24VA022; VATCH Alpelisib for TIE2/PIK3CA Pathway VAs (NCT07543822) | Clinical Trial Compass
Active — Not RecruitingPhase 2
24VA022; VATCH Alpelisib for TIE2/PIK3CA Pathway VAs
United States61 participantsStarted 2025-08-27
Plain-language 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.
Who can participate
Age range2 Years – 30 Years
SexALL
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Inclusion criteria
✓. Signed informed consent and assent (when applicable)
✓. Males or females age ≥ 2 years to \<=30 years at the time of informed consent.
✓. Have a documented PIK3CA or somatic TIE2/TEK variant.
✓. Have a symptomatic vascular anomaly in need of medical therapy.
✓. Have a disease-related lesion or lesions which can be measured
✓. Have a Lansky or Karnofsky performance status score of ≥ 50.
✓. Have acceptable organ function
✓. (For persons who can get pregnant) Have a negative serum or urine pregnancy test within 7 days prior to starting study medication.
Exclusion criteria
✕. Subject with only epidermal nevus/nevi in the absence of other PIK3CA vascular anomaly/overgrowth
✕. Debulking or other major surgery performed within 30 days, at time of informed consent
What they're measuring
1
Percentage of subjects with a Substantial Response or Intermediate Response to Alpelisib using an Individualized Response Criteria
Timeframe: 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)
2
Relative size of target lesion(s) as determined by radiologic assessment
Timeframe: Measured at screening and after cycles 6, 12, and 24. Each cycle is 28 days.
3
Quality of life as evaluated by PROMIS Patient Reported Outcome (PRO) questionnaire T-scores
Timeframe: Begin at screening and after cycles 6, 12, and 24. If subject enters extension phase, will continue to measure every 6 cycles until end of therapy (approximately 1 year). Each cycle is 28 days.
✕. Clinically meaningful bleeding related to VA: Grade 2 within 14 days or Grade 3 and more within 28 days before study treatment start as per CTCAE v. 5.0
✕. Sclerotherapy/embolization for vascular complications performed within 14 days before informed consent.
✕. Prior medications that are not allowable per the study protocol
✕. Participation in any other prior investigational study within 4 weeks prior to study treatment start and within 5 half-lives of the investigational product, whichever is longer
✕. Supportive care use of anticoagulants and compression garments is allowed. Subjects must have been using these interventions in an unchanged manner for 30 days before study enrollment.
✕. History of prior and or ongoing malignancy, or ongoing investigations or treatment for malignancy at time of informed consent.