Back to resultsStudy of Interest of Stiripentol and Carbamazepine in the Treatment of Patients With Pharmacoresistant Focal Epilepsies
Primary Purpose
Pharmacoresistant Focal Epilepsies
Status
Recruiting
Phase
Phase 4
Locations
France
Study Type
Interventional
Intervention
Stiripentol
Sponsored by
About this trial
This is an interventional treatment trial for Pharmacoresistant Focal Epilepsies
Eligibility Criteria
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:
- 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,
- Receiving or having received a treatment combining stiripentol and carbamazepine for a period of at least 15 days,
- 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),
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:
- Currently treated with stiripentol in combination with carbamazepine for at least 15 days and still being followed in the center,
- Weighing at least 5 kg (minimum weight in accordance with the blood volume taken),
- 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,
- Having sufficient knowledge, or whose parents or legal guardians have sufficient knowledge, of the French language to read, understand and complete the research documents,
- 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:
- Participating simultaneously in another interventional clinical trial or in a period of exclusion following a previous trial,
- Whose state of health does not allow him to give his consent,
- Under guardianship or curatorship,
- Under judicial protection or person deprived of liberty.
Sites / Locations
- Centre de Référence Epilepsies Rares / Centre Investigation Clinique (CIC) - Hôpital Necker-Enfants MaladesRecruiting
Arms of the Study
Arm 1
Arm Type
Other
Arm Label
stiripentol
Arm Description
Outcomes
Primary Outcome Measures
Age
Age of onset, nature of the first seizure and description of the type of the first seizure.
Previous antiepileptic treatments received
proportion of patients having received each antiepileptic treatment; for the treatments received, description of the reasons for stopping.
Surgery
proportion of patients having undergone epilepsy surgery.
Adjustment of carbamazepine treatment over the time
mean and median duration of treatment, mean and median daily dose received, dose change(s) during treatment...
Adjustment of stiripentol treatment over the time
mean and median duration of treatment, mean and median daily dose received, dose change(s) during treatment...
Adjustment of concomitant treatments over the time
continuation without change, change in dosage, discontinuation
monthly seizures over the time
description of the average number of monthly seizures since the last visit (total and by type of seizure)
status epilepticus in the past
proportion of patients who presented with status epilepticus
duration of the status epileticus over the time
duration of status epilepticus
emergency room consultation and/or hospitalization over the time
Proportion of patients requiring an emergency room consultation and/or hospitalized during the period preceding the visit.
emergency antiepileptic treatment over the time
Proportion of patients taking emergency antiepileptic treatment in the period before each visit
frequency of the status epileticus over the time
frequency of status epilepticus
Secondary Outcome Measures
Retention rate of the combination
The retention rate of the combination will correspond to the proportion of patients still receiving the combination (carbamazepine and stiripentol) at a given time.
Response to the combination
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.
Proportion of patients with Adverse Effects (AEs) related to the combination or to one of the treatments,
Frequency and nature of the adverse events over the time
Frequency and nature of the adverse events over the time
Abnormal biological parameters (NFS and liver)
proportion of patients with abnormal laboratory values (NFS and liver function) during
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05419180
Brief Title
Study of Interest of Stiripentol and Carbamazepine in the Treatment of Patients With Pharmacoresistant Focal Epilepsies
Official Title
Study of the Interest of the Combination of Stiripentol (Diacomit®) and Carbamazepine in the Treatment of Patients With Pharmacoresistant Focal Epilepsies
Study Type
Interventional
2. Study Status
Record Verification Date
May 2022
Overall Recruitment Status
Recruiting
Study Start Date
October 13, 2022 (Actual)
Primary Completion Date
September 2023 (Anticipated)
Study Completion Date
January 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Biocodex
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pharmacoresistant Focal Epilepsies
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Model Description
retrospective part and prospective part
Masking
None (Open Label)
Allocation
N/A
Enrollment
100 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
stiripentol
Arm Type
Other
Intervention Type
Drug
Intervention Name(s)
Stiripentol
Intervention Description
Since the treatment usually received by patients should not be modified, the sponsor will not intervene in any way in the therapeutic management of patients.
Primary Outcome Measure Information:
Title
Age
Description
Age of onset, nature of the first seizure and description of the type of the first seizure.
Time Frame
through study completion, an average of 1 year
Title
Previous antiepileptic treatments received
Description
proportion of patients having received each antiepileptic treatment; for the treatments received, description of the reasons for stopping.
Time Frame
through study completion, an average of 1 year
Title
Surgery
Description
proportion of patients having undergone epilepsy surgery.
Time Frame
through study completion, an average of 1 year
Title
Adjustment of carbamazepine treatment over the time
Description
mean and median duration of treatment, mean and median daily dose received, dose change(s) during treatment...
Time Frame
through study completion, an average of 1 year
Title
Adjustment of stiripentol treatment over the time
Description
mean and median duration of treatment, mean and median daily dose received, dose change(s) during treatment...
Time Frame
through study completion, an average of 1 year
Title
Adjustment of concomitant treatments over the time
Description
continuation without change, change in dosage, discontinuation
Time Frame
through study completion, an average of 1 year
Title
monthly seizures over the time
Description
description of the average number of monthly seizures since the last visit (total and by type of seizure)
Time Frame
through study completion, an average of 1 year
Title
status epilepticus in the past
Description
proportion of patients who presented with status epilepticus
Time Frame
through study completion, an average of 1 year
Title
duration of the status epileticus over the time
Description
duration of status epilepticus
Time Frame
through study completion, an average of 1 year
Title
emergency room consultation and/or hospitalization over the time
Description
Proportion of patients requiring an emergency room consultation and/or hospitalized during the period preceding the visit.
Time Frame
through study completion, an average of 1 year
Title
emergency antiepileptic treatment over the time
Description
Proportion of patients taking emergency antiepileptic treatment in the period before each visit
Time Frame
through study completion, an average of 1 year
Title
frequency of the status epileticus over the time
Description
frequency of status epilepticus
Time Frame
through study completion, an average of 1 year
Secondary Outcome Measure Information:
Title
Retention rate of the combination
Description
The retention rate of the combination will correspond to the proportion of patients still receiving the combination (carbamazepine and stiripentol) at a given time.
Time Frame
through study completion, an average of 1 year
Title
Response to the combination
Description
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.
Time Frame
through study completion, an average of 1 year
Title
Tolerance of the combination.
Description
Proportion of patients with Adverse Effects (AEs) related to the combination or to one of the treatments,
Time Frame
through study completion, an average of 1 year
Title
Frequency and nature of the adverse events over the time
Description
Frequency and nature of the adverse events over the time
Time Frame
through study completion, an average of 1 year
Title
Abnormal biological parameters (NFS and liver)
Description
proportion of patients with abnormal laboratory values (NFS and liver function) during
Time Frame
through study completion, an average of 1 year
Other Pre-specified Outcome Measures:
Title
Maximum Concentration (Cmax) for stiripentol, carbamazepines and its metabolites
Description
Cmax calculation
Time Frame
through study completion, an average of 1 year
Title
Area under the plasma concentration versus time curve (AUC) 0-8 for stiripentol, carbamazepines and its metabolites
Description
AUC0-8 calculation
Time Frame
through study completion, an average of 1 year
Title
Area under the plasma concentration versus time curve (AUC) 0-24 for stiripentol, carbamazepines and its metabolites
Description
AUC0-24 calculation
Time Frame
through study completion, an average of 1 year
Title
Time of Maximum (Tmax)concentration for stiripentol, carbamazepines and its metabolites
Description
Tmax calculation
Time Frame
through study completion, an average of 1 year
Title
half-life (t1/2) for stiripentol, carbamazepines and its metabolites
Description
t1/2 calculation
Time Frame
through study completion, an average of 1 year
Title
Pre-dose trough concentration (Ctrough) for stiripentol, carbamazepines and its metabolites
Description
Ctrough calculation
Time Frame
through study completion, an average of 1 year
10. Eligibility
Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
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:
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,
Receiving or having received a treatment combining stiripentol and carbamazepine for a period of at least 15 days,
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),
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:
Currently treated with stiripentol in combination with carbamazepine for at least 15 days and still being followed in the center,
Weighing at least 5 kg (minimum weight in accordance with the blood volume taken),
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,
Having sufficient knowledge, or whose parents or legal guardians have sufficient knowledge, of the French language to read, understand and complete the research documents,
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:
Participating simultaneously in another interventional clinical trial or in a period of exclusion following a previous trial,
Whose state of health does not allow him to give his consent,
Under guardianship or curatorship,
Under judicial protection or person deprived of liberty.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Roxane NOËL, PharmD
Phone
+33 1 44 86 75 79
Email
r.noel@biocodex.fr
First Name & Middle Initial & Last Name or Official Title & Degree
Carine FRANCOIS, PharmD
Phone
+33 1 44 86 82 28
Email
c.francois@biocodex.fr
Facility Information:
Facility Name
Centre de Référence Epilepsies Rares / Centre Investigation Clinique (CIC) - Hôpital Necker-Enfants Malades
City
Paris
ZIP/Postal Code
75015
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sandra COLAS
Phone
+33 1 71 19 64 32
Email
sandra.colas@aphp.fr
First Name & Middle Initial & Last Name & Degree
Rima NABBOUT, Pr
12. IPD Sharing Statement
Learn more about this trial
Study of Interest of Stiripentol and Carbamazepine in the Treatment of Patients With Pharmacoresistant Focal Epilepsies
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