Overview

Sirolimus for the Treatment of Hyperinsulinism

Status:
Withdrawn
Trial end date:
2018-05-29
Target enrollment:
Participant gender:
Summary
The purpose of this pilot study is to generate data to assess feasibility of study design/procedures and for formal sample size estimation for a larger multicenter study of the efficacy and safety of sirolimus in infants with medically-unresponsive congenital hyperinsulinism (HI) due to inactivating mutations of adenosine triphosphate-sensitive potassium (KATP) channels.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
Children's Hospital of Philadelphia
Treatments:
Everolimus
Sirolimus