Expanded Access Use of Stiripentol in Dravet Syndrome or Sodium Channel Mutation Epileptic Encephalopathies
Primary Purpose
Dravet Syndrome, Epileptic Encephalopathies Associated With SCN1A Mutations
Status
No longer available
Phase
Locations
United States
Study Type
Expanded Access
Intervention
Stiripentol
Sponsored by
About this trial
This is an expanded access trial for Dravet Syndrome focused on measuring Stiripentol, Dravet syndrome, Epilepsy, Epileptic Encephalopathies
Eligibility Criteria
Inclusion Criteria:
- 6 months and older
Diagnosis of Dravet Syndrome or epileptic encephalopathies associated with SCN1A mutations defined as:
- A documented gene mutation reported to result in Dravet syndrome phenotype; OR
- Clinical confirmation of Dravet syndrome by two pediatric neurologists; OR
- Clinical confirmation of other epileptic encephalopathies associated with sodium channel mutations
- Failure of at least 2 therapeutic anticonvulsants (excluding Na Channel blockers) indicative of intractable seizures
Exclusion Criteria:
- Hypersensitivity to the active substance or to any of the excipients
- Past history of psychoses in the form of episodes of delirium
- Impaired hepatic and/or renal function, defined as creatinine >2 and/or transaminase >4xULN
Sites / Locations
- Cook Children's Medical Center
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT02239276
First Posted
September 10, 2014
Last Updated
January 31, 2020
Sponsor
Cook Children's Health Care System
1. Study Identification
Unique Protocol Identification Number
NCT02239276
Brief Title
Expanded Access Use of Stiripentol in Dravet Syndrome or Sodium Channel Mutation Epileptic Encephalopathies
Official Title
Expanded Access Use of Stiripentol in Participants With Dravet Syndrome or Epileptic Encephalopathies Associated With Sodium Channel Mutations
Study Type
Expanded Access
2. Study Status
Record Verification Date
January 2020
Overall Recruitment Status
No longer available
Study Start Date
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Primary Completion Date
undefined (undefined)
Study Completion Date
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3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Cook Children's Health Care System
4. Oversight
5. Study Description
Brief Summary
This is an expanded access use of Stiripentol in Dravet Syndrome or epileptic encephalopathies associated with sodium channel mutations who have failed other drugs in an effort to give them the best chance at seizure control and quality of life. As a treatment protocol and not a research study, children will only be monitored on a clinical basis for seizure improvement and side effects predominantly by parent and caregiver report.
Detailed Description
The initial dose of Stiripentol will be determined by the prescribing neurologist and titrated up to an initial goal dose of 50 mg/kg/day divided into 2 to 3 doses per day. Further dose increases by 10-20 mg/kg/day increments up to a max of 100 mg/kg/day or 4000 mg total daily dose may be necessary for improved seizure control.
Stiripentol is available as gelatin capsules and powder sachets (250 mg, 500 mg). The same granule formulation (i.e. active, PVP and portion of sodium starch glycolate) used for the capsule is used in the final powder blend with a few additional excipients. Depending upon patient weight, the 250 mg or 500 mg formulation will be utilized for each participant.
Caretakers will be queried about common adverse effects including drowsiness, tremor, ataxis, nausea, anorexia, weight loss, and emesis. Intolerable adverse effects will prompt dose reduction or withholding medication.
Monitoring of these and other potential AEs will occur during study visits and participant-initiated telephone calls throughout the study. Safety events and tolerability will be recorded as adverse events (AE) or serious adverse events (SAE).
Physical examination, weight, vital signs, and laboratory tests (cbc, complete metabolic panel, and AED levels) will be conducted at baseline and at least every 6 months and as clinically warranted.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Dravet Syndrome, Epileptic Encephalopathies Associated With SCN1A Mutations
Keywords
Stiripentol, Dravet syndrome, Epilepsy, Epileptic Encephalopathies
7. Study Design
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
Stiripentol
Other Intervention Name(s)
Diacomit
Intervention Description
The initial dose of Stiripentol will be determined by the prescribing neurologist and titrated up to an initial goal dose of 50 mg/kg/day divided into 2 to 3 doses per day. Further dose increases by 10-20 mg/kg/day increments up to a max of 100 mg/kg/day or 4000 mg total daily dose may be necessary for improved seizure control.
10. Eligibility
Sex
All
Minimum Age & Unit of Time
6 Months
Maximum Age & Unit of Time
18 Years
Eligibility Criteria
Inclusion Criteria:
6 months and older
Diagnosis of Dravet Syndrome or epileptic encephalopathies associated with SCN1A mutations defined as:
A documented gene mutation reported to result in Dravet syndrome phenotype; OR
Clinical confirmation of Dravet syndrome by two pediatric neurologists; OR
Clinical confirmation of other epileptic encephalopathies associated with sodium channel mutations
Failure of at least 2 therapeutic anticonvulsants (excluding Na Channel blockers) indicative of intractable seizures
Exclusion Criteria:
Hypersensitivity to the active substance or to any of the excipients
Past history of psychoses in the form of episodes of delirium
Impaired hepatic and/or renal function, defined as creatinine >2 and/or transaminase >4xULN
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Scott Perry, MD
Organizational Affiliation
Cook Children's Health Care System
Official's Role
Principal Investigator
Facility Information:
Facility Name
Cook Children's Medical Center
City
Fort Worth
State/Province
Texas
ZIP/Postal Code
76104
Country
United States
12. IPD Sharing Statement
Links:
URL
http://www.cookchildrens.org/Pages/Default.aspx
Description
Cook Children's Health Care System website
Learn more about this trial
Expanded Access Use of Stiripentol in Dravet Syndrome or Sodium Channel Mutation Epileptic Encephalopathies
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