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

NCT ID NCT06748001

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

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.

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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

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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