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

NCT05534672 | Recruiting Phase 3

• 1 other identifier: RaRE-TS
• Study Type: interventional
• Target: 200
• Locations: 1 country
• Sites: 2

Brief 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).

Conditions

Tuberous Sclerosis Complex

Keywords

tuberous sclerosis complex, epilepsy, rapamycin, drug resistant epilepsy

Outcome Measures

Primary Outcomes (2)

• 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

• 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 Outcomes (2)

• 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

• severity of adverse events (according to CTCAE) and the number of patients withdrawn from the study due to adverse events in the rapamycin vs placebo group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

Study Arms (2)

Rapamycin arm

EXPERIMENTAL

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

Placebo arm

PLACEBO COMPARATOR

The patients assigned to the placebo arm will receive placebo in liquid, analogically to the rapamycin group.

Other: Placebo

Interventions

Rapamycin OTHER

Rapamycin in liquid administered orally

Rapamycin arm

Placebo OTHER

Placebo in liquid administered orally

Placebo arm

Eligibility Criteria

• Age: 3 Months - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Katarzyna Kotulska: lead

Study Sites (2)

Medical University of Lodz

Lodz, 90-419, Poland

NOT YET RECRUITING

Children's Memorial Health Institute, Neurology and Epileptology

Warsaw, 04-730, Poland

RECRUITING

MeSH Terms

Conditions

Tuberous Sclerosis; Epilepsy; Drug Resistant Epilepsy

Interventions

Sirolimus

Condition Hierarchy (Ancestors)

Hamartoma; Neoplasms; Neoplasms, Multiple Primary; Neoplastic Syndromes, Hereditary; Malformations of Cortical Development, Group I; Malformations of Cortical Development; Nervous System Malformations; Nervous System Diseases; Neurocutaneous Syndromes; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Brain Diseases; Central Nervous System Diseases

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals

Central Study Contacts

Katarzyna Kotulska-Jozwiak

CONTACT

+48 22 8157404; k.kotulska@ipczd.pl

Monika Szkop

CONTACT

+48 22 815 74 88; m.szkop@ipczd.pl

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Head of the Department of Neurology and Epileptology at The Children's Memorial Health Institute

Study Record Dates

• First Submitted: September 6, 2022
• First Posted: September 9, 2022
• Study Start: January 23, 2023
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): June 1, 2027
• Last Updated: August 8, 2023

Record last verified: 2023-08

Locations

PL (2)