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Could a cancer drug help kids with rare brain disorder?

NCT ID NCT05577754

Recruiting now Disease control Sponsor: Centre Hospitalier Universitaire Dijon Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 2 trial tests alpelisib, a drug originally developed for cancer, in 20 people aged 2 to 40 with MCAP syndrome—a rare condition causing an enlarged brain, blood vessel issues, and developmental delays. Participants receive either alpelisib or a placebo for 6 months, then all get the drug for up to 24 months. The goal is to see if it improves daily living skills like communication and socialization.

What this could mean

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

Active substance
alpelisib (BYL719)
What this could lead to
If it works, this could point toward a treatment that improves daily living skills and brain function in people with MCAP syndrome.
What could go wrong
This is a small, early-phase trial with only 20 participants, so results may not apply to everyone. The drug may cause side effects or fail to show benefit over placebo.

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.

hereditary sensory and autonomic neuropathy megalencephaly-capillary malformation-polymicrogyria syndrome neurodevelopmental disorder

As listed by the trial registrant

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

MEGALENCEPHALY-CAPILLARY MALFORMATION POLYMICROGYRIA SYNDROME (MCAP)

Contacts and locations

Locations

  • AP-HP Hôpital Necker-Enfants Malades

    RECRUITING

    Paris, 75015, France

  • AP-HP Hôpital Necker-Enfants Malades - CIC

    ACTIVE_NOT_RECRUITING

    Paris, 75015, France

  • CHRU Brest

    NOT_YET_RECRUITING

    Brest, 29200, France

  • CHRU Nîmes

    RECRUITING

    Nîmes, 30029, France

  • CHRU Tours

    RECRUITING

    Tours, 37044, France

  • CHU Amiens

    RECRUITING

    Amiens, 80054 Amiens, France

  • CHU Dijon Bourgogne - CIC-P

    ACTIVE_NOT_RECRUITING

    Dijon, 21079, France

  • CHU Rennes

    RECRUITING

    Rennes, 35023, France

  • CHU d'Angers

    RECRUITING

    Angers, 49933, France

  • CHU de Lille

    RECRUITING

    Lille, 59037, France

  • Chu Dijon Bourgogne

    RECRUITING

    Dijon, 21000, France

  • Chu Estaing

    RECRUITING

    Clermont-Ferrand, 63003, France

  • HCL - Groupement Hospitalier Est Hôpital Femme-Mère-Enfant

    RECRUITING

    Bron, 69677, France

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