Dravet SyndromeNCT02239276
Expanded Access Use of Stiripentol in Dravet Syndrome or Sodium Channel Mutation Epileptic Encephalopathies
A study testing Stiripentol in people with Dravet Syndrome and Epileptic Encephalopathies Associated With SCN1A Mutations, run by Cook Children's Health Care System at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 months to 18 years
- Conditions the study is for
- Dravet Syndrome, Epileptic Encephalopathies Associated With SCN1A Mutations
- What is being tested?
- Stiripentol
- How many people
- Not given
- Where
- 1 siteUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 18 years
- All sexes
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Cook Children's Medical Center
Fort Worth, Texas, 76104
About this study
Official title: 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.
Read the 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.
- Being tested
- Stiripentol
- Keywords
- Stiripentol, Dravet syndrome, Epilepsy, Epileptic Encephalopathies
- Medical subject headings
- Epilepsies, Myoclonic, Epilepsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
StiripentolDrug
Also called Diacomit
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.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Sep 2014 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Dravet SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Cook Children's Health Care System
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Scott Perry, MD, Principal investigator, Cook Children's Health Care System
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02239276
- Sponsor's protocol ID
- 2014-047
- Status last verified
- Jan 2020
- First submitted
- 10 Sep 2014
- Last updated
- 5 Feb 2020
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Feb 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access Use of Stiripentol in Participants With Dravet Syndrome or Epileptic Encephalopathies Associated With Sodium Channel Mutations
- ClinicalTrials.gov ID
- NCT02239276
- Acronym / sponsor ID
- 2014-047
- Keywords
- Stiripentol, Dravet syndrome, Epilepsy, Epileptic Encephalopathies
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Cook Children's Health Care SystemUniversity, hospital or other
- First posted
- 12 Sep 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
Who may qualify
- Ages
- 6 months to 18 years
- Sex
- All
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- 6 months and older
- Diagnosis of Dravet Syndrome or epileptic encephalopathies associated with SCN1A mutations defined as:
- Failure of at least 2 therapeutic anticonvulsants (excluding Na Channel blockers) indicative of intractable seizures
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| StiripentolAlso: DiacomitThe 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. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Feb 2020.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Scott Perry, MD principal investigator
Cook Children's Health Care System
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Stiripentol in people with Dravet Syndrome and Epileptic Encephalopathies Associated With SCN1A Mutations, run by Cook Children's Health Care System at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 5 Feb 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Feb 2020.