NCT04589650 - Study Assessing the Efficacy, Safety and PK of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum | Biotech Hunter

Enrollment

205

Participants

Timeline

Start Date

April 19, 2021

Primary Completion Date

March 20, 2024

Study Completion Date

February 28, 2031

Conditions

PIK3CA-related Overgrowth Spectrum (PROS)

Interventions

DRUG

Alpelisib

Adult participants (group 1) will receive 125 mg of alpelisib oral tablets once daily. Pediatric participants (Group 2: 6 to 17 years old) will receive 50 mg of alpelisib oral tablets once daily. Pediatric participants (Group 4: 2 to 5 years old) will receive 50 mg of alpelisib oral tablets once daily, Pediatric participants (Group 3: 0 to 5 years old) will receive alpelisib granules formulation with an age-dependent starting dose and maximum dose levels ranging from 20 mg every other day to 50 mg once daily. Pediatric participants (Group 5: 6 to 17 years old) will receive 125 mg of alpelisib oral tablets once daily.

DRUG

Placebo

Participants will receive matching placebo once daily up to week 16.

Trial Locations (36)

3010

Novartis Investigative Site, Bern

10016

NYU Langone Health, New York

10126

Novartis Investigative Site, Torino

19104

CHOP Abramson Pediatric Resch Ctr, Philadelphia

21034

Novartis Investigative Site, Dijon

22149

Novartis Investigative Site, Hamburg

27599

UNC Chapel Hill, Chapel Hill

28046

Novartis Investigative Site, Madrid

33076

Novartis Investigative Site, Bordeaux

37044

Novartis Investigative Site, Tours

40225

Novartis Investigative Site, Düsseldorf

63110

Washington Univ School Of Medicine, St Louis

69120

Novartis Investigative Site, Heidelberg

75015

Novartis Investigative Site, Paris

75235

University of Texas Southwestern Medical Center, Dallas

77030

Baylor College Of Medicine, Houston

79106

Novartis Investigative Site, Freiburg im Breisgau

80045

Childrens Hospital Colorado, Aurora

94158

UCSF Birthmarks and Vascular Center, San Francisco

98105

Childrens Hospital and Regional Medical Center, Seattle

100730

Novartis Investigative Site, Beijing

200011

Novartis Investigative Site, Shanghai

999077

Novartis Investigative Site, Pokfulam

45229-3039

Cincinnati Children s Hospital Medical Center, Cincinnati

Cinn Children Hosp Medical Center, Cincinnati

H2W 1T8

Novartis Investigative Site, Montreal

H3T 1C5

Novartis Investigative Site, Montreal

04103

Novartis Investigative Site, Leipzig

00165

Novartis Investigative Site, Roma

6500HB

Novartis Investigative Site, Nijmegen

0372

Novartis Investigative Site, Oslo

08950

Novartis Investigative Site, Esplugues de Llobregat

CH - 8032

Novartis Investigative Site, Zurich

B4 6NH

Novartis Investigative Site, West Midlands

SW17 0QT

Novartis Investigative Site, London

M13 9WL

Novartis Investigative Site, Manchester