Symptoms and General Pathology

All Conditions

Nervous System Diseases

Neoplasms

Diseases and Abnormalities at or Before Birth

Skin and Connective Tissue Diseases

Rare Diseases

Sclerosis

Seizures

Tuberous Sclerosis

Neurologic Manifestations

Hamartoma

Neoplasms, Multiple Primary

Syndrome

Neoplastic Syndromes, Hereditary

Congenital Abnormalities

Malformations of Cortical Development

Nervous System Malformations

Neurocutaneous Syndromes

Heredodegenerative Disorders, Nervous System

Neurodegenerative Diseases

Genetic Diseases, Inborn

Tuberous Sclerosis Complex

Anticonvulsants

All Drugs and Chemicals

Vigabatrin

Enzyme Inhibitors

Neurotransmitter Agents

This is a single arm study. All subjects who are eligible for treatment will begin taking vigabatrin (Sabril) during the third month of the study. Treatment will be in accordance with the FDA-approved prescribing information: upward titration will happen at a rate of 500mg per week until subjects reach their maximum tolerated dose, or 3g per day (whichever is lower). This dose may be decreased if needed under the supervision of the study doctor. Subjects who need to lower their dose or who stop taking Sabril will have their dosage decreased at a rate of 1 gm/week for one month under the supervision of the study doctor.

Subjects will begin taking vigabatrin (Sabril) during the third month of the study. Upward titration will happen at a rate of 500mg per week until subjects reach their maximum tolerated dose, or 3g per day (whichever is lower). This dose may be decreased if needed under the supervision of the study doctor. Subjects who need to lower their dose or who stop taking Sabril will have their dosage decreased at a rate of 1 gm/week for one month under the supervision of the study doctor.

vigabatrin

John Pollard, MD

This is an open-label, phase 4 study to examine the safety and efficacy of vigabatrin (Sabril) in Tuberous Sclerosis patients, a subset of the larger refractory complex partial epilepsy population for which the drug is approved. While enrolled on this trial, subjects will continue to take all of their normally prescribed medications, including their other antiepileptic drugs (AEDs).

Alternatively, there is a prospective observational arm that subjects who are about to take Sabril as treatment for seizures associated with Tuberous Sclerosis may join. Subjects who join this arm will not have any study visits and will not be asked to do anything specifically for the study. The study team will collect all study data from subjects' medical records only.

Sabril for Complex Partial Seizures in Adult Tolerability Study (TS) Patients

Sabril for Treatment of Adult Refractory Partial Seizures Symptomatic of Tuberous Sclerosis: An Open Label, Phase IV Prospective Safety and Tolerability Study

* Antiepileptic Drug (AED) levels in blood
* Comprehensive panel (blood test)
* Complete Blood Count with differential (blood test)
* Visual field tests testing
* Ophthalmology exam assessment
* Frequency and severity of adverse events reported by subjects throughout their involvement with the study

* Seizure freedom
* Responder rate (complex partial seizures only)

H. Lundbeck A/S

University of Pennsylvania

The study team used clinicaltrials.gov definitions for adverse events and serious adverse events.

Treatment With Sabril (Vigabatrin)

This is a single arm study. All subjects who are eligible for treatment will begin taking vigabatrin (Sabril) during the third month of the study. Treatment will be in accordance with the FDA-approved prescribing information: upward titration will happen at a rate of 500mg per week until subjects reach their maximum tolerated dose, or 3g per day (whichever is lower). This dose may be decreased if needed under the supervision of the study doctor. Subjects who need to lower their dose or who stop taking Sabril will have their dosage decreased by 1 gm/week for one month under the supervision of the study doctor.

Age, Categorical

Age, Continuous

Treatment

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

United States

Region of Enrollment

The study was terminated due to insufficient enrollment; 98 potentially eligible patients were pre-screened and only one enrolled. The study was not terminated due to concerns regarding safety or efficacy.

One 30 year old female, Caucasian, non-Hispanic subject enrolled in the study. Due to adverse events, she did not complete the study, however, she completed all required follow-up.

Number of Participants Safely Tolerating Sabril

Number of Patients Who Become Seizure Free While Taking Sabril

The interventional arm of this phase 4 study involved subjects taking vigabatrin (Sabril) in accordance with standard of care, FDA approved dosing instructions. There were no planned arms; all subjects followed the FDA-approved prescribing label.

As there were not multiple treatment arms under investigation, per the FDA-approved prescribing label, the one and only subject who enrolled underwent upward titration happened at a rate of 500mg per week until she reached her maximum tolerated dose, or 3g per day. This dose was decreased as needed under the supervision of the study doctor. Again in accordance with standard of care, FDA-approved prescribing guidelines, when she stopped taking Sabril, her dosage decreased at a rate of 1 gm/week for one month under the supervision of the study doctor.

Overall Study

Epilepsy

Brain Diseases

Central Nervous System Diseases

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).

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).

Enrolled subjects who never develop EEG abnormalities or clinical seizures

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.

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.

Placebo

Martina Bebin, MD, MPA

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

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.

Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex

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

De-identified data will be shared with National Database for Autism Research (NDAR)

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.

Evaluate the number of subjects that develop seizures when treated with vigabatrin as a seizure prevention.

Time to the subject's first clinical seizure will be measured for both subjects on placebo and vigabatrin.

The prevalence of drug resistant epilepsy.

Evaluate Vineland II scores and the impact of early versus late treatment with vigabatrin at 12, 24, 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, severe adverse events as assessed by CTCAE v4.0 and risk evaluation and mitigation strategy (REMS) measures as required by the FDA.

Feasibility of the routine 1 hour video EEG in determining the EEG biomarker for developing epilepsy

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Sabril for Complex Partial Seizures in Adult Tolerability Study (TS) Patients	NCT01266291	Complex Partial...	vigabatrin	18 Years -	University of Pennsylvania
Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex	NCT02849457	Tuberous Sclero...	Vigabatrin Placebo	1 Day - 6 Months	University of Alabama at Birmingham
Sabril for Complex Partial Seizures in Adult Tolerability Study (TS) Patients	NCT01266291	Complex Partial...	vigabatrin	18 Years -	University of Pennsylvania

Spots Global Cancer Trial Database for vigabatrin

Every month we try and update this database with for vigabatrin cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Spots Global Cancer Trial Database for vigabatrin

Every month we try and update this database with for vigabatrin cancer trials from around the world to help patients and their families find trials that might be right for them. We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Every month we try and update this database with for vigabatrin cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.