NCT04595513

Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 5 (actual)
• Sponsor: Children's Hospital Medical Center, Cincinnati · Academic / Other
• Sex: All
• Age: 1 Day – 6 Months
• Healthy volunteers: Not accepted

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).

Detailed description

Tuberous Sclerosis Complex (TSC) is caused by genetic mutation in TSC1 or TSC2, resulting in dysregulation of the mechanistic target of rapamycin (mTOR) signaling pathway. Age at time of seizure onset in TSC infants has been linked to long-term neurodevelopmental outcome in this high-risk population. TAVT-18 is a novel formulation of sirolimus, an mTOR inhibitor. This study evaluates TAVT-18 as a targeted, disease-modifying drug therapy for preventing or delaying seizure onset in TSC using a rational, mechanism-based therapeutic approach.

Conditions

• Tuberous Sclerosis Complex
• Epilepsy

Interventions

Type	Name	Description
DRUG	TAVT-18 (sirolimus)	The investigational drug product to be used in this study is TAVT-18, a proprietary formulation of sirolimus in clinical development, by Tavanta Therapeutics, Inc. It is provided in a powder formulation in pre-measured vials.

Timeline

• Start date: 2020-09-08
• Primary completion: 2022-12-15
• Completion: 2022-12-15
• First posted: 2020-10-20
• Last updated: 2024-05-16
• Results posted: 2024-05-16

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study