Sirolimus for the Treatment of Hyperinsulinism

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.

Timeline

Start

2015-08-12

Primary completion

2018-05-29

Completion

2018-05-29

Drugs

Indications