ClinicalTrials.gov
ClinicalTrials.gov Menu

Compassionate Use of Stiripentol in Dravet Syndrome

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01835314
Recruitment Status : Available
First Posted : April 18, 2013
Last Update Posted : April 5, 2017
Sponsor:
Information provided by (Responsible Party):
University of Colorado, Denver

Study Description
Go to 
Study Description Study Design Interventions Eligibility Criteria Contacts and Locations More Information
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.

Condition or disease Intervention/treatment
Dravet Syndrome Drug: Stiripentol

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.

Study Design
Go to 
Study Description Study Design Interventions Eligibility Criteria Contacts and Locations More Information

Study Type : Expanded Access
Official Title: Compassionate Use of Stiripentol in Dravet Syndrome

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Interventions
Go to 
Study Description Study Design Interventions Eligibility Criteria Contacts and Locations More Information

Intervention Details:
    Drug: Stiripentol
    Stiripentol 5-50mg/kg/d divided twice or three times a day by mouth
Eligibility Criteria
Go to 
Study Description Study Design Interventions Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   1 Year to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
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.
Contacts and Locations
Go to 
Study Description Study Design Interventions Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01835314


Contacts
Contact: Kelly Knupp, MD 720-777-6895 kelly.knupp@childrenscolorado.org
Contact: Alicia M Camuto, BA 720-777-5514 alicia.camuto@childrenscolorado.org

Locations
United States, Colorado
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Contact: Kelly Knupp, MD    720-777-6895    kelly.knupp@childrenscolorado.org   
Contact: Alicia Camuto, BA    720-777-5514    alicia.camuto@childrenscolorado.org   
Principal Investigator: Kelly Knupp, MD         
Sponsors and Collaborators
University of Colorado, Denver
Investigators
Principal Investigator: Kelly Knupp, MD University of Colorado/Children's Hospital Colorado
More Information
Go to 
Study Description Study Design Interventions Eligibility Criteria Contacts and Locations More Information

Responsible Party: University of Colorado, Denver
ClinicalTrials.gov Identifier: NCT01835314     History of Changes
Other Study ID Numbers: 12-0315
First Posted: April 18, 2013    Key Record Dates
Last Update Posted: April 5, 2017
Last Verified: December 2016

Additional relevant MeSH terms:
Syndrome
Epilepsies, Myoclonic
Disease
Pathologic Processes
Epilepsy
 Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Stiripentol
Anticonvulsants