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Trial Title:
Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With Tuberous Sclerosis Complex
NCT ID:
NCT05534672
Condition:
Tuberous Sclerosis Complex
Conditions: Official terms:
Tuberous Sclerosis
Epilepsy
Drug Resistant Epilepsy
Sclerosis
Sirolimus
Conditions: Keywords:
tuberous sclerosis complex, epilepsy, rapamycin, drug resistant epilepsy
Study type:
Interventional
Study phase:
Phase 3
Overall status:
Recruiting
Study design:
Allocation:
Randomized
Intervention model:
Parallel Assignment
Primary purpose:
Treatment
Masking:
Double (Participant, Investigator)
Intervention:
Intervention type:
Other
Intervention name:
Rapamycin
Description:
Rapamycin in liquid administered orally
Arm group label:
Rapamycin arm
Intervention type:
Other
Intervention name:
Placebo
Description:
Placebo in liquid administered orally
Arm group label:
Placebo arm
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).
Detailed description:
This is a two-arm, randomized, double-blind, placebo controlled study to evaluate the
efficacy, tolerability, and safety of rapamycin versus placebo in a drug resistant
epilepsy associated with TSC. The study consists of 3 phases for each patient: screening,
dose adjustment blinded phase, core blinded phase, followed by open-label observation.
Patients who meet the eligibility criteria will be randomized to receive rapamycin or
placebo. The randomization ratio is 1:1. Randomization will be stratified by age, sex and
and the number of antiepileptic drugs ever used in the patient's history (up to 3 drugs /
more than 3 drugs).
Criteria for eligibility:
Criteria:
Inclusion Criteria:
- 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
Exclusion Criteria:
- history of treatment with mTOR inhibitor in the three months prior to screening,
- history of pseudo-epileptic seizures,
- history of progressive CNS disease other than TSC
- recent surgery within 2 weeks prior to the screening
- severe infection within 2 weeks prior to the screening
- use of the cannabis derivatives
- contraindications for MRI or general anesthesia
- occurrence of the serious comorbidities 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 the investigator
- pregnancy
Gender:
All
Minimum age:
3 Months
Maximum age:
50 Years
Healthy volunteers:
No
Locations:
Facility:
Name:
Medical University of Lodz
Address:
City:
Lodz
Zip:
90-419
Country:
Poland
Status:
Not yet recruiting
Contact:
Last name:
Wojciech Mlynarski
Phone:
+48 42 617-77-91
Email:
wojciech.mlynarski@umed.lodz.pl
Facility:
Name:
Children's Memorial Health Institute, Neurology and Epileptology
Address:
City:
Warsaw
Zip:
04-730
Country:
Poland
Status:
Recruiting
Contact:
Last name:
Katarzyna Kotulska-Jozwiak
Phone:
+48 22 8157404
Email:
k.kotulska@ipczd.pl
Contact backup:
Last name:
Monika Szkop
Phone:
+48 22 815 74 04
Email:
m.szkop@ipczd.pl
Start date:
January 23, 2023
Completion date:
June 2027
Lead sponsor:
Agency:
Katarzyna Kotulska
Agency class:
Other
Source:
Children's Memorial Health Institute, Poland
Record processing date:
ClinicalTrials.gov processed this data on November 12, 2024
Source: ClinicalTrials.gov page:
https://clinicaltrials.gov/ct2/show/NCT05534672
