Open, randomized, IInd phase clinical study evaluating the safety and efficacy of rapamycin in the treatment of drug-resistant epilepsy in children with rare and ultra rare diseases of the central nervous system associated with the activation of the mTOR pathway : BraimTOR-NEURO
interventional
N/A
1 country
1
The primary objective of the study is to determine safety and tolerability of rapamycin in a drug resistant epilepsy associated with rare and ultrarare diseases of the central nervous system associated with the activation of the mTOR pathway.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
Not reported
Date not reportedClick on a node to explore related trials.
Some registry-reported names shown below are not yet linked in this graph.
Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
October 16, 2024
Actual (reported)The primary safety endpoint of rapamycin will be the assessment of the incidence of adverse reactions (according to the CTCAE classification) during the treatment phase and follow-up of patients with drug-resistant epilepsy. The primary efficacy endpoint will be the proportion of patients achieving a ≥50% reduction in seizures.
A secondary safety endpoint will be the CTCAE severity of adverse reactions and the number of patients experiencing adverse reactions requiring exclusion from the study or premature termination of participation during the therapeutic dose period. The effect of treatment on the results of laboratory tests will also be analyzed. The secondary endpoint of the trial will be maintenance of short-term effects, improvement of quality of life in ≥50% of patients / families
Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.
These entries preserve how the registry describes the intervention. Their treatment roles are shown separately from molecular identities.
Formulated product
Rapamune 1 mg/mL oral solution
The registry describes a specific formulation. Its exact chemical ingredients and approval status require separate evidence.
ORAL SOLUTION
View registry recordThe registry names these ingredients. The formulation’s exact chemical form has not been confirmed.
Rapamune 1 mg/mL oral solution
Named ingredient: Sirolimus
View registry recordInstytut Pomnik Centrum Zdrowia Dziecka
Warsaw, 04-730, Poland
Anna Drużdżel
First Submitted
October 16, 2024
Download the complete CTIS (EMA) record imported by Syfrah, including fields not shown in this page. The registry may contain a newer revision.
Source: European Medicines Agency (EMA), Clinical Trials Information System (CTIS). Original record and public documents are available on CTIS.
JSON downloads include source attribution and retrieval details alongside the unchanged original record or the separately normalized values.
Source retrieved:
Contact the study team to confirm eligibility.