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

August 7, 2023 updated by: Katarzyna Kotulska

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

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).

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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).

Study Type

Interventional

Enrollment (Estimated)

200

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Poland
      • Lodz, Poland, 90-419
      • Warsaw, Poland, 04-730
        • Recruiting
        • Children's Memorial Health Institute, Neurology and Epileptology
        • Contact:
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

3 months to 50 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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
Other: Rapamycin
Rapamycin in liquid administered orally
Placebo Comparator: Placebo arm
The patients assigned to the placebo arm will receive placebo in liquid, analogically to the rapamycin group.
Other: Placebo
Placebo in liquid administered orally

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
Time Frame: final analyses after the formal final database lock, planned within one month after the last patient last visit in the study
final analyses after the formal final database lock, planned within one month after the last patient last visit in the study
number of adverse events (according to CTCAE classification) in the rapamycin vs placebo group during the double-blind core phase
Time Frame: final analyses after the formal final database lock, planned within one month after the last patient last visit in the study
final analyses after the formal final database lock, planned within one month after the last patient last visit in the study

Secondary Outcome Measures

Outcome Measure
Time Frame
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
Time Frame: final analyses after the formal final database lock, planned within one month after the last patient last visit in the study
final analyses after the formal final database lock, planned within one month after the last patient last visit in the study
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
Time Frame: final analyses after the formal final database lock, planned within one month after the last patient last visit in the study
final analyses after the formal final database lock, planned within one month after the last patient last visit in the study

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Katarzyna Kotulska

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 23, 2023

Primary Completion (Estimated)

June 1, 2027

Study Completion (Estimated)

June 1, 2027

Study Registration Dates

First Submitted

September 6, 2022

First Submitted That Met QC Criteria

September 6, 2022

First Posted (Actual)

September 9, 2022

Study Record Updates

Last Update Posted (Actual)

August 8, 2023

Last Update Submitted That Met QC Criteria

August 7, 2023

Last Verified

August 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • RaRE-TS

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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