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Clinical Trials and Novel Therapies Study Assessing the Efficacy, Safety and Pharmacokinetics of Alpelisib in Pediatric and Adult Patients with PIK3CA-related Overgrowth Spectrum (PROS)

<p>Study Assessing the Efficacy, Safety and Pharmacokinetics of Alpelisib in Pediatric and Adult Patients with PIK3CA-related Overgrowth Spectrum (PROS)</p>

Condition

PIK3CA-related Overgrowth Spectrum (PROS)

Description

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).

Eligibility

  • Age 2 years and older
  • PIK3CA-related Overgrowth Spectrum (PROS)
  • Symptomatic and progressive overgrowth
  • Documented PIK3CA mutation
  • At least one measurable PROS-related lesion (2 cm or larger)
  • Performance status Karnofsky/Lansky 50 or higher
  • Adequate bone marrow and organ function

Detailed inclusion and exclusion criteria are listed at clinicaltrials.gov.

Contacts

Netta Schneller, MD

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Ionela Iacobas, MD

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