BridgeMD
Alpelisib for the treatment of lymphatic malformations associated with a PIK3CA mutation
EU CTIS ID: 2023-504146-60-00
What this study is testing
Stage 2: To demonstrate the efficacy of alpelisib as measured by the proportion of participants randomized to alpelisib with a radiological response at Week 24 (confirmatory phase) of Stage 2 as compared to those randomized to placebo in at least one of the following groups: Group 3 (participants ≥ 18 years of age) Group 4 (participants 6 - 17 years of age)
- Phase II and Phase III (Integrated)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
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 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 at the time of informed consent.
- 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.
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 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.
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 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 at the time of informed consent.
- 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 in tablet or as an oral suspension (Groups 1 to 4) or granules or as an oral suspension (Group 5) as assessed within 7 days before study treatment start. Drug administration via feeding tubes is allowed.
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- United States
- United Kingdom
- Australia
- Argentina
- Switzerland
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, 0-17 years, 65+ years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; United Kingdom; Australia; Argentina; Switzerland. 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 EU CTIS; the study team decides eligibility.