Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants
Condition(s) studied
Investigational drug(s) / intervention(s)
Vigabatrin: Vigabatrin in capsules administered orally, initially (between V1 and V2) once daily in the evening,and starting from V2 administered two times daily.
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.
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.
Placebo: Placebo in granules administered orally, initially once daily in the evening,and after reaching the targeted dose administered two times daily.
Study 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).
Eligibility
Primary outcome measure(s)
- Occurrence of clinical seizures in the blinded phase of the study, — 730 days
- Summarized volume of TSC-associated tumors ≥ 125% of initial value within the blinded phase of the study — 730 days
Trial sites (2)
| Facility | City | Region | Status |
|---|---|---|---|
| Medical University of Warsaw, Department of Pediatric Neurology | Warsaw | Poland | Not Yet Recruiting |
| Children's Memorial Health Institute, Neurology and Epileptology | Warsaw | Poland | Recruiting |
More Katarzyna Kotulska trials in Poland
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT04987463 on ClinicalTrials.gov ↗ ← All trials in Poland