Clinical Trial: Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex (PREVeNT Trial) A Randomized, Double-blind, Placebo-controlled Seizure Prevention Clinical

Brief 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

Detailed Summary: 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.
Sponsor: Martina Bebin

Current Primary Outcome: Cognitive Assessment Scores and Developmental Impact [ Time Frame: 24 months ]

The primary outcome measure will be the cognitive assessment scores on the Bayley Scales of Infant and Toddler Development at 24 months.

The Bayley Scales of Infant and Toddler Development at 24 months will be used for the data analysis and compare the developmental impact of early versus delayed treatment with vigabatrin.



Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Number of subjects that develop seizures when treated with vigabatrin [ Time Frame: 24 months ]
    Evaluate the number of subjects that develop seizures when treated with vigabatrin as a seizure prevention.
  • Time to the Subject's First Clinical Seizure [ Time Frame: 24 months ]
    Time to the subject's first clinical seizure will be measured for both subjects on placebo and vigabatrin.
  • Prevalence of Drug Resistant Epilepsy [ Time Frame: 24 months ]
    The prevalence of drug resistant epilepsy.
  • Evaluate Vineland II Scores and Impact of Early Versus Late Treatment [ Time Frame: 12 months, 24 months and 36 months ]
    Evaluate Vineland II scores and the impact of early versus late treatment with vigabatrin at 12, 24, and 36 months.
  • Evaluate Autism Diagnostic Observation Schedule 2nd Edition (ADOS2) Scores and Impact of Early Versus Late Treatment [ Time Frame: 24 months and 36 months ]
    Evaluate ADOS2 scores and the impact of early versus late treatment at 24 and 36 months.
  • Number of Subjects with Vigabatrin Related Adverse Events and Severe Adverse Events [ Time Frame: 24 months ]
    Number of subjects with vigabatrin related adverse events, severe adverse events as assessed by CTCAE v4.0 and risk evaluation and mitigation strategy (REMS) measures as required by the FDA.
  • EEG Biomarker for Developing Epilepsy [ Time Frame: 24 months ]
    Feasibility of the routine 1 hour video EEG in determining the EEG biomarker for developing epilepsy


Original Secondary Outcome: Same as current

Information By: University of Alabama at Birmingham

Dates:
Date Received: July 13, 2016
Date Started: December 2016
Date Completion: May 2021
Last Updated: March 14, 2017
Last Verified: March 2017