Back to resultsCompassionate Use of Stiripentol in Dravet Syndrome
Primary Purpose
Dravet Syndrome
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
Eligibility Criteria
Children with genetic confirmation of Dravet syndrome (a documented sodium channel, voltage-gated, type I, alpha subunit (SCN1A) mutation) or clinical confirmation of Dravet syndrome by two pediatric neurologist will be considered to have Dravet syndrome. In order to enter the treatment protocol there will be documented treatment failure of at least two therapeutic anticonvulsants excluding Na channel blockers. Anticonvulsants that are Na channel blockers such as carbamazepine and lamotrigine as known to provoke seizures in this patient population.
Sites / Locations
- Children's Hospital Colorado
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT01835314
First Posted
April 15, 2013
Last Updated
October 2, 2019
Sponsor
University of Colorado, Denver
1. Study Identification
Unique Protocol Identification Number
NCT01835314
Brief Title
Compassionate Use of Stiripentol in Dravet Syndrome
Official Title
Compassionate Use of Stiripentol in Dravet Syndrome
Study Type
Expanded Access
2. Study Status
Record Verification Date
October 2019
Overall Recruitment Status
No longer available
Study Start Date
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Primary Completion Date
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Study Completion Date
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3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University of Colorado, Denver
4. Oversight
5. Study Description
Brief Summary
Compassionate use of Stiripentol in Dravet Syndrome. This is a treatment protocol, not a research study, therefore children will only be monitored on a clinical basis for seizure improvement predominantly by parent and caregiver report.
Detailed Description
This is a treatment protocol for compassionate use, not a research study, therefore children will only be monitored on a clinical basis for seizure improvement predominantly by parent and caregiver report.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Dravet Syndrome
7. Study Design
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
Stiripentol
Intervention Description
Stiripentol 5-50mg/kg/d divided twice or three times a day by mouth
10. Eligibility
Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
21 Years
Eligibility Criteria
Children with genetic confirmation of Dravet syndrome (a documented sodium channel, voltage-gated, type I, alpha subunit (SCN1A) mutation) or clinical confirmation of Dravet syndrome by two pediatric neurologist will be considered to have Dravet syndrome. In order to enter the treatment protocol there will be documented treatment failure of at least two therapeutic anticonvulsants excluding Na channel blockers. Anticonvulsants that are Na channel blockers such as carbamazepine and lamotrigine as known to provoke seizures in this patient population.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Kelly Knupp, MD
Organizational Affiliation
University of Colorado/Children's Hospital Colorado
Official's Role
Principal Investigator
Facility Information:
Facility Name
Children's Hospital Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Compassionate Use of Stiripentol in Dravet Syndrome
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