Vascular Anomalies study: Alpelisib
Official title 24VA022; VATCH Alpelisib for TIE2/PIK3CA Pathway VAs
ClinicalTrials.gov ID: NCT07543822
What this study is testing
What is alpelisib?
an investigational oral medicine being studied as a potential treatment for PIK3CA/TIE-2/TEK pathway driven vascular anomalies is designed to block the PI3Kα enzyme, which can reduce abnormal cell growth signaling
Also referred to as BYL719, Vijoice.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study is testing whether Alpelisib works and is safe for people with PIK3CA/TIE-2/TEK pathway driven vascular anomalies.
- Who it's for
- You may be able to join if you are 2 months to 30 years old and have a vascular anomaly caused by that pathway.
- What's involved
- You would take Alpelisib every day for at least 2 years, up to 3 years. You will attend up to 16 clinic visits, have pictures taken, fill out a drug diary, and answer questionnaires. An optional blood draw may be done for testing.
- Time commitment
- The study lasts up to 3 years with clinic visits that take about 30 minutes to 2 hours each.
- Phase 2: a mid-size study of how well it works
- Time commitment: about 3 years
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 30
You may be able to join if
- 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
You likely can't join if
- 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
- 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.
- 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...
See the full eligibility 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.
- Must agree to the use an acceptable method of contraception
- people that have received alpelisib previously either via Managed Access Program from Novartis or commercial supply are eligible to enroll IF they have been off alpelisib for at least 7 days and there is clinical...
- 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
- 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. people 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.
- Clinically significant heart disease not due to VA
- Documented pneumonitis or interstitial lung disease not due to VA
- History of acute pancreatitis within 1 year before informed consent or past medical history of chronic pancreatitis
- people with an established diagnosis of type I diabetes mellitus or uncontrolled type II diabetes mellitus
- Impaired gastrointestinal (GI) function that may significantly alter the absorption of the study drug (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection)
- History of hypersensitivity to any drugs or metabolites of PI3K inhibitor or any of the excipients of alpelisib
- Known history of Steven Johnson's syndrome, erythema multiform or toxic epidermal necrolysis
- History of seizures or epilepsy on enzyme inducing antiepileptic drug(s); uncontrolled seizures are only allowed if due to PIK3CA variation
- Concurrent severe and/or uncontrolled medical conditions not due to PIK3CA variation. Resolution of symptoms for at least 28 days must have occurred before consent
- Subject may not be pregnant or breast feeding.
- Female people of childbearing age and male people who do not agree to abstinence or to a highly effective method of contraception (condoms and/or oral, injected or implanted hormonal methods of contraception, or...
- Human Immunodeficiency Virus (HIV) infection by history or by eligibility testing
- Concomitant treatment with Strong inducers of CYP3A4
- people not able to understand and to comply with study instructions and requirements (in patients, legally authorized representatives, or guardians as applicable) at time of informed consent.
The study team makes the final eligibility decision.
Where it is enrolling
- Philadelphia, Pennsylvania, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The study runs about 3 years per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years to 30 years. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Philadelphia, Pennsylvania, United States. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.