Actively Recruiting

Phase 2
Age: 2Years - 40Years
All Genders
NCT05577754

Assessment of the Efficacy and Safety of Alpelisib (BYL719) in Pediatric and Adult Patients With Megalencephaly-CApillary Malformation Polymicrogyria Syndrome (MCAP)

Led by Centre Hospitalier Universitaire Dijon · Updated on 2026-02-24

20

Participants Needed

13

Research Sites

222 weeks

Total Duration

On this page

Sponsors

C

Centre Hospitalier Universitaire Dijon

Lead Sponsor

N

Novartis Pharmaceuticals

Collaborating Sponsor

AI-Summary

What this Trial Is About

This study is a two periods multi-center Phase II trial, with a 6 months double-blind, placebo-controlled period followed by open label period, to assess the efficacy and safety of alpelisib (BYL719) in pediatric and adult patients with Megalencephaly-CApillary malformation Polymicrogyria syndrome (MCAP)

CONDITIONS

Official Title

Assessment of the Efficacy and Safety of Alpelisib (BYL719) in Pediatric and Adult Patients With Megalencephaly-CApillary Malformation Polymicrogyria Syndrome (MCAP)

Who Can Participate

Age: 2Years - 40Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Signed informed consent and assent as applicable before any study procedures
  • Male or female patients aged 2 to 40 years at consent
  • Diagnosis of MCAP with neurodevelopmental disorder symptoms
  • Confirmed mutation in the PIK3CA gene using validated DNA-based testing
  • Adequate bone marrow and organ function as defined by specific blood counts and biochemistry
  • Ability to swallow study drug appropriate to age (tablets, suspension, or granules)
  • Negative pregnancy test for women of childbearing potential
  • Male patients with pregnant or potentially pregnant partners agree to use condoms during study and one week after
  • Optional signed consent for lumbar puncture for exploratory study
Not Eligible

You will not qualify if you...

  • Previous treatment with alpelisib
  • Significant gastrointestinal issues affecting drug absorption
  • Uncontrolled diabetes mellitus
  • Hypersensitivity to PI3K inhibitors or alpelisib components
  • Other severe or uncontrolled medical conditions that contraindicate alpelisib use
  • Participants unwilling to use effective contraception or abstinence as required
  • Use of mTOR or PI3K-AKT pathway inhibitors within one month before study
  • History or current malignancy within 5 years except certain treated cancers
  • Use of strong CYP3A4 inducers or BCRP inhibitors that cannot be stopped before screening
  • Major surgery within 3 months before consent
  • History of severe skin reactions like Stevens-Johnson syndrome
  • Pneumonitis or interstitial lung disease with impaired lung function for participants 6 years and older
  • Suspicious or documented pneumonitis in participants 2 to 5 years old
  • History of acute or chronic pancreatitis
  • Clinically significant heart disease including heart failure, arrhythmias, long QT syndrome, or low creatinine clearance
  • Currently enrolled in another interventional trial or not affiliated with national health insurance
  • Inability to provide consent or attend all trial visits
  • Contraindications to lumbar puncture for those consenting to the optional procedure

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 13 locations

1

CHU Amiens

Amiens, France, 80054 Amiens

Actively Recruiting

2

CHU d'Angers

Angers, France, 49933

Actively Recruiting

3

CHRU Brest

Brest, France, 29200

Not Yet Recruiting

4

HCL - Groupement Hospitalier Est Hôpital Femme-Mère-Enfant

Bron, France, 69677

Actively Recruiting

5

Chu Estaing

Clermont-Ferrand, France, 63003

Actively Recruiting

6

Chu Dijon Bourgogne

Dijon, France, 21000

Actively Recruiting

7

CHU Dijon Bourgogne - CIC-P

Dijon, France, 21079

Active, Not Recruiting

8

CHU de Lille

Lille, France, 59037

Actively Recruiting

9

CHRU Nîmes

Nîmes, France, 30029

Actively Recruiting

10

AP-HP Hôpital Necker-Enfants Malades - CIC

Paris, France, 75015

Active, Not Recruiting

11

AP-HP Hôpital Necker-Enfants Malades

Paris, France, 75015

Actively Recruiting

12

CHU Rennes

Rennes, France, 35023

Actively Recruiting

13

CHRU Tours

Tours, France, 37044

Actively Recruiting

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Research Team

L

Laurence OLIVIER-FAIVRE

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

DOUBLE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

2

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Assessment of the Efficacy and Safety of Alpelisib (BYL719) in Pediatric and Adult Patients With Megalencephaly-CApillary Malformation Polymicrogyria Syndrome (MCAP) | DecenTrialz