Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex

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Brief Title

Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex

Official Title

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

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 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.
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Cognitive Assessment Scores and Developmental Impact

Secondary Outcome

 Number of subjects that develop seizures when treated with vigabatrin

Condition

Tuberous Sclerosis Complex

Intervention

Vigabatrin

Study Arms / Comparison Groups

 Vigabatrin or Placebo
Description:  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).

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

84

Start Date

December 2016

Completion Date

December 2022

Primary Completion Date

May 2022

Eligibility Criteria

        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)
      

Gender

All

Ages

N/A - 6 Months

Accepts Healthy Volunteers

No

Contacts

Martina Bebin, MD, MPA, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT02849457

Organization ID

PREVeNT

Secondary IDs

1U01NS092595-01A1

Responsible Party

Sponsor-Investigator

Study Sponsor

Martina Bebin

Collaborators

 National Institute of Neurological Disorders and Stroke (NINDS)

Study Sponsor

Martina Bebin, MD, MPA, Principal Investigator, University of Alabama at Birmingham


Verification Date

May 2021