Health TrialsbyHealthPanel

Sirolomus Adjuvant Treatment of Focal Refractory Epilepsy (SATFRE)

ConditionEpilepsy, Drug Resistant
Trial statusRecruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age18-60
SponsorXuanwu Hospital, Beijing

About this trial

The aim of this study is to evaluate the clinical efficacy of sirolimus as an adjunctive therapy for refractory epilepsy. The significance lies in addressing whether the mTOR inhibitor sirolimus has antiepileptic adjunctive effects for a broader range of patients with refractory epilepsy, with the hope of providing a new mTOR-targeted antiepileptic adjunctive medication regimen that is administered only during epileptic events and can be widely used for various types of refractory epilepsy.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

18-60 years of age.

Meet the 2017 International League Against Epilepsy (ILAE) diagnostic criteria for for focal seizures or focal seizures progressing to bilateral tonic-clonic seizures.

Diagnosed with refractory epilepsy, having used at least two AEDs without effectiveness for 2 years. No drug interaction between current AEDs and sirolomus, and a stable dose for at least 12 weeks prior to enrollment.

Seizure duration ≥1 minute, with accompanying sensory impairment.

Disqualifiers

Diagnosed with generalized or hereditary epilepsy with ion channel gene mutations

Psychogenic non-epileptic seizures within 12 months;

Treatable causes of epilepsy (such as metabolic disorders, toxicity, infections, space occupying lesions, or identified genetic abnormalities)

Patients with only non-motor focal seizures, as classified by the 2017 ILAE.

Trial design

Design model

Single group

Treatments tested in this trial

  • Sirolimus

    Drug

    Oral administration is given solely for the epilepsy seizure event. Dosage: The dosage is determined based on body surface area (BSA), with 0.5 mg per dose for BSA \<1.2 m², 1 mg per dose for BSA 1.3-2.1 m², and 1.5 mg per dose for BSA \>2.2 m².

Treatment groups

15 Participants
are divided into 1 treatment group
Group A: Open label single arm, drug sirolomusExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Percent Change From Baseline in Seizure Frequency Per 28 Days

Percent change in 28-day frequency of seizures during the 40 week treatment and follow-up period relative to baseline

Time frame
assessed per 28 days during the treatment.
2

50% Seizure Responder Rate

The proportion of patients with a ≥ 50% reduction from Baseline in seizure frequency.

Time frame
short-term treatment period (ending at 3 months); long-term follow-up period (ending at 12 months).

Secondary outcomes

1

Seizure Severity

The percent change from baseline in seizure severity evaluated by Liverpool seizure severity scale (LSSS) .

Time frame
Baseline observation period; short-term treatment period (ending at 3 months); long-term follow-up period (ending at 12 months).
2

Life quality evaluation

Percentage change from baseline in Quality of Life in Epilepsy-31 inventory (QOLIE-31) score.

Time frame
Baseline observation period; short-term treatment period (ending at 3 months); long-term follow-up period (ending at 12 months).
3

Adverse Events

Rate of adverse events which were judged to be study-related throughout the study.

Time frame
through study completion, 12 months.

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Xuanwu Hospital, Beijing

Lead sponsor

Peking University

Collaborator

Source ClinicalTrials.gov
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