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NCT06997588 · ClinicalTrials.gov registry record · Phase 2
EPIK-P4: A Phase II Single-arm Study to Assess the Efficacy, Safety and Pharmacokinetics of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum (PROS)
A Phase 2 study of PIK3CA-related Overgrowth Spectrum (PROS), sponsored by Novartis Pharmaceuticals.
- Recruiting
- Registry status
- Phase 2
- Development phase
- 104
- Enrollment target
- 20
- Study locations
NCT06997588: Recruiting Phase 2 study of PIK3CA-related Overgrowth Spectrum (PROS), sponsored by Novartis Pharmaceuticals.
NCT06997588 is a Phase 2 study of PIK3CA-related Overgrowth Spectrum (PROS) that is actively recruiting participants, run by Novartis Pharmaceuticals. The registered enrollment target is 104 participants, below the 175-participant average among 3 other PIK3CA-related Overgrowth Spectrum (PROS) trials with a reported enrollment target (41% lower). The trial reports 20 study locations across 10 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06997588, a Phase 2 study of PIK3CA-related Overgrowth Spectrum (PROS), is actively recruiting participants, sponsored by Novartis Pharmaceuticals.
- RECRUITING
- Registry status
- Phase 2
- Development phase
- 104 participants
- Enrollment target
- 20
- Study locations
Study 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).
Primary Outcome
Confirmed objective response is defined as achieving radiological response, confirmed by a subsequent assessment performed at least after 4 weeks. The achievement of radiological response requires ≥20% reduction from baseline in the sum of target lesion volumes (1 to 3 target lesions, assessed by Magnetic Resonance Imaging (MRI) by a blinded independent review committee (BIRC)), provided that none of the individual target lesions has ≥20% increase from nadir, and in absence of progression of non
Conditions Studied
Interventions
- DRUG Alpelisib
Study Locations (20)
Other
- Novartis Investigative Site - Salzburg
- Novartis Investigative Site - Vienna
- Novartis Investigative Site - Ghent
- Novartis Investigative Site - Angers
- Novartis Investigative Site - Bron
- Novartis Investigative Site - Dijon
- Novartis Investigative Site - Montpellier
- Novartis Investigative Site - Paris
- Novartis Investigative Site - Tours
- Novartis Investigative Site - Berlin
New South Wales
- Novartis Investigative Site - Sydney
- Novartis Investigative Site - Sydney
Missouri
- Washington University - St Louis
South Australia
- Novartis Investigative Site - North Adelaide
Baden-Wurttemberg
- Novartis Investigative Site - Freiburg im Breisgau
North Rhine-Westphalia
- Novartis Investigative Site - Düsseldorf
Saxony
- Novartis Investigative Site - Leipzig
Saxony-Anhalt
- Novartis Investigative Site - Halle
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 104 participants |
| Start Date | 2025-10-09 |
| Est. Completion | 2030-09-30 |
| Phase | Phase 2 |
What NCT06997588 shows while recruiting
NCT06997588 is an interventional study that assigns participants to a tested intervention. The registered 104 participants enrollment target is mid-sized for trials with a published cap, below the 175-participant average among 3 other PIK3CA-related Overgrowth Spectrum (PROS) trials with a reported enrollment target (41% lower).
The record links to 1 condition, with PIK3CA-related Overgrowth Spectrum (PROS) appearing as the primary indexed condition, and to 1 intervention - of which Alpelisib is the first listed.
NCT06997588 names 20 study sites across 10 states, led by Other, New South Wales, Missouri.
Frequently Asked Questions
What is clinical trial NCT06997588 about?
NCT06997588 is a clinical study titled "EPIK-P4: A Phase II Single-arm Study to Assess the Efficacy, Safety and Pharmacokinetics of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum (PROS)". 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).
What is the current status of trial NCT06997588?
This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 104 participants. The study started on 2025-10-09. Estimated completion is 2030-09-30.
What conditions does trial NCT06997588 study?
This clinical trial studies the following conditions: PIK3CA-related Overgrowth Spectrum (PROS).
What interventions are being tested in trial NCT06997588?
The interventions under investigation include: Alpelisib (DRUG).
Who is sponsoring clinical trial NCT06997588?
This trial is sponsored by Novartis Pharmaceuticals, which has 1,375 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06997588 being conducted?
This trial has 20 study locations across Missouri, New South Wales, South Australia, Baden-Wurttemberg, North Rhine-Westphalia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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