Multicenter, Randomized, Double-blind, Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With TSC
Status: Recruiting
Location: See all (2) locations...
Intervention Type: Other
Study Type: Interventional
Study Phase: Phase 3
SUMMARY
The purpose of the RaRE-TS study is to determine safety, tolerability and efficacy of rapamycin versus placebo in a drug resistant epilepsy associated with tuberous sclerosis complex (TSC).
Eligibility
Participation Requirements
Sex: All
Minimum Age: 3 months
Maximum Age: 50
Healthy Volunteers: f
View:
• male or female aged from 3 months up to 50 years at the day of randomization
• patients/parents/caregivers are willing to and able to give informed consent form for the participation in the study
• patients/parents/caregivers are willing to and able to comply with all study requirements
• definite diagnosis of TSC according to the Consensus criteria (Northrup, 2013)
• drug-resistant epilepsy associated with TSC with at least 8 seizures during 4 weeks
Locations
Other Locations
Poland
Medical University of Lodz
NOT_YET_RECRUITING
Lodz
Children's Memorial Health Institute, Neurology and Epileptology
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 88
Time Frame
Start Date: 2023-01-23
Estimated Completion Date: 2027-06
Participants
Target number of participants: 200
Treatments
Experimental: Rapamycin arm
Each patient randomized to the rapamycin arm will receive rapamycin in liquid. The rapamycin will be administered in individually calculated doses depending on the body surface of participants
Placebo_comparator: Placebo arm
The patients assigned to the placebo arm will receive placebo in liquid, analogically to the rapamycin group.
Related Therapeutic Areas
Sponsors
Leads: Katarzyna Kotulska