Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex
Purpose
Study design is a Phase IIb prospective multi-center, randomized, placebo-controlled, double-blind clinical trial. The goal will be to enroll 80 infants with Tuberous Sclerosis Complex who are less than 6 months of age prior to the onset of their first seizure
Condition
- Tuberous Sclerosis Complex
Eligibility
- Eligible Ages
- Between 1 Day and 6 Months
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- less than or equal to 6 months of age 2. No history of seizures or infantile spasms, or evidence of subclinical electrographic seizures on a previous video EEG 3. Meet genetic or clinical diagnostic criteria for TSC, the latter based on current recommendations for diagnostic evaluation, such as physical exam, neuroimaging, echocardiogram
Exclusion Criteria
- Is greater than 6 months of age 2. Has not been diagnosed with TSC 3. History of seizures or infantile spasms, or evidence of subclinical electrographic seizures on a previous video EEG 4. Has received any anticonvulsant medication including vigabatrin, other anti-seizure therapeutic agent including cannabidiol 5. Has received an oral mTOR inhibitor such as everolimus or sirolimus 6. Has taken an investigational drug, including but not limited to cannabidiol, as part of a research study 30 days prior to enrollment, or plans on taking an investigational drug at any time during the duration of the study 7. Is currently enrolled, or plans on enrolling at any time during the duration of the study, in an experimental behavioral early intervention study 8. Has a history of being born prematurely (born less than <30 weeks gestation at the time of delivery)
Study Design
- Phase
- Phase 2
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Prevention
- Masking
- Triple (Participant, Care Provider, Investigator)
Arm Groups
Arm | Description | Assigned Intervention |
---|---|---|
Placebo Comparator Vigabatrin or Placebo |
Vigabatrin or Placebo is given for administration, the entire content of one sachet (500 mg active drug) is dissolved in 10 ml water for oral administration that is dosed according to body weight 50-150 mg/kg/day divided BID. Dosing will follow established recommended guidelines (50 mg/kg/day and increased as needed by 50 mg/kg/day every 3 days up to a maximum dose of 150 mg/kg/day, divided BID). |
|
Other Vigabatrin |
Vigabatrin open label is given for administration, the entire content of one sachet (500 mg active drug) is dissolved in 10 ml water for oral administration that is dosed according to body weight 50-150 mg/kg/day divided BID. Dosing will follow established recommended guidelines (50 mg/kg/day and increased as needed by 50 mg/kg/day every 3 days up to a maximum dose of 150 mg/kg/day, divided BID). |
|
No Intervention Control Group |
Enrolled subjects who never develop EEG abnormalities or clinical seizures |
|
Recruiting Locations
More Details
- NCT ID
- NCT02849457
- Status
- Completed
- Sponsor
- Martina Bebin
Detailed Description
The central hypothesis of this Phase IIb trial is that early identification of electroencephalography (EEG) biomarkers and early treatment versus delayed treatment with vigabatrin in infants with tuberous sclerosis complex (TSC) will have a positive impact on developmental outcomes at 24 months of age. It would also prevent or lower the risk of developing infantile spasms and refractory seizures. This preventative approach would be expected to result in more favorable long-term cognitive, behavioral, developmental and psychiatric outcomes and significantly improve overall quality of life. It is a randomized, double-blind, placebo-controlled clinical trial design. Successful completion of this trial will also advance the field by demonstrating the value of systematic surveillance with EEG in asymptomatic infants with TSC.