Comparison of the efficacy and safety of rapamycin versus vigabatrin in the prevention of Tuberous Sclerosis Complex symptoms in infants in the randomized clinical trial

Phase 1

• Conditions: Tuberous Sclerosis ComplexEpilepsyTumors associated with Tuberous Sclerosis Complex; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2020-003231-19-PL
• Lead Sponsor: The Children's Memorial Health Institute

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-10-09
• Last Update Posted: 13 November 2023

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: 60

Inclusion Criteria

For the inclusion in the study, patients must fulfil all the following inclusion criteria:
-Male or female aged from 4 up to 16 weeks (41-56 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
Are the trial subjects under 18? yes
Number of subjects for this age range: 60
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Patient may not enter the study if any of the following exclusion criteria are met:
-history of seizures prior to randomization
-history of antiepileptic treatment
-history of treatment with mTOR inhibitor,
-gestational age below 41 weeks at the day of randomization
-body weight lower than 3 kg at the day of randomization
-SEGA 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 4 weeks 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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified