Current Environment:

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

Active, not recruiting

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 6, 2022

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: