Tests rapamycin and vigabatrin to prevent symptoms in infants with tuberous sclerosis
Compares two drugs to prevent symptoms in infants with tuberous sclerosis complex.
Plain-language title generated from the registry record
Registry title: Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants
Key facts
- Where
- Lodz and Warsaw, PolandSee the 2 sites
- Phase
- Phase 2/3
Where in Poland
- Institute Of Polish Mother's Health CenterLodz, PolandTaking people
- Instytut Pomnik Centrum Zdrowia DzieckaWarsaw, PolandTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Katarzyna Kotulska
- Trial code
- NCT04987463EU 2024-513068-25-00
- Record updated
- 22 October 2026
- Original description
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).Read full original descriptionShow less
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
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).
Inclusion: Male or female aged from 4 up to 16 weeks (44-56 weeks of gestational age) at the day of randomization; Parents/caregivers are willing to and able to give informed consent form for the participation in the study; Parents/caregivers are willing to and able to comply with all study requirements; Definite diagnosis of TSC according to the Consensus criteria (Northrup,2013); At least 1 focus of cortical dysplasia disclosed on brain MRI.
Exclusion: history of seizures prior to randomization,; history of treatment with mTOR (mammalian Target of Rapamycin) inhibitor,; SEGA (Subependymal Giant Cell Astrocytoma) or other TSC-associated lesion requiring urgent surgical intervention; recent surgery within 1 month prior to the randomization; live vaccination within 1 month prior to randomization*; Use of an investigational drug within 1 month prior to randomization; history of antiepileptic treatment,; intercurrent infection at the date of randomization; known history of HIV seropositivity; lack of first TBC and hepatitis B vaccinations; Any significant clinical, laboratory , ECG or other abnormalities, comorbidity or concomitant treatment which, in the opinion of the investigator, may either put a patient at significant risk associated with the participation in the study or may influence the results of the study; gestational age below 44 weeks at the day of randomization,; body weight lower than 3 kg at the day of randomization,.