Compassionate Use of Stiripentol in Dravet Syndrome

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Dravet Syndrome
Related Access Program
M. Scott Perry – Dravet Syndrome M. Scott Perry – Dravet Syndrome University of Colorado, Denver – Dravet Syndrome Children’s Hospital Medical Center, Cincinnati – Dravet Syndrome
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Brief Title

Compassionate Use of Stiripentol in Dravet Syndrome

Official Title

Compassionate Use of Stiripentol in Dravet Syndrome

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.

Study Type

Expanded Access

Condition

Dravet Syndrome

Intervention

Stiripentol

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status

Drug

Eligibility 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.

Gender

All

Ages

1 Year - 21 Years

Contacts

Kelly Knupp, MD, ,

Location Countries

United States

Location Countries

United States

Administrative Informations

NCT ID

NCT01835314

Organization ID

12-0315

Responsible Party

Sponsor

Study Sponsor

University of Colorado, Denver

Study Sponsor

Kelly Knupp, MD, Principal Investigator, University of Colorado/Children's Hospital Colorado

Verification Date

October 2019