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

Launched by KATARZYNA KOTULSKA · Sep 6, 2022

Keywords

ClinConnect Summary

The RaRE-TS study is a clinical trial looking at the safety and effectiveness of a medication called rapamycin for people with drug-resistant epilepsy linked to a condition known as Tuberous Sclerosis Complex (TSC). This trial is important because TSC can cause severe epilepsy that doesn't respond well to standard treatments, and researchers want to see if rapamycin can help manage these difficult seizures. The study is currently recruiting participants aged between 3 months and 50 years who have been diagnosed with TSC and have experienced at least 8 seizures in the past month.

If you or your child are eligible and decide to participate, you'll be asked to follow certain guidelines and complete some assessments throughout the study. Participants will either receive rapamycin or a placebo (a harmless substance that looks like the medication but has no active ingredients) to compare the effects. It's important to note that there are some criteria that would exclude participants, such as recent surgeries, certain medical conditions, or if you have used specific medications lately. This trial aims to provide valuable information that could lead to better treatment options for those struggling with epilepsy associated with TSC.

Gender

ALL

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