Study Assessing the Efficacy, Safety and Pharmacokinetics of Alpelisib in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum (PROS)
Novartis Pharmaceuticals
Summary
This study is designed to demonstrate the efficacy and assess safety and tolerability of oral daily alpelisib in participants with PIK3CA-related overgrowth spectrum (PROS).
Description
The study consists of a screening period of up to 42 days, a core period of 48 weeks and an extension period of up to 2 years to assess the efficacy, safety and pharmacokinetic (PK) of alpelisib in pediatric and adult participants with PROS. Screening Period: Potential participants will be assessed for eligibility and undergo a whole body MRI scan to evaluate PROS-related lesions. Only those who meet all inclusion criteria will be eligible for randomization. Core Period: Baseline is defined as the last available evaluation prior to the first dose of study treatment. Participants in Group 1 a…
Eligibility
- Age range
- 2–100 years
- Sex
- All
- Healthy volunteers
- No
Key Inclusion Criteria: 1. Male or female participants aged ≥2 years at the time of informed consent/assent. 2. Participants with diagnosis of PROS (according to Clinical Diagnostic Criteria for PROS proposed by Keppler Noreuil et al 2014) with symptomatic AND progressive overgrowth, who have syndromic disease or isolated features (with the exception of isolated macrodactyly, macrocephaly or epidermal nevus) at the time of informed consent/assent. 3. Documented evidence of a somatic mutation(s) in the PIK3CA gene performed in local laboratories using a DNA-based test AND available archival ti…
Interventions
- DrugAlpelisib
Oral Film-Coated Tablet (FCT): * Group 1: 250 mg once daily * Group 2, 6 to \<18 years: 125 mg once daily (starting dose) Granules: • Group 2, 2 to \<6 years: 50 mg once daily (starting dose)
Locations (30)
- Washington UniversitySt Louis, Missouri
- Cinn Children Hosp Medical CenterCincinnati, Ohio
- Novartis Investigative SiteSydney, New South Wales
- Novartis Investigative SiteSydney, New South Wales
- Novartis Investigative SiteNorth Adelaide, South Australia
- Novartis Investigative SiteSalzburg