STARS: Sabril for Complex Partial Seizures in Adult Tolerability Study (TS) Patients
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 4 |
Study Design
Arms and Interventions
| 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:
|
Outcome Measures
Primary Outcome Measures
- Number of Participants Safely Tolerating Sabril [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.]
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
Secondary Outcome Measures
- 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)]
Seizure freedom Responder rate (complex partial seizures only)
Eligibility Criteria
Criteria
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
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | University of Pennsylvania | Philadelphia | Pennsylvania | United States | 19103 |
Sponsors and Collaborators
- University of Pennsylvania
- H. Lundbeck A/S
Investigators
- Principal Investigator: John Pollard, MD, University of Pennsylvania
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- 811542
Study Results
Participant Flow
| Recruitment Details | Subjects were recruited for the treatment arm from within the Tuberous Sclerosis (TS) clinical at the University of Pennsylvania. Recruitment begain on 8/19/2010 and continued through 2013. All potential subjects had to be eligible to take Sabril (vigabatrin) under the FDA-approved prescribing information. |
|---|---|
| Pre-assignment Detail | Before taking Sabril, subjects were required to undergo visual field and depression screenings in response to the FDA's black box warning about Sabril causing vision loss, and an increased risk of suicidal ideation among patients taking antiepileptic drugs like Sabril. No potential subjects were excluded due to screening assessments. |
| Arm/Group Title | Treatment With Sabril (Vigabatrin) |
|---|---|
| Arm/Group Description | 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. |
| Period Title: Overall Study | |
| STARTED | 1 |
| COMPLETED | 0 |
| NOT COMPLETED | 1 |
Baseline Characteristics
| Arm/Group Title | Treatment With Sabril (Vigabatrin) |
|---|---|
| Arm/Group Description | 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. |
| Overall Participants | 1 |
| Age (Count of Participants) | |
| <=18 years |
0
0%
|
| Between 18 and 65 years |
1
100%
|
| >=65 years |
0
0%
|
| Age (years) [Mean (Full Range) ] | |
| Mean (Full Range) [years] |
30
|
| Sex: Female, Male (Count of Participants) | |
| Female |
1
100%
|
| Male |
0
0%
|
| Ethnicity (NIH/OMB) (Count of Participants) | |
| Hispanic or Latino |
0
0%
|
| Not Hispanic or Latino |
1
100%
|
| Unknown or Not Reported |
0
0%
|
| Race (NIH/OMB) (Count of Participants) | |
| American Indian or Alaska Native |
0
0%
|
| Asian |
0
0%
|
| Native Hawaiian or Other Pacific Islander |
0
0%
|
| Black or African American |
0
0%
|
| White |
1
100%
|
| More than one race |
0
0%
|
| Unknown or Not Reported |
0
0%
|
| Region of Enrollment (participants) [Number] | |
| United States |
1
100%
|
Outcome Measures
| Title | Number of Participants Safely Tolerating Sabril |
|---|---|
| Description | 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 |
| Time Frame | 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. |
Outcome Measure Data
| Analysis Population Description |
|---|
| 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. |
| Arm/Group Title | Treatment With Sabril (Vigabatrin) |
|---|---|
| Arm/Group Description | 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. |
| Measure Participants | 1 |
| Count of Participants [Participants] |
0
0%
|
| Title | Number of Patients Who Become Seizure Free While Taking Sabril |
|---|---|
| Description | Seizure freedom Responder rate (complex partial seizures only) |
| Time Frame | Seizure freedom will be assessed for the two month treatment phase of the study (months 4 and 5) |
Outcome Measure Data
| Analysis Population Description |
|---|
| 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. |
| Arm/Group Title | Treatment With Sabril (Vigabatrin) |
|---|---|
| Arm/Group Description | 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. |
| Measure Participants | 1 |
| Count of Participants [Participants] |
0
0%
|
Adverse Events
| Time Frame | 6 months | |
|---|---|---|
| Adverse Event Reporting Description | The study team used clinicaltrials.gov definitions for adverse events and serious adverse events. | |
| Arm/Group Title | Treatment With Sabril (Vigabatrin) | |
| Arm/Group Description | 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. | |
All Cause Mortality |
||
| Treatment With Sabril (Vigabatrin) | ||
| Affected / at Risk (%) | # Events | |
| Total | 0/1 (0%) | |
Serious Adverse Events |
||
| Treatment With Sabril (Vigabatrin) | ||
| Affected / at Risk (%) | # Events | |
| Total | 1/1 (100%) | |
| Nervous system disorders | ||
| Seizure Cluster | 1/1 (100%) | 2 |
Other (Not Including Serious) Adverse Events |
||
| Treatment With Sabril (Vigabatrin) | ||
| Affected / at Risk (%) | # Events | |
| Total | 1/1 (100%) | |
| General disorders | ||
| Fatigue | 1/1 (100%) | 1 |
| Nervous system disorders | ||
| seizures | 1/1 (100%) | 1 |
Limitations/Caveats
More Information
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
| Name/Title | John Pollard, MD |
|---|---|
| Organization | University of Pennsylvania |
| Phone | 215-873-5909 |
| john.pollard@uphs.upenn.edu |
- 811542