epilepsy, mTORopathies, focal cortical dysplasia, LEATS
Conditions
Brief summary
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.
Detailed description
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
Interventions
Sponsors
Instytut Pomnik Centrum Zdrowia Dziecka
Eligibility
Sex/Gender
All
Age
0 Years to 17 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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. | — |
Secondary
| Measure | Time frame |
|---|---|
| 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 | — |
Countries
Poland
Outcome results
None listed