24VA022; VATCH Alpelisib for TIE2/PIK3CA Pathway VAs

Summary

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.

Conditions

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

Interventions

DRUG

alpelisib (BYL719)

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.

Sponsors & Collaborators

• Novartis

  collaborator INDUSTRY

• CaNVAS (Consortium of Investigators of Vascular Anomalies)

  collaborator UNKNOWN

• Texas Children's

  collaborator UNKNOWN

• Arkansas Children's Hospital Research Institute

  collaborator OTHER

• Nationwide Children's Hospital

  collaborator OTHER

• Nemours Children's Health

  collaborator UNKNOWN

• Boston Children's Hospital

  collaborator OTHER

• NYU Langone Health

  collaborator OTHER

• Children's Healthcare of Atlanta

  collaborator OTHER

• Oregon Health and Science University

  collaborator OTHER

• Children's Hospital Colorado

  collaborator OTHER

• Riley Children's Health

  collaborator UNKNOWN

• Children's Hospital of Los Angeles (CHLA)

  collaborator UNKNOWN

• Seattle Children's Hospital

  collaborator OTHER

• Children's Hospital of Orange County (CHOC)

  collaborator UNKNOWN

• Children's Wisconsin - Milwaukee

  collaborator UNKNOWN

• University of North Carolina

  collaborator OTHER

• Children's Hospital Medical Center, Cincinnati

  collaborator OTHER

• University of California, San Francisco

  collaborator OTHER

• Columbia University Irving Medical Center, New York, NY

  collaborator UNKNOWN

• Children's Hospital of Pittsburg

  collaborator UNKNOWN

• Dell Children's Medical Center of Central Texas

  collaborator OTHER

• Vanderbilt University Medical Center

  collaborator OTHER

• Helen DeVos Children's Hospital

  collaborator OTHER

• Washington University School of Medicine

  collaborator OTHER

• Lucile Packard Children's Hospital Stanford

  collaborator UNKNOWN

• Children's Hospital of Philadelphia

  lead OTHER

Study Design

Allocation

NA

Purpose

TREATMENT

Masking

NONE

Model

SEQUENTIAL

Eligibility

Min Age

2 Years

Max Age

30 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2025-08-27

Primary Completion

2029-08-27

Completion

2030-08-27

FDA Drug

Yes

Countries

• United States

Study Locations