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Was not possible to enroll sufficient number of subjects to draw any worthwhile conclusions from the study.
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| Name | Class |
|---|---|
| H. Lundbeck A/S | INDUSTRY |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| John Pollard, MD | University of Pennsylvania | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Pennsylvania | Philadelphia | Pennsylvania | 19103 | United States |
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.
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.
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| ID | Title | Description |
|---|---|---|
| FG000 | Treatment With Sabril (Vigabatrin) | 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. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | 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. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants Safely Tolerating Sabril |
| 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. | Posted | Count of Participants | Participants | 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. |
|
6 months
The study team used clinicaltrials.gov definitions for adverse events and serious adverse events.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Seizure Cluster | Nervous system disorders | MedDRA (5.1) | Non-systematic Assessment | Subject was admitted to her local emergency room when she experienced seizure clusters that did not resolve with Diastat. SAE happened on twice. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Fatigue | General disorders | MedDRA (5.1) | Non-systematic Assessment |
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.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| John Pollard, MD | University of Pennsylvania | 215-873-5909 | john.pollard@uphs.upenn.edu |
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| ID | Term |
|---|---|
| D012640 | Seizures |
| D014402 | Tuberous Sclerosis |
| ID | Term |
|---|---|
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
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| ID | Term |
|---|---|
| D020888 | Vigabatrin |
| ID | Term |
|---|---|
| D005680 | gamma-Aminobutyric Acid |
| D000613 | Aminobutyrates |
| D002087 | Butyrates |
| D000144 | Acids, Acyclic |
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|
|
| Participants |
|
| Age, Continuous | Mean | Full Range | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
|
|
| Secondary | Number of Patients Who Become Seizure Free While Taking Sabril |
| 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. | Posted | Count of Participants | Participants | Seizure freedom will be assessed for the two month treatment phase of the study (months 4 and 5) |
|
|
|
| 0 |
| 1 |
| 1 |
| 1 |
| 1 |
| 1 |
|
| seizures | Nervous system disorders | MedDRA (5.1) | Non-systematic Assessment | Increased seizure severity |
|
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| D006222 | Hamartoma |
| D009369 | Neoplasms |
| D009378 | Neoplasms, Multiple Primary |
| D009386 | Neoplastic Syndromes, Hereditary |
| D065703 | Malformations of Cortical Development, Group I |
| D054220 | Malformations of Cortical Development |
| D009421 | Nervous System Malformations |
| D020752 | Neurocutaneous Syndromes |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |
| D002264 |
| Carboxylic Acids |
| D009930 | Organic Chemicals |
| D000596 | Amino Acids |
| D000602 | Amino Acids, Peptides, and Proteins |