[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"pik3ca-related-overgrowth-spectrum-pros\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:pik3ca-related-overgrowth-spectrum-pros":26},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,5,0,[8,41,77,107,136],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":17,"targetDuration":4,"studyType":20,"phases":21,"briefSummary":24,"conditions":25,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":29,"lastUpdatePostDateStruct":30,"startDateStruct":33,"completionDateStruct":35,"leadSponsor":37,"locationsCount":40},"100591068","phase-1-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-100591068",false,"NCT06975618","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)","A Phase I\u002FII, Multicenter Study to Evaluate the Safety, Tolerability, Pharmacokinetic Characteristics, and Efficacy of CYH33 (a Selective PI3Kα Inhibitor) in Patients With PIK3CA-related Overgrowth Spectrum (PROS) and PIK3CA-related Vascular Malformations (PRVM)","Key inclusion criteria:\n\n1. The patient or the patient's legal guardian (if applicable) voluntarily signs the Informed Consent Form.\n2. At the time of signing the informed consent, adult patients should be ≥18 years old (or meet the legal adult age according to local regulations), and adolescent patients should be ≥12 years old and \\\u003C18 years old (or meet the legal definition of adolescent according to local regulations; additionally, adolescent patients should weigh ≥35 kg).\n3. The patient is diagnosed with PIK3CA-related overgrowth spectrum (PROS) or PIK3CA-related vascular malformations (PRVM), and provides a report confirming PIK3CA mutation detected by local laboratory or the Sponsor-designated central laboratory, with at least one measurable lesion related to PROS or PRVM.\n4. Patients should demonstrate adequate organ and bone marrow function during the 28-day screening period.\n\nKey exclusion criteria:\n\n1. PROS patients presenting solely with isolated macrodactyly, epidermal nevi\u002Fnevus, and megalencephaly (only one clinical feature or any combination of these three features) without other PROS-related lesions.\n2. Patients who have received any systemic treatment for PROS or PRVM within 8 weeks prior to the first dose of study drug, or any drug treatment for PROS or PRVM (e.g., mTOR inhibitors) within 28 days prior to the first dose of study drug.\n3. Patients who have previously received any PI3K inhibitor treatment.","ALL",{"count":18,"type":19},141,"ESTIMATED","INTERVENTIONAL",[22,23],"PHASE1","PHASE2","This study is a multi-center, open-label, single arm, phase I\u002FII 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)",[26,27],"PIK3CA-Related Overgrowth Spectrum (PROS)","PIK3CA-related Vascular Malformations (PRVM)","RECRUITING","2026-06-26",{"date":31,"type":32},"2026-06-29","ACTUAL",{"date":34,"type":32},"2023-08-22",{"date":36,"type":19},"2029-12-31",{"name":38,"class":39},"Haihe Biopharma Co., Ltd.","INDUSTRY",15,{"id":42,"slug":43,"hasResults":11,"nctId":44,"briefTitle":45,"officialTitle":46,"acronym":47,"eligibilityCriteria":48,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":49,"targetDuration":4,"studyType":20,"phases":51,"briefSummary":52,"conditions":53,"keywords":55,"overallStatus":67,"whyStopped":4,"lastUpdateSubmitDate":68,"lastUpdatePostDateStruct":69,"startDateStruct":71,"completionDateStruct":73,"leadSponsor":75,"locationsCount":4},"100645206","phase-2-roll-over-study-of-alpelisib-byl719-for-continued-access-and-long-term-safety-100645206","NCT07679269","Roll-Over Study of Alpelisib (BYL719) for Continued Access and Long-Term Safety.","An Open Label, Multi-center Alpelisib Roll-over Study to Assess Long-term Safety in Participants Who Have Completed a Novartis Sponsored Alpelisib (BYL719) Study and Are Judged by the Investigator to Benefit From Continued Treatment With Alpelisib.","EPIK-RO","Key Inclusion Criteria:\n\n* Written informed consent\u002Fassent, according to local guidelines, signed by the participants and\u002For by the parents or legal guardian prior to enrolling in the roll-over study.\n* Participant currently enrolled in a Novartis-sponsored study, is currently receiving alpelisib as a single agent or in combination with other drugs, and has fulfilled all on-treatment requirements in the parent study.\n* Participant is currently benefiting from the treatment with alpelisib as determined by the Investigator in the parent study.\n* Participant demonstrated compliance with the visit schedule in the parent study, and in the opinion of the Investigator has shown willingness and ability to comply with future visit schedules, treatment plans, and any other study procedures in this protocol.\n\nKey Exclusion Criteria:\n\n* Participant had permanently discontinued from alpelisib in the parent study for any reason including withdrawal of consent.\n* Participant currently has ongoing\u002Funresolved treatment related Grade 3 or higher AEs, and\u002For any ongoing\u002Funresolved AE or toxicities for which alpelisib dosing has been interrupted in the parent study. Participants meeting all other eligibility criteria may be enrolled once toxicities have improved to allow alpelisib dosing to resume as stated in the parent protocol.\n* Participant's ongoing treatment is currently approved and reimbursed for their indication at their country level. In exceptional cases where the treatment is reimbursed at the country level, but not individual level, please contact the Novartis Study Team.\n* Concurrent participation in any other investigational clinical trial other than the parent study.\n* Pregnant or nursing (breastfeeding) women.\n* Female participants of childbearing potential who do not consent to use a highly effective method of contraception, and male participants who do not consent to use a condom and\u002For a highly effective method of contraception, including refraining from sperm donation and complying with measures to prevent exposure of a partner to alpelisib via seminal fluid, for the duration of the study and for one week following discontinuation of alpelisib (or longer if required per parent protocol).\n\nOther inclusion\u002Fexclusion criteria may apply.",{"count":50,"type":19},51,[23],"The purpose of this study is to provide post-trial access to alpelisib and to assess its long-term safety when administered as a single agent or in combination with other drugs. This study is intended for participants who are currently receiving alpelisib in a Novartis-sponsored clinical trial (parent study) and, in the Investigator's judgment, would benefit from continued treatment with alpelisib.",[26,54],"Breast Neoplasms (Including Advanced or Metastatic Breast Cancer With PIK3CA Mutation)",[56,57,58,59,60,61,62,63,64,65,66],"Alpelisib (BYL719)","PI3K inhibitor","PIK3CA mutation","Post-trial access","Roll-over study","Long-term safety","Open-label","Continued treatment access","Solid tumors","Breast cancer","PROS","NOT_YET_RECRUITING","2026-06-25",{"date":70,"type":32},"2026-07-01",{"date":72,"type":19},"2027-01-01",{"date":74,"type":19},"2031-07-25",{"name":76,"class":39},"Novartis Pharmaceuticals",{"id":78,"slug":79,"hasResults":11,"nctId":80,"briefTitle":81,"officialTitle":82,"acronym":4,"eligibilityCriteria":83,"healthyVolunteers":11,"sex":16,"minAge":84,"maxAge":85,"enrollmentInfo":86,"targetDuration":4,"studyType":20,"phases":88,"briefSummary":89,"conditions":90,"keywords":92,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":98,"lastUpdatePostDateStruct":99,"startDateStruct":101,"completionDateStruct":103,"leadSponsor":105,"locationsCount":106},"100592757","phase-2-epik-p4-a-phase-ii-single-arm-study-to-assess-the-efficacy-safety-and-pharmacokinetics-of-alpelisib-byl719-in-pediatric-and-adult-patients-with-pik3ca-related-overgrowth-spectrum-pros-100592757","NCT06997588","EPIK-P4: A Phase II Single-arm Study to Assess the Efficacy, Safety and Pharmacokinetics of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum (PROS)","Study Assessing the Efficacy, Safety and Pharmacokinetics of Alpelisib in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum (PROS)","Key Inclusion Criteria:\n\n1. Male or female participants aged ≥2 years at the time of informed consent\u002Fassent.\n2. Participants with diagnosis of PROS (according to Clinical Diagnostic Criteria for PROS proposed by Keppler Noreuil et al 2014) with symptomatic AND progressive overgrowth, who have syndromic disease or isolated features (with the exception of isolated macrodactyly, macrocephaly or epidermal nevus) at the time of informed consent\u002Fassent.\n3. Documented evidence of a somatic mutation(s) in the PIK3CA gene performed in local laboratories using a DNA-based test AND available archival tissue (if archival tissue sample is not available, a fresh biopsy should be performed, if it is not clinically contraindicated) at the time of informed consent\u002Fassent.\n4. Karnofsky (in participants \\>16 years of age at study entry) or Lansky (≤16 years of age at study entry) performance status index ≥50.\n5. PGI-S score of mild, moderate, severe, or very severe at screening.\n6. Adequate bone marrow and organ function.\n7. Presence of at least 1 PROS-related measurable lesion (longest diameter ≥2 cm) confirmed by BIRC assessment and associated with complaints, clinical symptoms or functional limitations affecting the participant's everyday life.\n\nKey Exclusion Criteria:\n\n1. Participant with only isolated macrodactyly, epidermal nevus\u002Fnevi and macroencephaly (the only clinical feature or a combination of any of three of them), in absence of other PROS-related lesions at the time of informed consent\u002Fassent.\n2. Previous treatment with alpelisib and\u002For any other phosphatidylinositol 3-kinase (PI3K) inhibitor(s) (except treatment attempt, defined as the attempt to treat PROS with any of PI3K inhibitors, with treatment duration less than 2 weeks and stopped at least 4 weeks prior to the first dose of study medication with alpelisib).\n3. Debulking or other major surgery performed within 3 months at the time of informed consent\u002Fassent.\n4. Radiation exposure for PROS treatment purpose within 12 months prior to informed consent\u002Fassent.\n5. Clinically meaningful PROS-related thrombotic event (Grade 2 and more as per CTCAE v4.03) within 30 days before informed consent\u002Fassent, and\u002For sclerotherapy\u002Fembolization for vascular complications performed within 6 weeks before informed consent\u002Fassent.\n6. Clinically meaningful bleeding from PROS-related lesion (Grade 2 and more as per CTCAE v4.03) within 30 days before study treatment initiation.\n7. Participants with clinically significant worsening of PROS-related laboratory abnormalities, physical signs and symptoms (such as, but not limited to increase of D-dimers, worsening of underlying pain, newly occurring swelling or redness) indicating an uncontrolled condition during the screening phase.\n\nOther inclusion\u002Fexclusion criteria may apply","2 Years","100 Years",{"count":87,"type":19},104,[23],"This study is designed to demonstrate the efficacy and assess safety and tolerability of oral daily alpelisib in participants with PIK3CA-related overgrowth spectrum (PROS).",[91],"PIK3CA-related Overgrowth Spectrum (PROS)",[93,56,94,95,96,97],"PIK3CA-related overgrowth spectrum (PROS)","Phase II","Adult","Pediatric","Adolescent","2026-06-18",{"date":100,"type":32},"2026-06-22",{"date":102,"type":32},"2025-10-09",{"date":104,"type":19},"2030-09-30",{"name":76,"class":39},33,{"id":108,"slug":109,"hasResults":11,"nctId":110,"briefTitle":111,"officialTitle":112,"acronym":4,"eligibilityCriteria":113,"healthyVolunteers":11,"sex":16,"minAge":84,"maxAge":4,"enrollmentInfo":114,"targetDuration":4,"studyType":20,"phases":116,"briefSummary":117,"conditions":118,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":126,"lastUpdatePostDateStruct":127,"startDateStruct":129,"completionDateStruct":131,"leadSponsor":133,"locationsCount":135},"100576793","phase-2-a-phase-2-study-of-mutant-selective-pi3k-inhibitor-rly-2608-in-adults-and-children-with-pik3ca-related-overgrowth-spectrum-and-malformations-driven-by-pik3ca-mutation-the-reinspire-study-100576793","NCT06789913","A Phase 2 Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, in Adults and Children With PIK3CA Related Overgrowth Spectrum and Malformations Driven by PIK3CA Mutation (The ReInspire Study)","A Phase 2 Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, in Adults and Children With PIK3CA Related Overgrowth Spectrum and Malformations Driven by PIK3CA Mutation","Key Inclusion Criteria:\n\n* The participant must have a clinical diagnosis of PROS or a malformation within the ISSVA classification.\n* One or more documented activating PIK3CA mutation(s) that are targeted by selective PI3Kα inhibitors in lesional tissue and\u002For cell-free DNA from the lesion or blood. Some participants may be eligible without a documented PIK3CA mutation, with the sponsor's approval, as long as no other genetic driver has been documented.\n* Lansky (\\\u003C16 yo) or Karnofsky (≥16 yo) performance status of ≥50.\n* Agree to provide archived lesional fluid and\u002For tissue or be willing to undergo pretreatment lesional biopsy (if considered safe and medically feasible) to assess PIK3CA status.\n\nKey Exclusion Criteria:\n\n* Known hypersensitivity to RLY-2608.\n* Any factors that increase the risk of QTc prolongation or risk of arrhythmic events\n* Clinically significant, uncontrolled cardiovascular disease\n* Received disease-directed therapy prior to the first dose of study drug:\n\n  1. Systemic therapy or antibody within 5 half-lives of the therapy.\n  2. Local therapy including radiation, surgery, or other procedures within 28 days; lesion(s) must have demonstrated progression after the procedure.",{"count":115,"type":19},277,[23],"This is a 3-part Phase 2 randomized study evaluating the safety and efficacy of the mutant-selective PI3Kα inhibitor, zovegalisib (RLY-2608), in adults and children with PIK3CA Related Overgrowth Spectrum (PROS) and malformations driven by PIK3CA mutation. Part 1 is a dose selection, Part 2 is a basket design with exploratory single-arm cohorts for various subpopulations of participants, and Part 3 is randomized, double-blinded study vs placebo.",[26,119,120,121,122,123,124,125],"Lymphatic Malformations","Vascular Malformations","PIK3CA Mutation","CLOVES Syndrome","Klippel Trenaunay Syndrome","Megalencephaly-capillary Malformation Polymicrogyria Syndrome (MCAP)","Vascular Anomalies","2026-06-10",{"date":128,"type":32},"2026-06-12",{"date":130,"type":32},"2025-06-13",{"date":132,"type":19},"2031-10",{"name":134,"class":39},"Relay Therapeutics, Inc.",34,{"id":137,"slug":138,"hasResults":11,"nctId":139,"briefTitle":140,"officialTitle":140,"acronym":4,"eligibilityCriteria":141,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":4,"targetDuration":4,"studyType":142,"phases":4,"briefSummary":143,"conditions":144,"keywords":146,"overallStatus":150,"whyStopped":4,"lastUpdateSubmitDate":151,"lastUpdatePostDateStruct":152,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":154,"locationsCount":4},"100369079","managed-access-programs-for-byl719-alpelisib-100369079","NCT04085653","Managed Access Programs for BYL719, Alpelisib","Inclusion Criteria:\n\nAn independent request was received from a licensed physician. The patient has a serious or life-threatening disease or condition and there is no comparable or satisfactory alternative therapy available for diagnosis, monitoring, or treatment.\n\nThe patient is not eligible or able to enrol in a clinical trial or continue participation in such trial.\n\nThere is a potential patient benefit to justify the potential risk of the treatment use, and the potential risk is not unreasonable in the context of the disease or condition to be treated.\n\nThe patient must meet any other medical criteria established by the medical experts responsible for the product or by the health authority in the country of request (as applicable).\n\nProvision of the product will not interfere with the initiation, conduct, or completion of a Novartis clinical trial or overall development program.\n\nManaged Access provision is allowed per local laws\u002Fregulations.","EXPANDED_ACCESS","The purpose of this registration is to list Managed Access Programs (MAPs) related to BYL719, Alpelisib",[26,145],"Breast Cancer",[147,148,149],"BYL719","alpelisib","MAP","AVAILABLE","2026-04-09",{"date":153,"type":32},"2026-04-14",{"name":76,"class":39}]