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NCT04589650 · ClinicalTrials.gov registry record · Phase 2
Study Assessing the Efficacy, Safety and PK of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum
A Phase 2 study of PIK3CA-related Overgrowth Spectrum (PROS), sponsored by Novartis Pharmaceuticals.
- Active
- Registry status
- Phase 2
- Development phase
- 206
- Enrollment target
- 20
- Study locations
NCT04589650: Active Phase 2 study of PIK3CA-related Overgrowth Spectrum (PROS), sponsored by Novartis Pharmaceuticals.
NCT04589650 is a Phase 2 study of PIK3CA-related Overgrowth Spectrum (PROS) that is active but no longer recruiting, run by Novartis Pharmaceuticals. The registered enrollment target is 206 participants, above the 141-participant average among 3 other PIK3CA-related Overgrowth Spectrum (PROS) trials with a reported enrollment target (46% higher). The trial reports 20 study locations across 12 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04589650, a Phase 2 study of PIK3CA-related Overgrowth Spectrum (PROS), is active but no longer recruiting, sponsored by Novartis Pharmaceuticals.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 2
- Development phase
- 206 participants
- Enrollment target
- 20
- Study locations
Study Summary
This is a prospective Phase II multi-center study with an initial 16-week, randomized, double-blind, placebo-controlled period, followed by two extension periods to assess the efficacy, safety and pharmacokinetics (PK) of alpelisib in pediatric and adult patients with PIK3CA-related overgrowth spectrum (PROS)
Primary Outcome
A responder is defined by achieving a \>=20% reduction from baseline in the sum of target lesion volumes (via BIRC), provided that none of the individual target lesions have a \>=20% increase from baseline and in absence of progression of non target lesions and without new lesions. Confirmation of response requires a subsequent imaging assessment performed at least 4 weeks after the onset of response. Participants who permanently discontinued alpelisib prior to confirmation of response, and part
Conditions Studied
Interventions
- DRUG Placebo
- DRUG Alpelisib
Study Locations (20)
Other
- Novartis Investigative Site - Beijing
- Novartis Investigative Site - Shanghai
- Novartis Investigative Site - Bordeaux
- Novartis Investigative Site - Dijon
- Novartis Investigative Site - Paris
- Novartis Investigative Site - Tours
Ohio
- Cincinnati Children s Hospital Medical Center - Cincinnati
- Cinn Children Hosp Medical Center - Cincinnati
Texas
- Unv of TX Southwestern Medical Center - Dallas
- Baylor College Of Medicine - Houston
Quebec
- Novartis Investigative Site - Montreal
- Novartis Investigative Site - Montreal
California
- UCSF Birthmarks and Vascular Center - San Francisco
Colorado
- Childrens Hospital Colorado - Aurora
Missouri
- Washington Univ School Of Medicine - St Louis
New York
- Fink Childrens Ambulatory Care Ctr - New York
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 206 participants |
| Start Date | 2021-04-19 |
| Est. Completion | 2031-01-09 |
| Phase | Phase 2 |
What the registry record for NCT04589650 still lists
NCT04589650 is an interventional study that assigns participants to a tested intervention. The registered 206 participants enrollment target is mid-sized for trials with a published cap, above the 141-participant average among 3 other PIK3CA-related Overgrowth Spectrum (PROS) trials with a reported enrollment target (46% higher).
The record links to 1 condition, with PIK3CA-related Overgrowth Spectrum (PROS) appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT04589650 names 20 study sites across 12 states, led by Other, Ohio, Texas.
Frequently Asked Questions
What is clinical trial NCT04589650 about?
NCT04589650 is a clinical study titled "Study Assessing the Efficacy, Safety and PK of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum". This is a prospective Phase II multi-center study with an initial 16-week, randomized, double-blind, placebo-controlled period, followed by two extension periods to assess the efficacy, safety and pharmacokinetics (PK) of alpelisib in pediatric and adult patients with PIK3CA-related overgrowth spect...
What is the current status of trial NCT04589650?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 206 participants. The study started on 2021-04-19. Estimated completion is 2031-01-09.
What conditions does trial NCT04589650 study?
This clinical trial studies the following conditions: PIK3CA-related Overgrowth Spectrum (PROS).
What interventions are being tested in trial NCT04589650?
The interventions under investigation include: Placebo (DRUG), Alpelisib (DRUG).
Who is sponsoring clinical trial NCT04589650?
This trial is sponsored by Novartis Pharmaceuticals, which has 1,375 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04589650 being conducted?
This trial has 20 study locations across California, Colorado, Missouri, New York, North Carolina. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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