CHOP Phase 2 Alpelisib Trial for Vascular Anomalies
Summary
NIH ClinicalTrials.gov registers a Phase 2 single-site trial at Children's Hospital of Philadelphia (Philadelphia Campus) evaluating Alpelisib (BYL719) in patients aged 2 months to 30 years with TIE2/PIK3CA pathway-driven vascular anomalies. The study will enroll approximately 24 participants, who must complete up to 16 clinic visits over a participation period of up to 3 years, with at least 2 years of continuous study-drug administration. Alpelisib is FDA-approved for certain breast-cancer indications but is used experimentally here, as no VA indication has received regulatory approval.
“Alpelisib has been approved by the U.S. Food and Drug Administration (FDA) for treating adults and children with certain types of breast cancer.”
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ClinicalTrials.gov is the NIH-run registry of every clinical trial conducted in the United States, plus most international trials sponsored by US-based companies or institutions. By federal law, sponsors must register Phase 2 through Phase 4 studies before enrolling patients and post results within a year of completion. This feed tracks every new trial registration and study update, around 700 a month: drug interventions, device studies, behavioral protocols, observational research. Watch this if you scout drug candidates moving into mid or late-stage development, monitor competitor pipelines, or follow rare disease research where new trials signal patient hope. GovPing parses sponsor, phase, intervention, and target indication on each entry.
What changed
NIH ClinicalTrials.gov registered a new Phase 2 interventional trial (NCT07543822) at CHOP's Philadelphia Campus. The study assesses Alpelisib (BYL719) for TIE2/PIK3CA pathway-driven vascular anomalies in participants aged 2 months to 30 years. The experimental drug is already FDA-approved for certain breast cancers but is unapproved for vascular anomalies. Participation lasts up to 3 years with at least 16 clinic visits and at least 2 years of drug administration.
Pharmaceutical companies sponsoring similar PI3K-inhibitor trials and clinical investigators running pediatric or rare-disease studies should note the inclusion age range (2 months through 30 years) and the optional biomarker blood-collection component. Healthcare providers treating vascular-anomaly patients may refer potential participants to the trial site. This registry entry does not create compliance obligations but signals ongoing experimental investigation in a pathway-linked VA population.
Archived snapshot
Apr 23, 2026GovPing captured this document from the original source. If the source has since changed or been removed, this is the text as it existed at that time.
24VA022; VATCH Alpelisib for TIE2/PIK3CA Pathway VAs
Phase 2 NCT07543822 Kind: PHASE2 Apr 22, 2026
Abstract
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: alpelisib (BYL719)
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