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

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

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.

Study Overview

• Status: Terminated
• Conditions: Complex Partial Seizures
• Intervention / Treatment: Drug: vigabatrin

• Study Type: Interventional
• Enrollment (Actual): 1
• Phase: Phase 4

Contacts and Locations

Study Locations

• United States
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19103
      • University of Pennsylvania

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Other: 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 at a rate of 1 gm/week for one month under the supervision of the study doctor.

Drug: vigabatrin; 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.; Other Names: Sabril

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Patients Who Become Seizure Free While Taking Sabril	Seizure freedom; Responder rate (complex partial seizures only)	Seizure freedom will be assessed for the two month treatment phase of the study (months 4 and 5)

Collaborators and Investigators

• Sponsor: University of Pennsylvania
• Collaborators: H. Lundbeck A/S
• Investigators: Principal Investigator: John Pollard, MD, University of Pennsylvania

Study record dates

Study Major Dates

• Study Start: August 1, 2010
• Primary Completion (Actual): February 1, 2013
• Study Completion (Actual): December 1, 2013

Study Registration Dates

• First Submitted: December 22, 2010
• First Submitted That Met QC Criteria: December 23, 2010
• First Posted (Estimate): December 24, 2010

Study Record Updates

• Last Update Posted (Actual): July 12, 2017
• Last Update Submitted That Met QC Criteria: June 12, 2017
• Last Verified: June 1, 2017

More Information

Terms related to this study

• Keywords: Seizures; Tuberous sclerosis; TS
• Additional Relevant MeSH Terms: Nervous System Diseases; Neoplasms; Neurologic Manifestations; 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; Seizures; Tuberous Sclerosis; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; GABA Agents; Anticonvulsants; Vigabatrin
• Other Study ID Numbers: 811542

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No