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

NCT02239276 | Unknown

• 1 other identifier: 2014-047
• Study Type: expanded_access
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Dravet Syndrome; Epileptic Encephalopathies Associated With SCN1A Mutations

Keywords

Stiripentol; Dravet syndrome; Epilepsy; Epileptic Encephalopathies

Interventions

Stiripentol DRUG

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.

Also known as: Diacomit

Eligibility Criteria

• Age: 6 Months - 18 Years
• Sex: all
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Cook Children's Health Care System: lead

Study Sites (1)

Cook Children's Medical Center

Fort Worth, Texas, 76104, United States

Location

Related Links

• Cook Children's Health Care System website

MeSH Terms

Conditions

Epilepsies, Myoclonic; Epilepsy

Interventions

stiripentol

Condition Hierarchy (Ancestors)

Epilepsy, Generalized; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epileptic Syndromes

Study Officials

• Scott Perry, MD

  Cook Children's Health Care System

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: expanded access
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 10, 2014
• First Posted: September 12, 2014
• Last Updated: February 5, 2020

Record last verified: 2020-01

Locations

US (1)