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Sirolimus for PIK3CA-related segmental overgrowth (pilot, non-randomised, open-label)
2014-000484-41Phase 2Completed
- Sponsor
- Cambridge University Hospitals NHS Foundation Trust
- Start
- Mar 2016
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of sirolimus in PIK3CA-related overgrowth spectrum (PROS) and Congenital, Hereditary, and Neonatal Diseases and Abnormalities, sponsored by Cambridge University Hospitals NHS Foundation Trust. Primary endpoint: Primary effect size of sirolimus therapy on PIK3CA-related overgrowth. Started Mar 2016; status: completed.
02
Drugs
sirolimus
03
Conditions
PIK3CA-related overgrowth spectrum (PROS)Congenital, Hereditary, and Neonatal Diseases and Abnormalities
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