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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05534672
Other study ID # RaRE-TS
Secondary ID
Status Recruiting
Phase Phase 3
First received
Last updated
Start date January 23, 2023
Est. completion date June 2027

Study information

Verified date August 2023
Source Children's Memorial Health Institute, Poland
Contact Katarzyna Kotulska-Jozwiak
Phone +48 22 8157404
Email k.kotulska@ipczd.pl
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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).


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).


Recruitment information / eligibility

Status Recruiting
Enrollment 200
Est. completion date June 2027
Est. primary completion date June 2027
Accepts healthy volunteers No
Gender All
Age group 3 Months to 50 Years
Eligibility 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

Study Design


Intervention

Other:
Rapamycin
Rapamycin in liquid administered orally
Placebo
Placebo in liquid administered orally

Locations

Country Name City State
Poland Medical University of Lodz Lodz
Poland Children's Memorial Health Institute, Neurology and Epileptology Warsaw

Sponsors (1)

Lead Sponsor Collaborator
Katarzyna Kotulska

Country where clinical trial is conducted

Poland, 

Outcome

Type Measure Description Time frame Safety issue
Primary comparison of the number of patients with at least 50% reduction of seizures per week in the last month of the core blinded phase in comparison to screening phase in the rapamycin vs placebo group final analyses after the formal final database lock, planned within one month after the last patient last visit in the study
Primary number of adverse events (according to CTCAE classification) in the rapamycin vs placebo group during the double-blind core phase final analyses after the formal final database lock, planned within one month after the last patient last visit in the study
Secondary comparison of the number of seizures per week and the number of days free of seizures in the rapamycin vs placebo group, during 12-week treatment in double-blind core phase final analyses after the formal final database lock, planned within one month after the last patient last visit in the study
Secondary severity of adverse events (according to CTCAE) and the number of patients withdrawn from the study due to adverse events in the rapamycin vs placebo group final analyses after the formal final database lock, planned within one month after the last patient last visit in the study
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