Phase 2: testing Alpelisib for PIK3CA-related Overgrowth Spectrum (PROS)
This study is designed to demonstrate the how well it works and assess whether it is safe and how hard side effects are to live with of oral daily alpelisib in participants with PIK3CA-related overgrowth spectrum (PROS).
Official registry: https://clinicaltrials.gov/study/NCT06997588 · Novartis Pharmaceuticals · enrollment listed as 140
What this phase means
Phase 2: researchers already know a roughly safe dose (from phase 1). This stage asks whether the treatment actually helps the condition, and watches side effects more closely.
Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.
What the registry says happens
If you join, the listed intervention(s) are Alpelisib. The registry lists allocation as na. This study is designed to demonstrate the how well it works and assess whether it is safe and how hard side effects are to live with of oral daily alpelisib in participants with PIK3CA-related overgrowth spectrum (PROS).
They plan to enroll about 140 people at 40 sites. Taking part typically means taking a study drug (or dummy pill) plus check-ins.
Official title (unrewritten)
Study Assessing the Efficacy, Safety and Pharmacokinetics of Alpelisib in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum (PROS)
Interventions: Alpelisib
Primary outcome (registry wording): Proportion of participants with a confirmed objective response by BIRC
Who it is for (from inclusion criteria)
- Ages 2 Years to 100 Years
- Karnofsky (in participants \>16 years of age at study entry) or Lansky (≤16 years of age at study entry) performance status index ≥50
- PGI-S score of mild, moderate, severe, or very severe at screening
- Adequate bone marrow and organ function
- Presence of at least 1 PROS-related measurable lesion (longest diameter ≥2 cm) confirmed by BIRC assessment and associated with complaints, clinical symptoms or functional limitations affecting the participant's everyday life
Who the registry excludes
- Clinically meaningful bleeding from PROS-related lesion (Grade 2 and more as per CTCAE v4.03) within 15 days before study treatment initiation
Public study contact (from the registry)
Novartis Pharmaceuticals
Published on ClinicalTrials.gov. Contact them as a possible participant. They decide eligibility.
Sites listed on ClinicalTrials.gov
40 listed- Univ of California at Sacramento — Sacramento, California (recruiting)
- Childrens National Medical Center — Washington D.C., District of Columbia (recruiting)
- Washington University — St Louis, Missouri (recruiting)
- University of North Carolina at Chapel Hill — Chapel Hill, North Carolina (recruiting)
- Cinn Children Hosp Medical Center — Cincinnati, Ohio (recruiting)
- Nationwide Children s Hospital — Columbus, Ohio (recruiting)
- Oregon Health Sciences University — Portland, Oregon (recruiting)
- Childrens Hosp Pittsburgh UPMC — Pittsburgh, Pennsylvania (recruiting)
- Baylor College Of Medicine — Houston, Texas (recruiting)
- UT Health Science Center — Houston, Texas (recruiting)
- Childrens Hospital and Regional Medical Center — Seattle, Washington (recruiting)
+ 29 sites outside the US on the same record
FAQ about this NCT record
Is NCT06997588 still recruiting?
The sponsor posted status “Recruiting” on ClinicalTrials.gov at last ingest. Sites can pause independently. Confirm with the official record and the listed contact.
What phase is NCT06997588?
Phase 2. Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.
Can PlainTrials tell me if I qualify for NCT06997588?
No. The inclusion and exclusion lists on this page are readable extracts of the public eligibility block. Only the study team can screen you.
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