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

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Brief Title

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

Official Title

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

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.
    

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




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

        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
      

Gender

All

Ages

6 Months - 18 Years


Contacts

Scott Perry, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT02239276

Organization ID

2014-047


Responsible Party

Sponsor

Study Sponsor

Cook Children's Health Care System


Study Sponsor

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


Verification Date

January 2020