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Avapritinib rollover trial aims to confirm Long-Term safety for mastocytosis patients

NCT ID NCT06748001

Disease control Sponsor: Blueprint Medicines Corporation Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 2 times

Summary

This study looks at the long-term safety of the drug avapritinib (AYVAKIT®) in people with systemic mastocytosis who have already been taking it and benefiting from it in a previous study. About 60 participants will continue receiving the drug and be monitored for serious side effects. The goal is to see if avapritinib remains safe over time for controlling this rare disease.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
avapritinib (AYVAKIT®)
What this could lead to
If successful, this study could confirm that avapritinib is safe for long-term use in controlling systemic mastocytosis, supporting its continued use as a maintenance therapy.
What could go wrong
This is a small, open-label rollover study with no placebo group, so results may not prove long-term safety definitively. Side effects like swelling, nausea, or cognitive changes remain possible.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 4

Runs after approval, following long-term safety and how well the treatment works in everyday use.

Participants

About 60 people

The number the study aims to enrol. It can still change while the study runs.

Started

Nov 2024

Expected to finish

Dec 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * 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. Exclusion Criteria: * 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.

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Conditions

The condition(s) this trial relates to.

Mastocytosis, Systemic systemic mastocytosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Mastocytosis, systemic

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    7 sites in 6 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • A.O.U. San Giovanni di Dio e Ruggi d'Aragona di Salerno

    RECRUITING

    Salerno, 84131, Italy

  • Erasmus Medisch Centrum

    RECRUITING

    Rotterdam, 3015, Netherlands

  • Guys and St Thomas NHS Foundation Trust - St Thomas Hospital

    RECRUITING

    London, SE19RT, United Kingdom

  • Oslo University Hospital

    RECRUITING

    Oslo, 0424, Norway

  • The Hospital for Sick Children

    RECRUITING

    Toronto, Ontario, M5G 1X8, Canada

  • Universitair Medisch Centrum Groningen

    RECRUITING

    Groningen, 9700, Netherlands

  • Universitair Ziekenhuis Antwerpen (UZA)

    RECRUITING

    Edegem, 2650, Belgium

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