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

NCT01266291 | Terminated Phase 4 Results DMC

• 1 other identifier: 811542
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Complex Partial Seizures

Keywords

Tuberous sclerosis; TS; Seizures

Outcome Measures

Primary Outcomes (1)

• Number of Participants Safely Tolerating Sabril

  * 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

  Outcome measures will be assessed at the initiation of Sabril (titration), and at three and five months after starting Sabril. After this time, the subjects will have completed the study.

Secondary Outcomes (1)

• Number of Patients Who Become Seizure Free While Taking Sabril

  Seizure freedom will be assessed for the two month treatment phase of the study (months 4 and 5)

Study Arms (1)

Treatment with Sabril (vigabatrin)

OTHER

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.

Drug: vigabatrin

Interventions

vigabatrin DRUG

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.

Also known as: Sabril

Treatment with Sabril (vigabatrin)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• The patient or patient's legally authorized representative must sign and date the Institutional Review Board approved Informed Consent and HIPPA Authorization Form.
• Male and female patients 18+ years of age with a clinical diagnosis of Tuberous Sclerosis who experience an average of at least three partial seizures every two months, of which one must be a complex partial seizure
• Patient must be on at least one and a maximum of four AEDs. Patient must be on a stable AED dose regimen for at least 30 days prior to screening. Neither a Vagal Nerve Stimulator (VNS) nor the ketogenic diet will count as an AED
• In the investigator's opinion, the patient or caregiver must be able to keep a seizure diary
• An MRI in the last 5 years, or willingness to undergo an MRI as part of the screening process

You may not qualify if:

• Cause of patient's seizures is a neurologic disease that is not Tuberous Sclerosis
• Current, clinical diagnosis of a major depressive episode or suicidal ideation
• Patient is taking more than four concurrent AEDs. Note: VNS or ketogenic diet is allowed and will not be counted in the four allowed AEDs
• Patient has a progressive CNS lesion confirmed by magnetic resonance imaging (MRI) or computed tomography (CT) scan
• Patient is currently abusing drugs or alcohol

Sponsors & Collaborators

• University of Pennsylvania: lead
• H. Lundbeck A/S: collaborator

Study Sites (1)

University of Pennsylvania

Philadelphia, Pennsylvania, 19103, United States

Location

MeSH Terms

Conditions

Seizures; Tuberous Sclerosis

Interventions

Vigabatrin

Condition Hierarchy (Ancestors)

Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; 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; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Intervention Hierarchy (Ancestors)

gamma-Aminobutyric Acid; Aminobutyrates; Butyrates; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Amino Acids; Amino Acids, Peptides, and Proteins

Limitations and Caveats

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.

Results Point of Contact

• Title: John Pollard, MD
• Organization: University of Pennsylvania

john.pollard@uphs.upenn.edu

215-873-5909

Study Officials

• John Pollard, MD

  University of Pennsylvania

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 22, 2010
• First Posted: December 24, 2010
• Study Start: August 1, 2010
• Primary Completion: February 1, 2013
• Study Completion: December 1, 2013
• Last Updated: July 12, 2017
• Results First Posted: July 12, 2017

Record last verified: 2017-06

Locations

US (1)