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

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ClinicalTrials.gov Identifier: NCT02239276
Expanded Access Status : No longer available
First Posted : September 12, 2014
Last Update Posted : January 31, 2019
Sponsor:
Information provided by (Responsible Party):
M. Scott Perry, Cook Children's Health Care System

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.

Condition or disease Intervention/treatment
Dravet Syndrome Epileptic Encephalopathies Associated With SCN1A Mutations Drug: Stiripentol

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.


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Study Type : Expanded Access
Official Title: Expanded Access Use of Stiripentol in Participants With Dravet Syndrome or Epileptic Encephalopathies Associated With Sodium Channel Mutations



Intervention Details:
  • Drug: Stiripentol
    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.
    Other Name: Diacomit

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Ages Eligible for Study:   6 Months to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02239276


Locations
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United States, Texas
Cook Children's Medical Center
Fort Worth, Texas, United States, 76104
Sponsors and Collaborators
M. Scott Perry
Investigators
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Principal Investigator: Scott Perry, MD Cook Children's Health Care System

Additional Information:
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Responsible Party: M. Scott Perry, Medical Director, Epilepsy Monitoring Unit and Tuberous Sclerosis Complex Clinic, Cook Children's Health Care System
ClinicalTrials.gov Identifier: NCT02239276     History of Changes
Other Study ID Numbers: 2014-047
First Posted: September 12, 2014    Key Record Dates
Last Update Posted: January 31, 2019
Last Verified: January 2019

Keywords provided by M. Scott Perry, Cook Children's Health Care System:
Stiripentol
Dravet syndrome
Epilepsy
Epileptic Encephalopathies

Additional relevant MeSH terms:
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Syndrome
Brain Diseases
Epilepsy
Epilepsies, Myoclonic
Spasms, Infantile
Rett Syndrome
Disease
Pathologic Processes
Central Nervous System Diseases
Nervous System Diseases
Epilepsy, Generalized
Epileptic Syndromes
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Stiripentol
Anticonvulsants