Summary
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
Conditions
Tuberous Sclerosis Complex
Recruitment Status
Completed
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.
Eligibility Criteria
Inclusion Criteria:
less than or equal to 6 months of age
No history of seizures or infantile spasms, or evidence of subclinical electrographic seizures on a previous video EEG
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
Has not been diagnosed with TSC
History of seizures or infantile spasms, or evidence of subclinical electrographic seizures on a previous video EEG
Has received any anticonvulsant medication including vigabatrin, other anti-seizure therapeutic agent including cannabidiol
Has received an oral mTOR inhibitor such as everolimus or sirolimus
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
Is currently enrolled, or plans on enrolling at any time during the duration of the study, in an experimental behavioral early intervention study
Has a history of being born prematurely (born less than <30 weeks gestation at the time of delivery)
Intervention
Intervention Type
Intervention Name
Drug
Vigabatrin
Drug
Placebo
Phase
Phase 2
Gender
All
Min Age
1 Day
Max Age
6 Months
Download Date
May 9, 2023
Principal Investigator
Martina Bebin
Primary Contact Information
For more information on this trial, visit clinicaltrials.gov.
Contact
For more information and to contact the study team: