24VA022; VATCH Alpelisib for TIE2/PIK3CA Pathway VAs

Conditions

• TIE2/PIK3CA Pathway Driven Vascular Anomalies
• Vascular Anomalies
• Vascular Anomaly

Eligibility Criteria

Sex

ALL

Age

2 Years - 30 Years

Healthy Volunteers

Not accepted

Interventions

• alpelisib (BYL719) — DRUG
  Subjects will receive oral alpelisib daily in continuous 28-day cycles. Patients aged 18 years and older will start at 125 mg/day with a maximum dose of 250 mg/day; patients aged 6 - 17 years will start at 50 mg/day with a maximum dose of 200 mg/day. A single dose reduction will be permitted in individual subjects who experience toxicity while still having evidence of clinical benefit and is assessed per the investigator.

Study Details

The study will enroll participants 2 months of age up to 30 years of age. The purpose of this study is to assess the effectiveness and safety of Alpelisib (the "Study Drug") in patients with PIK3CA/TIE-2/TEK pathway driven vascular anomalies (VA). Alpelisib has been approved by the U.S. Food and Drug Administration (FDA) for treating adults and children with certain types of breast cancer. Its use in this study is considered experimental because FDA has not approved the study drug for treating people with VAs. Study participation will last for up to 3 years and will involve regular study visits to Children's Hospital of Philadelphia (CHOP)'s Philadelphia Campus. Participants will need to take the study drug Alpelisib for at least 2 years, or up to 3 years in total if there is a positive response. Participating in this research means you will attend up to 16 clinic visits for the purposes of the study. Most visits will take approximately 30 minutes, but some visits will take approximately 2 hours, because you will be asked to complete questionnaires about your experience. Participating in this research also means taking the study drug, having pictures taken, and completing study drug diaries. There is also an optional portion to this study that involves collecting blood for biomarker testing.

Key Dates

Start date

Aug 27, 2025

Status verified

Apr 2026

Primary completion

Aug 27, 2029

Completion

Aug 27, 2030

Study Design

Enrollment

61 participants (estimated)

Allocation

NA

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• Experimental: Main Study
  This arm will determine the proportion of subjects with an objective beneficial response to alpelisib at the end of cycle 6 using an individualized response criterion based on radiologic assessment, Patient Reported Outcomes (PROs) and Clinical Benefit Assessment (CBA). It will also determine the safety of oral trametinib in children and young adults with PIK3CA/TIE-2/TEK pathway driven vascular anomalies through various laboratory testing and clinical observations.

Primary Outcome Measure

Percentage of subjects with a Substantial Response or Intermediate Response to Alpelisib using an Individualized Response Criteria [ Time Frame: Individualized Response Criteria will be evaluated after 6 cycles (each cycle is 28 days) and then every 6 months until the end of the study (approximately 1.5 years) ]

Locations (1)

Find similar trials in Philadelphia, PA

Related Studies

• Imaging Lymphatic Function in Normal Subjects and in Persons With Lymphatic Disorders
  Enrolling By Invitation · The University of Texas Health Science Center, Houston · Houston, Texas
• Identification of Biomarkers for Patients with Vascular Anomalies
  Recruiting · Children's Hospital Medical Center, Cincinnati · Boston, Massachusetts