Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants
Who is this study for? Infant patients with tuberous sclerosis complex
What treatments are being studied? TAVT-18 (sirolimus)
Status: Completed
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY
This phase I/II clinical trial is an open-label clinical trial design to verify safety and dosing for TAVT-18 (sirolimus) powder for oral solution in TSC infants (N=5).
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 (randomization and treatment initiation must occur before 7 months of age and infants born prematurely must have a corrected age of at least 39 weeks, calculated by subtracting the number of weeks born before 40 weeks gestation from the actual chronological age, in weeks)
• Has a confirmed diagnosis of TSC based on established clinical or genetic criteria
Locations
United States
Ohio
Cincinnati Children's Hospital
Cincinnati
Time Frame
Start Date: 2020-09-08
Completion Date: 2022-12-15
Participants
Target number of participants: 5
Treatments
Experimental: Stage 1 Open Label
Phase I/II, open-label PK and initial safety analysis. TAVT-18 administered orally twice/daily to achieve precision dosing target of 10 ng/ml. Whole blood sirolimus levels are assessed at defined intervals on days 1, 7, and 14. After day 14, participants can elect to continue open-label treatment with TAVT-18 until 12 months of age. Final developmental outcomes are assessed at 24 months of age.
Authors
E. Martina Bebin, Hope Northrup, Mustafa Sahin
Related Therapeutic Areas
Sponsors
Leads: Children's Hospital Medical Center, Cincinnati