PIK3CA-RELATED OVERGROWTH SPECTRUM (PROS)
Clinical trials for PIK3CA-RELATED OVERGROWTH SPECTRUM (PROS) explained in plain language.
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Lifeline offered for rare growth disorder patients
Disease control AVAILABLEThis program provides access to the drug alpelisib for patients with PIK3CA-Related Overgrowth Spectrum (PROS), a rare genetic condition causing abnormal tissue growth. It's designed for patients with severe or life-threatening cases who have no other feasible treatment options. …
Matched conditions: PIK3CA-RELATED OVERGROWTH SPECTRUM (PROS)
Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Mar 30, 2026 14:35 UTC
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New drug trial targets root cause of rare, painful growth disorders
Disease control Recruiting nowThis early-stage study is testing an experimental drug called CYH33 for people with rare disorders that cause abnormal tissue and blood vessel overgrowth (PROS and PRVM). The main goals are to find a safe dose and see if the drug can shrink the overgrown lesions by at least 20% a…
Matched conditions: PIK3CA-RELATED OVERGROWTH SPECTRUM (PROS)
Phase: PHASE1, PHASE2 • Sponsor: Haihe Biopharma Co., Ltd. • Aim: Disease control
Last updated Mar 30, 2026 14:30 UTC
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Targeted drug trial offers hope for rare growth disorders
Disease control Recruiting nowThis study is testing an experimental drug called RLY-2608 in adults and children with rare conditions that cause abnormal tissue overgrowth due to a specific genetic mutation. The research aims to find the right dose, check safety, and see if the drug can reduce the size of over…
Matched conditions: PIK3CA-RELATED OVERGROWTH SPECTRUM (PROS)
Phase: PHASE2 • Sponsor: Relay Therapeutics, Inc. • Aim: Disease control
Last updated Mar 27, 2026 12:40 UTC
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New pill aims to shrink painful overgrowths in rare disorder
Disease control Recruiting nowThis study is testing whether a daily pill called alpelisib can safely shrink overgrowths and improve symptoms in children and adults with PROS, a rare genetic disorder that causes abnormal tissue growth. About 104 participants aged 2 and older will take the drug for up to 3 year…
Matched conditions: PIK3CA-RELATED OVERGROWTH SPECTRUM (PROS)
Phase: PHASE2 • Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Mar 17, 2026 13:10 UTC