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
Who is this study for? Infants with tuberous sclerosis complex
Status: Recruiting
Location: See all (2) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2/Phase 3
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
Participation Requirements
Sex: All
Minimum Age: 28 days
Maximum Age: 3 months
Healthy Volunteers: f
View:
• 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
Locations
Other Locations
Poland
Children's Memorial Health Institute, Neurology and Epileptology
RECRUITING
Warsaw
Medical University of Warsaw, Department of Pediatric Neurology
NOT_YET_RECRUITING
Warsaw
Contact Information
Primary
Katarzyna Kotulska-Jozwiak
k.kotulska@ipczd.pl
+48 22 8157404
Backup
Monika Szkop
m.szkop@ipczd.pl
+48 22 815 74 04
Time Frame
Start Date: 2021-05-07
Estimated Completion Date: 2026-03
Participants
Target number of participants: 60
Treatments
Experimental: Vigabatrin arm
Vigabatrin in capsules co-administered with placebo in liquid.
Experimental: Rapamycin arm
Rapamycin in liquid co-administered with placebo in capsules.
Related Therapeutic Areas
Sponsors
Leads: Katarzyna Kotulska