Compassionate Use of Stiripentol in Dravet Syndrome

NCT ID: NCT01835314

Last Updated: 2019-10-04

Basic Information

• Recruitment Status: NO_LONGER_AVAILABLE
• Study Classification: EXPANDED_ACCESS

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.

Conditions

Dravet Syndrome

Interventions

Stiripentol

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

Intervention Type: DRUG

Eligibility Criteria

Inclusion 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.

• Minimum Eligible Age: 1 Year
• Maximum Eligible Age: 21 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University of Colorado, Denver

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Kelly Knupp, MD

Role: PRINCIPAL_INVESTIGATOR

University of Colorado/Children's Hospital Colorado

Locations

Children's Hospital Colorado

Aurora, Colorado, United States

Countries

United States

Other Identifiers

12-0315

Identifier Type: -

Identifier Source: org_study_id