Comparing Rapamycin and Vigabatrin for Preventing Symptoms of Tuberous Sclerosis in Infants

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial evaluates the efficacy, tolerability, and safety of two medications, vigabatrin and rapamycin, as preventive treatments for infants diagnosed with Tuberous Sclerosis Complex (TSC). It is designed as a randomized, double-blind, and placebo-controlled study, involving three phases: screening, a core blinded phase, and an open-label follow-up phase. Approximately 60 infants will be enrolled, and participants will be randomly assigned to receive either vigabatrin or rapamycin, with randomization stratified by sex and baseline epileptiform activity. The study aims to determine which medication is more effective in preventing symptoms associated with TSC.

Why it matters

Who can join: the full rules

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.

Where it's running

• Children's Memorial Health Institute, Neurology and Epileptology
  Warsaw, Poland Recruiting
• Medical University of Warsaw, Department of Pediatric Neurology
  Warsaw, Poland Not yet recruiting

Study contacts

• Principal investigator: Katarzyna Kotulska-Jozwiak — The Children's Memorial Health Institute
• Study coordinator: Katarzyna Kotulska-Jozwiak
• Email: k.kotulska@ipczd.pl
• Phone: +48 22 8157404

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.