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Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation. (EPIK-L1)
Price Sarah - sarah.price@cchmc.org
ALL
0 years to 100 years old
PHASE2
NCT05948943
Key inclusion 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. Drug administration via feeding tubes is allowed. Key exclusion criteria:
• 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
• 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. Drug administration via feeding tubes is allowed. Key exclusion criteria:
• 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
DRUG: Alpelisib, DRUG: Placebo
Lymphatic Malformations
Lymphatic malformations (LyM), Cystic lymphangioma, Cystic hygroma, Lymphangioma circumscriptum, Cavernous lymphangioma, Lymphangioma, Macrocystic lymphatic malformation, Microcystic lymphatic malformationn, Alpelisib
Pompe Disease Registry Protocol
Trial Transparency email recommended (Toll free number for US & Canada) - contact-us@sanofi.com
ALL
NCT00231400
Inclusion Criteria:
All patients with a confirmed diagnosis of Pompe disease who have signed the informed consent and authorization form(s) are eligible for inclusion. Confirmed diagnosis is defined as documented GAA enzyme deficiency from blood, skin, or muscle tissue and/or documentation of 2 GAA gene mutations.
Exclusion Criteria:
There are no exclusion criteria in this Registry Glycogen Storage Disease Type II, Pompe Disease
Glycogen Storage Disease Type II (GSD-II), GSD-II, Pompe Disease, Pompe Disease (late-onset), Acid Maltase Deficiency Disease, Glycogenosis II