NCT03047980

Trial of Sirolimus for Cognitive Impairment in Sturge-Weber Syndrome

• Status: Completed
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: Anne Comi, MD · Academic / Other
• Sex: All
• Age: 3 Years – 31 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this research study is to gain a preliminary understanding of the safety of sirolimus in Sturge-Weber syndrome (SWS) and determine best outcomes to be used to assess the utility of sirolimus for the treatment of cognitive impairments related to Sturge-Weber syndrome.

Detailed description

Sirolimus will be administered as an adjunct to all current medications. The impact of sirolimus upon cognitive functioning in Sturge-Weber syndrome is the primary outcome measure. This outcome will be assessed using a panel of testing selected based upon extensive experience in testing cognitive function in adults and children with SWS at the Kennedy Krieger Sturge-Weber Center. Changes in a quantitative EEG before and after the trial, Sturge-Weber syndrome clinical neuroscore, port-wine birthmark score, and the impact of sirolimus upon seizures will be assessed.

Conditions

• Sturge-Weber Syndrome

Interventions

Type	Name	Description
DRUG	Sirolimus	Low dose oral sirolimus

Timeline

• Start date: 2017-01-01
• Primary completion: 2019-06-04
• Completion: 2020-10-27
• First posted: 2017-02-09
• Last updated: 2021-11-01
• Results posted: 2021-11-01

Locations

2 sites across 1 country: United States