Compassionate Use of Stiripentol in Dravet Syndrome
Expanded access is currently available for this treatment.
Verified March 2016 by University of Colorado, Denver
Sponsor:
University of Colorado, Denver
Information provided by (Responsible Party):
University of Colorado, Denver
ClinicalTrials.gov Identifier:
NCT01835314
First received: April 15, 2013
Last updated: March 17, 2016
Last verified: March 2016
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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:
pyridoxal 5'-phosphate-dependent epilepsy
U.S. FDA Resources
Further study details as provided by University of Colorado, Denver:
Intervention Details:
Detailed Description:
-
Drug: Stiripentol
Stiripentol 5-50mg/kg/d divided twice or three times a day by mouth
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) |
| 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
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: | March 17, 2016 |
| Health Authority: | United States: Institutional Review Board United States: Food and Drug Administration United States: Federal Government |
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 September 28, 2016