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

> **NCT04589650** · PHASE2 · ACTIVE_NOT_RECRUITING · sponsor: **Novartis Pharmaceuticals** · enrollment: 206 (actual)

## Conditions studied

- PIK3CA-related Overgrowth Spectrum (PROS)

## Interventions

- **DRUG:** Alpelisib
- **DRUG:** Placebo

## Key facts

- **NCT ID:** NCT04589650
- **Lead sponsor:** Novartis Pharmaceuticals
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** ACTIVE_NOT_RECRUITING
- **Start date:** 2021-04-19
- **Primary completion:** 2024-03-20
- **Final completion:** 2031-01-09
- **Target enrollment:** 206 (ACTUAL)
- **Last updated:** 2026-04-30


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT04589650

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT04589650, "Study Assessing the Efficacy, Safety and PK of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum". Retrieved via AI Analytics 2026-05-27 from https://api.ai-analytics.org/clinical/NCT04589650. Licensed CC0.

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*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*