria
- Medizinische Universität Wien
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Baden-Württemberg
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Mannheim, Baden-Württemberg, Germany
- Universitatmedizin Mannheim
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Toledo, Spain
- Hospital Virgen del Valle
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England
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London, England, United Kingdom
- Guy's and St. Thomas' NHS Foundation Trust
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California
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Palo Alto, California, United States, 94304
- Standford Cancer Center
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Massachusetts
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Boston, Massachusetts, United States, 02215
- Dana Farber Cancer Institute
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Sampling Method
Study Population
Description
Inclusion Criteria for Patients in the External Control Arm:
- 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
Exclusion Criteria for Patients in the External Control Arm
- 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.
Study Plan
How is the study designed?
Design Details
- Observational Models: Cohort
- Time Perspectives: Retrospective
Cohorts and Interventions
Group / Cohort |
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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
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External Control Group
Patients with advanced systemic mastocytosis that received best available therapy
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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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
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Overall Survival defined as time from initiation of systemic treatment to death from any cause
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up to 12 years
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Comparative evaluation between patients receiving best available therapy versus avapritinib of duration of treatment (DOT)
Time Frame: Up to 12 years
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DOT, defined as the duration from initiation of line of systemic treatment to discontinuation of same line of treatment for any reason and;
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Up to 12 years
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Comparative evaluation between patients receiving best available therapy versus avapritinib and time to next treatment line (TtNTL)
Time Frame: Up to 12 years
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TtNTL, defined as the time from initiation of the line of systemic treatment to the initiation of the next line of treatment
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Up to 12 years
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Comparative evaluation of change in serum tryptase concentration in patients receiving best available therapy versus avapritinib
Time Frame: Up to 12 years
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Change in serum tryptase concentration, defined as change in serum tryptase from baseline, for each line of therapy
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Up to 12 years
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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
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AEs that result in treatment modification or discontinuation, hospitalization, or death according to evaluation of responsible physician
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Up to 12 years
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Collaborators and Investigators
Sponsor
Collaborators
Publications and helpful links
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Skin Diseases
- Immune System Diseases
- Neoplasms, Connective and Soft Tissue
- Neoplasms by Histologic Type
- Neoplasms
- Neoplasms by Site
- Hematologic Diseases
- Hypersensitivity
- Leukemia
- Neoplasms, Connective Tissue
- Immune Complex Diseases
- Leukemia, Myeloid
- Leukemia, Myeloid, Acute
- Hematologic Neoplasms
- Mastocytosis
- Mastocytosis, Systemic
- Leukemia, Mast-Cell
Other Study ID Numbers
- BLU-285-2405
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
IPD Plan Description
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
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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