Expanded Access Use of Stiripentol in Dravet Syndrome or Sodium Channel Mutation Epileptic Encephalopathies

January 31, 2020 updated by: Cook Children's Health Care System

Expanded Access Use of Stiripentol in Participants With Dravet Syndrome or Epileptic Encephalopathies Associated With Sodium Channel Mutations

This is an expanded access use of Stiripentol in Dravet Syndrome or epileptic encephalopathies associated with sodium channel mutations who have failed other drugs in an effort to give them the best chance at seizure control and quality of life. As a treatment protocol and not a research study, children will only be monitored on a clinical basis for seizure improvement and side effects predominantly by parent and caregiver report.

Study Overview

Status

No longer available

Conditions

Intervention / Treatment

Detailed Description

The initial dose of Stiripentol will be determined by the prescribing neurologist and titrated up to an initial goal dose of 50 mg/kg/day divided into 2 to 3 doses per day. Further dose increases by 10-20 mg/kg/day increments up to a max of 100 mg/kg/day or 4000 mg total daily dose may be necessary for improved seizure control.

Stiripentol is available as gelatin capsules and powder sachets (250 mg, 500 mg). The same granule formulation (i.e. active, PVP and portion of sodium starch glycolate) used for the capsule is used in the final powder blend with a few additional excipients. Depending upon patient weight, the 250 mg or 500 mg formulation will be utilized for each participant.

Caretakers will be queried about common adverse effects including drowsiness, tremor, ataxis, nausea, anorexia, weight loss, and emesis. Intolerable adverse effects will prompt dose reduction or withholding medication.

Monitoring of these and other potential AEs will occur during study visits and participant-initiated telephone calls throughout the study. Safety events and tolerability will be recorded as adverse events (AE) or serious adverse events (SAE).

Physical examination, weight, vital signs, and laboratory tests (cbc, complete metabolic panel, and AED levels) will be conducted at baseline and at least every 6 months and as clinically warranted.

Study Type

Expanded Access

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Texas
      • Fort Worth, Texas, United States, 76104
        • Cook Children's Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 months to 18 years (Child, Adult)

Accepts Healthy Volunteers

N/A

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • 6 months and older

  • Diagnosis of Dravet Syndrome or epileptic encephalopathies associated with SCN1A mutations defined as:

    • A documented gene mutation reported to result in Dravet syndrome phenotype; OR
    • Clinical confirmation of Dravet syndrome by two pediatric neurologists; OR
    • Clinical confirmation of other epileptic encephalopathies associated with sodium channel mutations
  • Failure of at least 2 therapeutic anticonvulsants (excluding Na Channel blockers) indicative of intractable seizures

Exclusion Criteria:

  • Hypersensitivity to the active substance or to any of the excipients
  • Past history of psychoses in the form of episodes of delirium
  • Impaired hepatic and/or renal function, defined as creatinine >2 and/or transaminase >4xULN

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Cook Children's Health Care System

Investigators

  • Principal Investigator: Scott Perry, MD, Cook Children's Health Care System

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

September 10, 2014

First Submitted That Met QC Criteria

September 10, 2014

First Posted (Estimate)

September 12, 2014

Study Record Updates

Last Update Posted (Actual)

February 5, 2020

Last Update Submitted That Met QC Criteria

January 31, 2020

Last Verified

January 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2014-047

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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