Mastocytosis, SystemicPhase 4NCT06748001
Avapritinib Rollover Study
A Phase 4 study testing Avapritinib in people with Mastocytosis, Systemic, run by Blueprint Medicines Corporation at 7 sites in 6 countries.
Trial summary
The primary objective of the rollover study is to evaluate the long-term safety of avapritinib in participants who have completed a Blueprint Medicines sponsored study (parent study) and continued to benefit from avapritinib. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Mastocytosis, Systemic
- What is being tested?
- Avapritinib
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 7 sitesin 6 countries
- Study dates
- 28 Nov 2024to 31 Dec 2027
- Phase
- Phase 4
- Are there results?
- Not yet: the study is under way
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Participated in a Blueprint Medicines sponsored avapritinib clinical study. The Sponsor reserves the right to not roll over patients who have been on drug hold for an extended time period.
- Completed End of Trial / End of Study (EOT/EOS) visit in the parent study and demonstrated compliance with the parent study requirements.
- Continue to clinically benefit from treatment with avapritinib.
- Able to give written informed consent.
- Agree to continue to use highly effective contraception as defined in this protocol.
- Female patients of childbearing potential must have negative highly sensitive serum pregnancy test within 20 days before the first dose of avapritinib. Women of nonchildbearing potential do not require this test.
Who cannot join
- Participant is participating in another interventional study.
- Participant is unwilling or unable to comply with study procedures and study restrictions.
- Participant is breastfeeding.
- Other protocol-defined criteria apply.
Trial locations and contacts
7 sites in 6 countries. Contact the study team or a site near you to ask about taking part.
7 sites in 6 countries
Netherlands2 sites, 2 recruiting
- Recruiting
Universitair Medisch Centrum Groningen
Groningen, 9700
- Recruiting
Erasmus Medisch Centrum
Rotterdam, 3015
Belgium1 site, 1 recruiting
- Recruiting
Universitair Ziekenhuis Antwerpen (UZA)
Edegem, 2650
Canada1 site, 1 recruiting
- Recruiting
The Hospital for Sick Children
Toronto, Ontario, M5G 1X8
Italy1 site, 1 recruiting
- Recruiting
A.O.U. San Giovanni di Dio e Ruggi d'Aragona di Salerno
Salerno, 84131
United Kingdom1 site, 1 recruiting
- Recruiting
Guys and St Thomas NHS Foundation Trust - St Thomas Hospital
London, SE19RT
About this study
Official title: An Open-label, Multicenter, Rollover Study in Patients Who Participated in an Avapritinib Clinical Study
The primary objective of the rollover study is to evaluate the long-term safety of avapritinib in participants who have completed a Blueprint Medicines sponsored study (parent study) and continued to benefit from avapritinib.
- Conditions
- Mastocytosis, Systemic
- Being tested
- Avapritinib
- Keywords
- Indolent Systemic Mastocytosis, ISM, Advanced Systemic Mastocytosis, AdvSM, Rollover study
- Medical subject headings
- Mastocytosis, Systemic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AvapritinibDrug
Also called BLU-285, AYVAKIT®
Avapritinib will be administered in accordance with the parent study protocol.
| Study group | Role | What they receive |
|---|---|---|
| Avapritinib | Experimental | Participants who have participated in a Blueprint Medicines sponsored study (the parent study) and continue to benefit from avapritinib. Receives: Drug: Avapritinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Serious Adverse Events (SAEs), Adverse Events of Special Interest (AESIs), and AEs leading to Discontinuation
Measured: Up to approximately 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Dec 2024
- Study start28 Nov 2024
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mastocytosis, SystemicStudies 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
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Blueprint Medicines Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06748001
- Sponsor's protocol ID
- BLU-285-2404
- Status last verified
- Feb 2026
- First submitted
- 19 Dec 2024
- Last updated
- 9 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Multicenter, Rollover Study in Patients Who Participated in an Avapritinib Clinical Study
- ClinicalTrials.gov ID
- NCT06748001
- Acronym / sponsor ID
- BLU-285-2404
- Conditions
- Mastocytosis, Systemic
- Keywords
- Indolent Systemic Mastocytosis, ISM, Advanced Systemic Mastocytosis, AdvSM, Rollover study
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Blueprint Medicines CorporationIndustry (pharma, biotech, devices)
- Study start
- 28 Nov 2024 (actual)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 24 Dec 2024 (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.
The primary objective of the rollover study is to evaluate the long-term safety of avapritinib in participants who have completed a Blueprint Medicines sponsored study (parent study) and continued to benefit from avapritinib.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Avapritinib | Experimental | Drug: AvapritinibParticipants who have participated in a Blueprint Medicines sponsored study (the parent study) and continue to benefit from avapritinib. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Participated in a Blueprint Medicines sponsored avapritinib clinical study. The Sponsor reserves the right to not roll over patients who have been on drug hold for an extended time period.
- Completed End of Trial / End of Study (EOT/EOS) visit in the parent study and demonstrated compliance with the parent study requirements.
- Continue to clinically benefit from treatment with avapritinib.
- Able to give written informed consent.
- Agree to continue to use highly effective contraception as defined in this protocol.
Key exclusion criteria (4 listed)
- Participant is participating in another interventional study.
- Participant is unwilling or unable to comply with study procedures and study restrictions.
- Participant is breastfeeding.
- Other protocol-defined criteria apply.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AvapritinibAlso: BLU-285, AYVAKIT®Avapritinib will be administered in accordance with the parent study protocol. | Drug | Avapritinib |
Primary outcome
Incidence of Serious Adverse Events (SAEs), Adverse Events of Special Interest (AESIs), and AEs leading to Discontinuation
Time frame: Up to approximately 4 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Feb 2026.
- Individual participant dataSharing planned: No
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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.
7 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Avapritinib in people with Mastocytosis, Systemic, run by Blueprint Medicines Corporation at 7 sites in 6 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 6 inclusion and 4 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: looking for participants now, as of the sponsor's last update on 9 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in Netherlands, Belgium, Canada, Italy, Norway and 1 more country. 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.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Blueprint Medicines (617-714-6707, MedInfo@blueprintmedicines.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Serious Adverse Events (SAEs), Adverse Events of Special Interest (AESIs), and AEs leading to Discontinuation (time frame: Up to approximately 4 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 9 Feb 2026.