Study of Interest of Stiripentol and Carbamazepine in the Treatment of Patients With Pharmacoresistant Focal Epilepsies

Phase 4

Completed

• Conditions: Pharmacoresistant Focal Epilepsies
• Interventions: Drug: Stiripentol

• Registration Number: NCT05419180
• Lead Sponsor: Biocodex

Brief Summary

This is a monocentric, open-label clinical study, presenting a retrospective part and a prospective part, studying the data of patients with drug-resistant focal epilepsies and treated with the combination of stiripentol (Diacomit®) and Carbamazepine.

Detailed Description

Not available

Study Timeline

• Status Verified: 2022-05
• Study First Submitted: 2022-05-16
• Study First Submitted QC: 2022-06-10
• Study First Posted: 2022-06-15
• Study Start: 2022-10-13
• Primary Completion: 2024-06-13
• Study Completion: 2024-06-13
• Last Update Submitted: 2024-07-23
• Last Update Posted: 2024-07-24

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 52

Inclusion Criteria

• Retrospective part of the research:

The medical records of CRéER patients with the following criteria will be included in the retrospective part of the research by an investigator:

1. With pharmacoresistant epilepsy, i.e. a history of failure of two well-conducted and well-tolerated antiepileptic treatment regimens, either as monotherapy or as combination therapy,

2. Receiving or having received a treatment combining stiripentol and carbamazepine for a period of at least 15 days,

3. Having at least one evaluation data after the initiation of treatment with stiripentol and carbamazepine (a follow-up visit after the initiation of the study treatment),

4. For which an information note indicating the possibility of opposing the processing of data has been provided.

   • Prospective part of the research:

Among the patients whose medical records are included in the retrospective part of the research, those who meet the following criteria will be able to participate in the prospective part of the research:

1. Currently treated with stiripentol in combination with carbamazepine for at least 15 days and still being followed in the center,
2. Weighing at least 5 kg (minimum weight in accordance with the blood volume taken),
3. Having read, or whose parents have read, the information note and signed the consent form. For children, if their level of understanding allows it, their consent will also be sought,
4. Having sufficient knowledge, or whose parents or legal guardians have sufficient knowledge, of the French language to read, understand and complete the research documents,
5. Members or beneficiaries of a social security scheme. These patients, both children and adults, will be welcomed at the CIC.

Exclusion Criteria

• Retrospective part of the research : Patients objecting to the collection of their data will not participate in the research.

• Prospective part of the research :

Patients with the following criteria will not be able to participate in the prospective part of the research:

1. Participating simultaneously in another interventional clinical trial or in a period of exclusion following a previous trial,
2. Whose state of health does not allow him to give his consent,
3. Under guardianship or curatorship,
4. Under judicial protection or person deprived of liberty.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
stiripentol	Stiripentol	-

Primary Outcome Measures

Name	Time	Method
Previous antiepileptic treatments received	through study completion, an average of 1 year	proportion of patients having received each antiepileptic treatment; for the treatments received, description of the reasons for stopping.
Surgery	through study completion, an average of 1 year	proportion of patients having undergone epilepsy surgery.
monthly seizures over the time	through study completion, an average of 1 year	description of the average number of monthly seizures since the last visit (total and by type of seizure)
status epilepticus in the past	through study completion, an average of 1 year	proportion of patients who presented with status epilepticus
emergency room consultation and/or hospitalization over the time	through study completion, an average of 1 year	Proportion of patients requiring an emergency room consultation and/or hospitalized during the period preceding the visit.
emergency antiepileptic treatment over the time	through study completion, an average of 1 year	Proportion of patients taking emergency antiepileptic treatment in the period before each visit
Age	through study completion, an average of 1 year	Age of onset, nature of the first seizure and description of the type of the first seizure.
Adjustment of carbamazepine treatment over the time	through study completion, an average of 1 year	mean and median duration of treatment, mean and median daily dose received, dose change(s) during treatment...
Adjustment of stiripentol treatment over the time	through study completion, an average of 1 year	mean and median duration of treatment, mean and median daily dose received, dose change(s) during treatment...
Adjustment of concomitant treatments over the time	through study completion, an average of 1 year	continuation without change, change in dosage, discontinuation
frequency of the status epileticus over the time	through study completion, an average of 1 year	frequency of status epilepticus
duration of the status epileticus over the time	through study completion, an average of 1 year	duration of status epilepticus

Secondary Outcome Measures

Name	Time	Method
Response to the combination	through study completion, an average of 1 year	The rate of patients responding to the combination will be defined by the percentage of patients in whom a 50% reduction in visible seizures will be observed since the initiation of the carbamazepine and stiripentol combination.; Will be estimated: oThe percentage of responder patients at the post-initiation visit of the combination (first follow-up visit after initiation of stiripentol) oThe percentage of responder patients during treatment regardless of the duration of treatment after which the response is obtained.; oThe cumulative incidence of responder patients.
Tolerance of the combination.	through study completion, an average of 1 year	Proportion of patients with Adverse Effects (AEs) related to the combination or to one of the treatments,
Retention rate of the combination	through study completion, an average of 1 year	The retention rate of the combination will correspond to the proportion of patients still receiving the combination (carbamazepine and stiripentol) at a given time.
Frequency and nature of the adverse events over the time	through study completion, an average of 1 year	Frequency and nature of the adverse events over the time
Abnormal biological parameters (NFS and liver)	through study completion, an average of 1 year	proportion of patients with abnormal laboratory values (NFS and liver function) during

Trial Locations

Locations (1)

Centre de Référence Epilepsies Rares / Centre Investigation Clinique (CIC) - Hôpital Necker-Enfants Malades; 🇫🇷 Paris, France