Advanced Systemic MastocytosisNCT04695431
Retrospective Study Assessing the Effect of Avapritinib Versus Best Available Therapy in Patients With AdvSM
An observational study of Advanced Systemic Mastocytosis and Aggressive Systemic Mastocytosis, run by Blueprint Medicines Corporation at 6 sites in 5 countries.
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Advanced Systemic Mastocytosis, Aggressive Systemic Mastocytosis, Systemic Mastocytosis With an Associated Hematological Neoplasm
- How many people
- 317 participants
- Where
- 6 sitesin 5 countries
- Study dates
- 2 Dec 2020to 4 Oct 2021
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients age 18 or older with a diagnosis of advanced SM.
Who may be able to join
None listed.
Who cannot join
- 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.
Trial locations and contacts
6 sites in 5 countries.
6 sites in 5 countries
United States2 sites
Dana Farber Cancer Institute
Boston, Massachusetts, 02215
Standford Cancer Center
Palo Alto, California, 94304
United Kingdom1 site
Guy's and St. Thomas' NHS Foundation Trust
London, England
About this study
Official title: 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.
- Conditions
- Advanced Systemic Mastocytosis, Aggressive Systemic Mastocytosis, Systemic Mastocytosis With an Associated Hematological Neoplasm, Mast Cell Leukemia
- Medical subject headings
- Mastocytosis, Systemic, Leukemia, Mast-Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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 being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: up to 12 years
Secondary outcomes (4)
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;
Measured: Up to 12 years
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
Measured: Up to 12 years
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
Measured: Up to 12 years
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
Measured: Up to 12 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jan 2021
- Study start2 Dec 2020
- Primary completion4 Oct 2021
- Study completion4 Oct 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Advanced Systemic MastocytosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 317 participants (actual)
Sponsor and investigators
- Lead sponsor
- Blueprint Medicines Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Analysis Group, Inc.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04695431
- Sponsor's protocol ID
- BLU-285-2405
- Status last verified
- Dec 2021
- First submitted
- 15 Dec 2020
- Last updated
- 10 Jan 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: IPD will not be shared
Publications
Reiter A, Gotlib J, Alvarez-Twose I, Radia DH, Lubke J, Bobbili PJ, Wang A, Norregaard C, Dimitrijevic S, Sullivan E, Louie-Gao M, Schwaab J, Galinsky IA, Perkins C, Sperr WR, Sriskandarajah P, Chin A, Sendhil SR, Duh MS, Valent P, DeAngelo DJ. Efficacy of avapritinib versus best available therapy in the treatment of advanced systemic mastocytosis. Leukemia. 2022 Aug;36(8):2108-2120. doi: 10.1038/s41375-022-01615-z. Epub 2022 Jul 5.
PubMed 35790816
Study at a glance
From the registry record, last updated by the sponsor on 10 Jan 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An External Control, Observational, Retrospective Study Assessing the Effect of Avapritinib Compared With Best Available Therapy for Patients With Advanced Systemic Mastocytosis
- ClinicalTrials.gov ID
- NCT04695431
- Acronym / sponsor ID
- BLU-285-2405
- Conditions
- Advanced Systemic Mastocytosis, Aggressive Systemic Mastocytosis, Systemic Mastocytosis With an Associated Hematological Neoplasm, Mast Cell Leukemia
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 317 participants (actual)
- Lead sponsor
- Blueprint Medicines CorporationIndustry (pharma, biotech, devices)
- Collaborators
- Analysis Group, Inc.
- Study start
- 2 Dec 2020 (actual)
- Primary completion
- 4 Oct 2021 (actual)
- Study completion
- 4 Oct 2021 (actual)
- First posted
- 5 Jan 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 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 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients age 18 or older with a diagnosis of advanced SM.
Key inclusion criteria (0 listed)
Key exclusion criteria (6 listed)
- 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:
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
4 secondary outcomes
Comparative evaluation between patients receiving best available therapy versus avapritinib of duration of treatment (DOT)
Time frame: 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
Comparative evaluation of change in serum tryptase concentration in patients receiving best available therapy versus avapritinib
Time frame: 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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jan 2022.
- Derived from this study: PubMed 35790816Reiter A, Gotlib J, Alvarez-Twose I, Radia DH, Lubke J, Bobbili PJ, Wang A, Norregaard C, Dimitrijevic S, Sullivan E, Louie-Gao M, Schwaab J, Galinsky IA, Perkins C, Sperr WR, Sriskandarajah P, Chin A, Sendhil SR, Duh MS, Valent P, DeAngelo DJ. Efficacy of avapritinib versus best available therapy in the treatment of advanced systemic mastocytosis. Leukemia. 2022 Aug;36(8):2108-2120. doi: 10.1038/s41375-022-01615-z. Epub 2022 Jul 5.
- Individual participant dataSharing planned: No. IPD will not be shared
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
6 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Advanced Systemic Mastocytosis and Aggressive Systemic Mastocytosis, run by Blueprint Medicines Corporation at 6 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 0 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Jan 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States, Austria, Germany, Spain, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04695431, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Jan 2022.