Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With Tuberous Sclerosis Complex

Tuberous Sclerosis Complex Clinical Trial

— RaRE-TS  

Official title:

Multicenter, Randomized, Double-blind, Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With TSC

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05534672
• Other study ID #: RaRE-TS
• Status: Recruiting
• Phase: Phase 3
• 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
• 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

Related Conditions & MeSH terms

• Drug Resistant Epilepsy
• Epilepsy
• Sclerosis
• Tuberous Sclerosis
• Tuberous Sclerosis Complex

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