Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex

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.

Inclusion Criteria:

1. 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:

1. 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)