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

• Status: Recruiting
• Trial end date: 2026-03-01
• Target enrollment: 0
• Participant gender: All

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).

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Katarzyna Kotulska
• Treatments: Sirolimus; Vigabatrin

Criteria

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.