NCT05577754 - Assessment of the Efficacy and Safety of Alpelisib (BYL719) in Pediatric and Adult Patients With Megalencephaly-CApillary Malformation Polymicrogyria Syndrome (MCAP) | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

November 28, 2022

Primary Completion Date

March 31, 2027

Study Completion Date

March 31, 2027

Conditions

Megalencephaly-capillary Malformation Polymicrogyria Syndrome (MCAP)

Interventions

DRUG

Alpelisib (BYL719)

Administration during open label and double-blind period of group B: alpelisib will be taken once a day each day over 24 mois During open label period of group A: alpelisib will be taken once a day each day over 24 months

DRUG

Matching placebo

During double-blind period of group A: matching placebo will be taken once a day each day over 6 months

PROCEDURE

Optional lumbar puncture + blood sample

Between 6 and 24 months of treatment with Alpelisib

Trial Locations (13)

21000

RECRUITING

Chu Dijon Bourgogne, Dijon

21079

ACTIVE_NOT_RECRUITING

CHU Dijon Bourgogne - CIC-P, Dijon

29200

NOT_YET_RECRUITING

CHRU Brest, Brest

30029

RECRUITING

CHRU Nîmes, Nîmes

35023

RECRUITING

CHU Rennes, Rennes

37044

RECRUITING

CHRU Tours, Tours

49933

RECRUITING

CHU d'Angers, Angers

59037

RECRUITING

CHU de Lille, Lille

63003

RECRUITING

Chu Estaing, Clermont-Ferrand

69677

RECRUITING

HCL - Groupement Hospitalier Est Hôpital Femme-Mère-Enfant, Bron

75015

ACTIVE_NOT_RECRUITING

AP-HP Hôpital Necker-Enfants Malades - CIC, Paris

RECRUITING

AP-HP Hôpital Necker-Enfants Malades, Paris

80054 Amiens

RECRUITING

CHU Amiens, Amiens