Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study
Who is this study for? Infants with tuberous sclerosis complex
What treatments are being studied? Sirolimus
Status: Recruiting
Location: See all (11) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY
This trial is a Phase II randomized, double-blind, placebo controlled multi-site study to evaluate the safety and efficacy of early sirolimus to prevent or delay seizure onset in TSC infants. This study is supported by research funding from the Office of Orphan Products Division (OOPD) of the US Food and Drug Administration (FDA).
Eligibility
Participation Requirements
Sex: All
Minimum Age: 1 day
Maximum Age: 6 months
Healthy Volunteers: f
View:
• 0-6 months of age at the time of enrollment (subject must be \<7 months of chronological age at time of randomization and treatment initiation). Corrected age must be at least 39 weeks (calculated by subtracting the number of weeks born before 40 weeks gestation from the chronological age).
• Has a confirmed diagnosis of TSC based on established clinical or genetic criteria
Locations
United States
Alabama
University of Alabama at Birmingham
RECRUITING
Birmingham
California
University of California at Los Angeles
RECRUITING
Los Angeles
Stanford University
RECRUITING
Palo Alto
Colorado
Children's Hospital Colorado
RECRUITING
Aurora
Illinois
Lurie Children's Hospital of Chicago
RECRUITING
Chicago
Massachusetts
Boston Children's Hospital
RECRUITING
Boston
Missouri
Washington University -- St. Louis
RECRUITING
St Louis
North Carolina
University of North Carolina at Chapel Hill
RECRUITING
Chapel Hill
Ohio
Cincinnati Children's Hospital Medical Center
RECRUITING
Cincinnati
Texas
University of Texas HSC at Houston
RECRUITING
Houston
Washington
Seattle Children's Hospital
RECRUITING
Seattle
Contact Information
Primary
Molly S Griffith, BA
info@tscsteps.org
513-636-9669
Backup
Jessica Krefting, RN
info@tscsteps.org
256-533-0833
Time Frame
Start Date: 2021-10-13
Estimated Completion Date: 2027-06-30
Participants
Target number of participants: 64
Treatments
Experimental: Sirolimus
Sirolimus
Placebo_comparator: Placebo
Placebo
Authors
Related Therapeutic Areas
Sponsors
Leads: Darcy Krueger