NCT04987463

Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants

Randomized, Placebo-controlled, Double-blind and Double-dummy Clinical Trial Comparing the Safety, Tolerability, and Efficacy of Vigabatrin and Rapamycin in a Preventive Treatment of Infants With Tuberous Sclerosis Complex

• Status: Unknown
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 60 (estimated)
• Sponsor: Katarzyna Kotulska · Academic / Other
• Sex: All
• Age: 4 Weeks – 16 Weeks
• Healthy volunteers: Not accepted

Summary

The purpose of the study is to evaluate the efficacy, tolerability, and safety of vigabatrin versus rapamycin as a preventive treatment in infants with Tuberous Sclerosis Complex (TSC).

Detailed description

This is a two-arm, randomized, double-blind and double-dummy, placebo controlled study to evaluate the efficacy, tolerability, and safety of vigabatrin versus rapamycin as a preventive treatment in infants with TSC. The study consists of 3 phases for each patient: screening, core blinded phase, and open-label follow-up phase. Patients who meet the eligibility criteria will be randomized to receive vigabatrin or rapamycin. The randomization ratio is 1:1. Randomization will be stratified by the sex and the presence of epileptiform activity on baseline videoEEG (video electroencephalography) recording (yes versus no). Approximately 60 infants are planned to be enrolled in the study.

Conditions

• Tuberous Sclerosis Complex

Interventions

Type	Name	Description
DRUG	Vigabatrin	Vigabatrin in capsules administered orally, initially (between V1 and V2) once daily in the evening,and starting from V2 administered two times daily.
DRUG	Rapamycin	Rapamycin in liquid administered orally, in the morning, every other day or daily depending on the patient's body weight. The starting dose of rapamycin will be calculated according to the body weight of the patient measured at V1.
DRUG	Placebo	Placebo in liquid administered orally, once daily, in the morning. The starting dose of placebo in liquid will be calculated according to the body weight of the patient measured at V1.
DRUG	Placebo	Placebo in granules administered orally, initially once daily in the evening,and after reaching the targeted dose administered two times daily.

Timeline

• Start date: 2021-05-07
• Primary completion: 2026-03-01
• Completion: 2026-03-01
• First posted: 2021-08-03
• Last updated: 2024-02-08

Locations

2 sites across 1 country: Poland