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Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex

This study is currently recruiting participants.
Verified May 2017 by Martina Bebin, University of Alabama at Birmingham
Sponsor:
ClinicalTrials.gov Identifier:
NCT02849457
First Posted: July 29, 2016
Last Update Posted: May 30, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborator:
National Institute of Neurological Disorders and Stroke (NINDS)
Information provided by (Responsible Party):
Martina Bebin, University of Alabama at Birmingham
  Purpose
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

Condition Intervention Phase
Tuberous Sclerosis Complex Drug: Vigabatrin Drug: Placebo Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Prevention
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

Resource links provided by NLM:


Further study details as provided by Martina Bebin, University of Alabama at Birmingham:

Primary Outcome Measures:
  • 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.



Secondary Outcome Measures:
  • 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


Estimated Enrollment: 80
Study Start Date: December 2016
Estimated Study Completion Date: May 2021
Estimated Primary Completion Date: May 2020 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Placebo Comparator: Vigabatrin or Placebo
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).
Drug: Vigabatrin
Subjects randomized to vigabatrin in Arm A will be treated with vigabatrin 100mg/kg/day until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on study drug, they will transition into the Open label phase of the study (Arm B) and continue to be followed until 36 months of age.
Other Name: Sabril
Drug: Placebo
Subjects randomized to placebo in Arm A will be treated with matching placebo at 100mg/kg/day until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on study drug, they will transition into the Open label phase of the study (Arm B) and continue to be followed until 36 months of age.
Vigabatrin
Vigabatrin open label 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).
Drug: Vigabatrin
Subjects randomized to vigabatrin in Arm A will be treated with vigabatrin 100mg/kg/day until 24 months of age or until they show evidence of clinical seizures or electrographic seizures on video EEG. If electrographic or clinical seizures occur while on study drug, they will transition into the Open label phase of the study (Arm B) and continue to be followed until 36 months of age.
Other Name: Sabril
No Intervention: Control Group
Enrolled subjects who never develop EEG abnormalities or clinical seizures

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

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 6 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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)
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02849457


Contacts
Contact: Jessica Krefting, RN 205-975-2890 jessicakrefting@uabmc.edu
Contact: Regina Ryan, MS 205-975-2890 reginaryan@uabmc.edu

Locations
United States, Alabama
University of Alabama at Birmingham Recruiting
Birmingham, Alabama, United States, 35233
Contact: Jessica Krefting, RN    205-975-2890    jessicakrefting@uabmc.edu   
Contact: Regina Ryan, MS    205-975-2890    reginaryan@uabmc.edu   
Principal Investigator: E. Martina Bebin, MD, MPA         
United States, California
University of California, Los Angeles Recruiting
Los Angeles, California, United States, 90095
Contact: Natalie Solis    310-206-4037    nsolis@mednet.ucla.edu   
Principal Investigator: Joyce Wu, MD         
Stanford University Recruiting
Palo Alto, California, United States, 94304
Contact: Swetapadma Patnaik    650-721-1458    sweta@stanford.edu   
Principal Investigator: Brenda Porter, MD         
United States, Massachusetts
Boston Children's Hospital Recruiting
Boston, Massachusetts, United States, 02215
Contact: Amelia Diplock    617-919-1476    amelia.diplock@childrens.harvard.edu   
Principal Investigator: Mustafa Sahin, MD, PhD         
United States, Minnesota
Minnesota Epilepsy Group, PA Recruiting
Saint Paul, Minnesota, United States, 55102
Contact: Alisha Olson, RN    651-241-5206    aolson@mnepilepsy.net   
Principal Investigator: Michael Frost, MD         
United States, Ohio
Cincinnati's Children Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Molly Griffith    513-636-9699    molly.griffith@cchmc.org   
Principal Investigator: Darcy Kreuger, MD, PhD         
United States, Texas
University of Texas Health Science Center at Houston Recruiting
Houston, Texas, United States, 77054
Contact: Elida Salazar    713-500-5766    Elida.L.Salazar@uth.tmc.edu   
Principal Investigator: Mary K Koenig, MD         
Sponsors and Collaborators
Martina Bebin
National Institute of Neurological Disorders and Stroke (NINDS)
Investigators
Principal Investigator: Martina Bebin, MD, MPA University of Alabama at Birmingham
  More Information

Responsible Party: Martina Bebin, Professor of Neurology and Pediatrics, University of Alabama at Birmingham
ClinicalTrials.gov Identifier: NCT02849457     History of Changes
Other Study ID Numbers: PREVeNT
1U01NS092595-01A1 ( U.S. NIH Grant/Contract )
First Submitted: July 13, 2016
First Posted: July 29, 2016
Last Update Posted: May 30, 2017
Last Verified: May 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: De-identified data will be shared with National Database for Autism Research (NDAR)

Additional relevant MeSH terms:
Sclerosis
Epilepsy
Tuberous Sclerosis
Pathologic Processes
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Hamartoma
Neoplasms
Neoplasms, Multiple Primary
Neoplastic Syndromes, Hereditary
Malformations of Cortical Development, Group I
Malformations of Cortical Development
Nervous System Malformations
Neurocutaneous Syndromes
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Congenital Abnormalities
Genetic Diseases, Inborn
Vigabatrin
Anticonvulsants
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
GABA Agents
Neurotransmitter Agents
Physiological Effects of Drugs


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