Compassionate Use of Stiripentol in Dravet Syndrome

Expanded access is currently available for this treatment.
Verified December 2013 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: December 16, 2013
Last verified: December 2013
  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:


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

Intervention Details:
    Drug: Stiripentol
    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
Genders Eligible for Study:   Both
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

No publications provided

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: December 16, 2013
Health Authority: United States: Institutional Review Board
United States: Food and Drug Administration
United States: Federal Government

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

ClinicalTrials.gov processed this record on October 20, 2014