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New treatment option for Lymphatic Malformations
Official title Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.
ClinicalTrials.gov ID: NCT05948943
What this study is testing
What is Alpelisib?
Alpelisib is an investigational medicine, given as an injection under the skin, being studied as a potential treatment for lymphatic malformations.
Also referred to as BYL719.
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
- The main purpose of this study in participants with PIK3CA-mutated LyM is to assess the change in radiological response and symptom severity upon treatment with alpelisib film-coated tablets (FCT) as compared to placebo.
- Phase 3: a large, late-stage study
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 0 to 100
You may be able to join if
- Signed informed consent and assent (when applicable) from the participant, parent, legal authorized representative or guardian.
- Participant must be willing to remain at the clinical site as required by the protocol and be willing to adhere to study restrictions and examination...
- Participant has a physician confirmed and documented diagnosis of a symptomatic LyM at the time of informed consent (Note: the physician must confirm...
- Participant is not considered as a candidate for or is not willing to receive non-drug therapies including but not limited to sclerotherapy...
- Participant has evidence of a somatic mutation(s) in the PIK3CA gene prior to randomization.
You likely can't join if
- Participant has a physician-confirmed and documented diagnosis of PROS at the time of informed consent.
- Participant has a physician-confirmed and documented diagnosis of a Central Conducting Lymphatic Anomaly, General Lymphatic Anomaly, Gorham-Stout...
- Participant has a known history of Stevens-Johnson syndrome, erythema multiforme, or toxic epidermal necrolysis at the time of informed consent.
- Participant has an established diagnosis of type I diabetes mellitus or uncontrolled type II diabetes mellitus at the time of informed consent.
- Participant had previous treatment with alpelisib and/or any other PI3K inhibitors with treatment duration longer than 2 weeks at the time of...
See the full eligibility criteria
- Signed informed consent and assent (when applicable) from the participant, parent, legal authorized representative or guardian.
- Participant must be willing to remain at the clinical site as required by the protocol and be willing to adhere to study restrictions and examination schedules.
- Participant has a physician confirmed and documented diagnosis of a symptomatic LyM at the time of informed consent (Note: the physician must confirm that the LyM cannot be included under the PROS diagnostic criteria).
- Participant is not considered as a candidate for or is not willing to receive non-drug therapies including but not limited to sclerotherapy, embolization, and surgery until the completion of Week 24 in Stage 1 and 2.
- Participant has evidence of a somatic mutation(s) in the PIK3CA gene prior to randomization.
- Participant has at least one measurable LyM lesion confirmed by BIRC assessment prior to randomization.
- Participants must be able to ingest study drug (either in tablet form or as a drinkable suspension [Groups 1 to 4] or granules or as an oral suspension [Group 5]) as assessed within 7 days before study treatment start...
- Participant has a physician-confirmed and documented diagnosis of PROS at the time of informed consent.
- Participant has a physician-confirmed and documented diagnosis of a Central Conducting Lymphatic Anomaly, General Lymphatic Anomaly, Gorham-Stout disease, Kaposiform lymphangiomatosis at the time of informed consent.
- Participant has a known history of Stevens-Johnson syndrome, erythema multiforme, or toxic epidermal necrolysis at the time of informed consent.
- Participant has an established diagnosis of type I diabetes mellitus or uncontrolled type II diabetes mellitus at the time of informed consent.
- Participant had previous treatment with alpelisib and/or any other PI3K inhibitors with treatment duration longer than 2 weeks at the time of informed consent. Other inclusion/exclusion criteria may apply
The study team makes the final eligibility decision.
Where it's taking place
- Oakland, California, United States
- Orange, California, United States
- Palo Alto, California, United States
- Washington D.C., District of Columbia, United States
- Jacksonville, Florida, United States
- Boston, Massachusetts, United States
- St Louis, Missouri, United States
- Chapel Hill, North Carolina, United States
- Cincinnati, Ohio, United States
- Cleveland, Ohio, United States
- Columbus, Ohio, United States
- Portland, Oregon, United States
- Philadelphia, Pennsylvania, United States
- Pittsburgh, Pennsylvania, United States
- Houston, Texas, United States
- Seattle, Washington, United States
- CABA, Buenos Aires, Argentina
- Capital Federal, Argentina
- Sydney, New South Wales, Australia
- Brisbane, Queensland, Australia
+ 33 more site(s).
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 - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0 years to 100 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Oakland, California, United States; Orange, California, United States; Palo Alto, California, United States; Washington D.C., District of Columbia, United States; Jacksonville, Florida, United States; Boston, Massachusetts, United States and 47 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.