- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02849457
Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex
May 6, 2023 updated by: Martina Bebin
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
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
Study Overview
Status
Completed
Conditions
Intervention / Treatment
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 Type
Interventional
Enrollment (Actual)
84
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Alabama
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Birmingham, Alabama, United States, 35233
- University of Alabama at Birmingham
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California
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Los Angeles, California, United States, 90095
- University of California, Los Angeles
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Palo Alto, California, United States, 94304
- Stanford University
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District of Columbia
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Washington, District of Columbia, United States, 20010
- Children's National Medical Center
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Massachusetts
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Boston, Massachusetts, United States, 02215
- Boston Children's Hospital
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Michigan
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Royal Oak, Michigan, United States, 48073
- Beaumont Children's Hospital
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Minnesota
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Saint Paul, Minnesota, United States, 55102
- Minnesota Epilepsy Group, PA
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Missouri
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Saint Louis, Missouri, United States, 63110
- Washington University in St. Louis
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North Carolina
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Durham, North Carolina, United States, 37710
- Duke University
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Ohio
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Cincinnati, Ohio, United States, 45229
- Cincinnati's Children Hospital Medical Center
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Pennsylvania
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Philadelphia, Pennsylvania, United States, 19104
- The Children's Hospital of Philadelphia
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Texas
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Houston, Texas, United States, 77054
- University of Texas Health Science Center at Houston
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Washington
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Seattle, Washington, United States, 98105
- Seattle Children's Hospital
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
1 day to 6 months (Child)
Accepts Healthy Volunteers
No
Description
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)
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Prevention
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Triple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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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).
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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 Names:
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.
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Other: 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).
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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 Names:
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No Intervention: Control Group
Enrolled subjects who never develop EEG abnormalities or clinical seizures
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Cognitive Assessment Scores and Developmental Impact
Time Frame: 24 months
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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. |
24 months
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Number of subjects that develop seizures when treated with vigabatrin
Time Frame: 24 months
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Evaluate the number of subjects that develop seizures when treated with vigabatrin as a seizure prevention.
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24 months
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Time to the Subject's First Clinical Seizure
Time Frame: 24 months
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Time to the subject's first clinical seizure will be measured for both subjects on placebo and vigabatrin.
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24 months
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Prevalence of Drug Resistant Epilepsy
Time Frame: 24 months
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The prevalence of drug resistant epilepsy.
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24 months
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Evaluate Vineland II Scores and Impact of Early Versus Late Treatment
Time Frame: 12 months, 24 months and 36 months
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Evaluate Vineland II scores and the impact of early versus late treatment with vigabatrin at 12, 24, and 36 months.
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12 months, 24 months and 36 months
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Evaluate Autism Diagnostic Observation Schedule 2nd Edition (ADOS2) Scores and Impact of Early Versus Late Treatment
Time Frame: 24 months and 36 months
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Evaluate ADOS2 scores and the impact of early versus late treatment at 24 and 36 months.
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24 months and 36 months
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Number of Subjects with Vigabatrin Related Adverse Events and Severe Adverse Events
Time Frame: 24 months
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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.
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24 months
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EEG Biomarker for Developing Epilepsy
Time Frame: 24 months
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Feasibility of the routine 1 hour video EEG in determining the EEG biomarker for developing epilepsy
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24 months
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: Martina Bebin, MD, MPA, University of Alabama at Birmingham
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
December 1, 2016
Primary Completion (Actual)
April 26, 2023
Study Completion (Actual)
May 5, 2023
Study Registration Dates
First Submitted
July 13, 2016
First Submitted That Met QC Criteria
July 28, 2016
First Posted (Estimate)
July 29, 2016
Study Record Updates
Last Update Posted (Actual)
May 9, 2023
Last Update Submitted That Met QC Criteria
May 6, 2023
Last Verified
May 1, 2023
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Neoplasms
- Congenital Abnormalities
- Genetic Diseases, Inborn
- Neurodegenerative Diseases
- Heredodegenerative Disorders, Nervous System
- Neoplastic Syndromes, Hereditary
- Malformations of Cortical Development, Group I
- Malformations of Cortical Development
- Nervous System Malformations
- Neurocutaneous Syndromes
- Hamartoma
- Neoplasms, Multiple Primary
- Epilepsy
- Sclerosis
- Tuberous Sclerosis
- Physiological Effects of Drugs
- Neurotransmitter Agents
- Molecular Mechanisms of Pharmacological Action
- Enzyme Inhibitors
- GABA Agents
- Anticonvulsants
- Vigabatrin
Other Study ID Numbers
- PREVENT (Janssen Biotech Inc.)
- 1U01NS092595-01A1 (U.S. NIH Grant/Contract)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
YES
IPD Plan Description
De-identified data will be shared with National Database for Autism Research (NDAR)
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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