Back to resultsPlacebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With Tuberous Sclerosis Complex (RaRE-TS)
Primary Purpose
Tuberous Sclerosis Complex
Status
Recruiting
Phase
Phase 3
Locations
Poland
Study Type
Interventional
Intervention
Rapamycin
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Tuberous Sclerosis Complex focused on measuring tuberous sclerosis complex, epilepsy, rapamycin, drug resistant epilepsy
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
Sites / Locations
- Medical University of Lodz
- Children's Memorial Health Institute, Neurology and EpileptologyRecruiting
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Rapamycin arm
Placebo arm
Arm Description
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
The patients assigned to the placebo arm will receive placebo in liquid, analogically to the rapamycin group.
Outcomes
Primary Outcome Measures
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
number of adverse events (according to CTCAE classification) in the rapamycin vs placebo group during the double-blind core phase
Secondary Outcome Measures
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
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
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05534672
Brief Title
Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With Tuberous Sclerosis Complex
Acronym
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
Study Type
Interventional
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Recruiting
Study Start Date
January 23, 2023 (Actual)
Primary Completion Date
June 2027 (Anticipated)
Study Completion Date
June 2027 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Katarzyna Kotulska
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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).
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).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Tuberous Sclerosis Complex
Keywords
tuberous sclerosis complex, epilepsy, rapamycin, drug resistant epilepsy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
200 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Rapamycin arm
Arm Type
Experimental
Arm Description
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
Arm Title
Placebo arm
Arm Type
Placebo Comparator
Arm Description
The patients assigned to the placebo arm will receive placebo in liquid, analogically to the rapamycin group.
Intervention Type
Other
Intervention Name(s)
Rapamycin
Intervention Description
Rapamycin in liquid administered orally
Intervention Type
Other
Intervention Name(s)
Placebo
Intervention Description
Placebo in liquid administered orally
Primary Outcome Measure Information:
Title
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
Title
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
Secondary Outcome Measure Information:
Title
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
Title
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
10. Eligibility
Sex
All
Minimum Age & Unit of Time
3 Months
Maximum Age & Unit of Time
50 Years
Accepts Healthy Volunteers
No
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Katarzyna Kotulska-Jozwiak
Phone
+48 22 8157404
Email
k.kotulska@ipczd.pl
First Name & Middle Initial & Last Name or Official Title & Degree
Monika Szkop
Phone
+48 22 815 74 88
Email
m.szkop@ipczd.pl
Facility Information:
Facility Name
Medical University of Lodz
City
Lodz
ZIP/Postal Code
90-419
Country
Poland
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Wojciech Mlynarski
Phone
+48 42 617-77-91
Email
wojciech.mlynarski@umed.lodz.pl
Facility Name
Children's Memorial Health Institute, Neurology and Epileptology
City
Warsaw
ZIP/Postal Code
04-730
Country
Poland
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Katarzyna Kotulska-Jozwiak
Phone
+48 22 8157404
Email
k.kotulska@ipczd.pl
First Name & Middle Initial & Last Name & Degree
Monika Szkop
Phone
+48 22 815 74 04
Email
m.szkop@ipczd.pl
12. IPD Sharing Statement
Learn more about this trial
Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With Tuberous Sclerosis Complex
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