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Recruiting Phase 2/3

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

NCT04987463 · tracked via the Priya Life Science Poland tracker
Sponsor
Katarzyna Kotulska
Phase
Phase 2/3
Started
2021-05-07
Last updated
2026-10-22

Condition(s) studied

Tuberous Sclerosis Complex

Investigational drug(s) / intervention(s)

VigabatrinRapamycin →PlaceboPlacebo

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

Sex
ALL
Min age
4 Weeks
Max age
16 Weeks
Healthy volunteers
No
Inclusion Criteria: * 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 Criteria: * history of seizures prior to randomization, * history of antiepileptic treatment, * history of treatment with mTOR (mammalian Target of Rapamycin) inhibitor, * gestational age below 44 weeks at the day of randomization, * body weight lower than 3 kg at the day of randomization, * SEGA (Subependymal Giant Cell Astrocytoma) or other TSC-associated lesion requiring urgent surgical intervention * recent surgery within 1 month prior to the randomization * intercurrent infection at the date of randomization * known history of HIV seropositivity * live vaccination within 1 month prior to randomization\* * 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. * Use of an investigational drug within 1 month prior to randomization.

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)

FacilityCityRegionStatus
Medical University of Warsaw, Department of Pediatric Neurology Warsaw Poland Not Yet Recruiting
Children's Memorial Health Institute, Neurology and Epileptology Warsaw Poland Recruiting
Official registry record

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