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NCT04085653
The purpose of this registration is to list Managed Access Programs (MAPs) related to BYL719, Alpelisib
NCT04980833
This is a prospective interventional Phase II multi center study, open label, preceded by a retrospective non-interventional period, to assess the long-term safety and efficacy of alpelisib, in pediatric and adult participants with PROS.
NCT04589650
This is a prospective Phase II multi-center study with an initial 16-week, randomized, double-blind, placebo-controlled period, followed by two extension periods to assess the efficacy, safety and pharmacokinetics (PK) of alpelisib in pediatric and adult patients with PIK3CA-related overgrowth spectrum (PROS)
NCT06975618
This study is a multi-center, open-label, single arm, phase I/II study to evaluate the safety, tolerability, pharmacokinetics and preliminary efficacy of CYH33 in patients with PIK3CA-related overgrowth spectrum (PROS) and PIK3CA-related vascular malformations (PRVM)
NCT04980872
This is a study of the safety and tolerability of oral miransertib (MK-7075) administered to participants at least 2 years of age with phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit alpha (PIK3CA)-related overgrowth spectrum (PROS) or Proteus Syndrome (PS). This is an extension of other miransertib studies (MK-7075-002 \[NCT03094832\] or ArQule CU/EAP \[NCT03317366\]), and may also enroll participants who are approved for MK-7075-002 but have not yet started miransertib therapy.
NCT03094832
This is an open label, Phase 1/2 study of oral miransertib (MK-7075) administered to participants at least 2 years of age with phosphatidylinositol-4,5-bisphosphate 3-kinase, catalytic subunit alpha (PIK3CA)-related Overgrowth Spectrum (PROS) and Proteus Syndrome (PS) (MOSAIC).
NCT02428296
Background: \- PIK3CA-related overgrowth spectrum (PROS) is caused by changes in the PIK3CA gene. This gene makes a protein that communicates with other proteins in the body to cause cells to grow. Alterations in PIK3CA change the chemical signals in the body and cause overgrowth in fatty, vascular and other tissues. Sirolimus is a drug that reduces the signals sent by one of the proteins in this chemical signaling pathway. Researchers want to learn whether the drug sirolimus can reduce or stabilize some of the overgrowth that patients with PROS experience. Objectives: \- To measure how the overgrowth of patients with PROS changes over time and whether taking a drug called sirolimus can reduce or stabilize a person s overgrowth. Eligibility: \- People ages 3 to 65 years old with a confirmed mutation or alteration of the PIK3CA gene in the person s affected tissues (a somatic mutation). Design: * Participants will be screened with medical history and genetic counseling. * First 6 months: Participants will have their overgrowth monitored. * Next 6 months: Participants will take sirolimus once or twice a day. * Participants will have to visit the clinic several times, and stay in the area for 4 to 5 days each time. * Participants will have a one month-long visit to the clinic. * During clinic visits, participants will have: * Blood and urine tests. * Photographs of their physical features. * Scans, including an MRI and DEXA, and possibly x-rays and CT scans. * For the MRI and CT scans, participants will lie in a machine that takes pictures of their body. * The DEXA involves a small amount of radiation. * They may have: * Non-invasive heart function tests. * Lung function tests. * Participants will have several blood and urine tests between visits. * Participants will complete surveys and keep a diary of their treatment and side effects. * Participants may visit other health specialists or undergo other tests based on side effects. * One month after stopping the study drug, participants will have 1 clinic visit.