Compassionate Use of Stiripentol in Dravet Syndrome

Expanded access is currently available for this treatment.

Verified December 2016 by University of Colorado, Denver

Sponsor:

Information provided by (Responsible Party):

University of Colorado, Denver

ClinicalTrials.gov Identifier:

NCT01835314

First received: April 15, 2013

Last updated: April 3, 2017

Last verified: December 2016

History of Changes

Purpose

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	Intervention
Dravet Syndrome	Drug: Stiripentol


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

Resource links provided by NLM:

Genetics Home Reference related topics: CHD2 myoclonic encephalopathy pyridoxal 5'-phosphate-dependent epilepsy

Genetic and Rare Diseases Information Center resources: Dravet Syndrome

U.S. FDA Resources

Further study details as provided by University of Colorado, Denver:

Intervention Details:

Stiripentol 5-50mg/kg/d divided twice or three times a day by mouth

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.

Eligibility


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

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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: 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


Responsible Party:	University of Colorado, Denver
ClinicalTrials.gov Identifier:	NCT01835314 History of Changes
Other Study ID Numbers:	12-0315
Study First Received:	April 15, 2013
Last Updated:	April 3, 2017

Additional relevant MeSH terms:


Syndrome Epilepsies, Myoclonic Disease Pathologic Processes Epilepsy	Brain Diseases Central Nervous System Diseases Nervous System Diseases Stiripentol Anticonvulsants

ClinicalTrials.gov processed this record on June 27, 2017

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