A Pilot Efficacy and Safety Study of ST101 in Essential Tremor
This study will look at the ability of ST101 to treat symptoms in subjects with Essential Tremor. This study will also examine the safety and tolerability of the drug. This study is evaluating one dose level of ST101 versus placebo in a cross-over fashion. This means that all patients will receive both ST101 and placebo, but they will receive it in a random (by chance) order.
|Study Design:||Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
|Official Title:||A Randomized, Double-Blind, Placebo-Controlled, Cross-Over, Safety, Tolerability and Proof of Concept Study of ST101 for Essential Tremor|
- Washington Heights-Inwood Genetic Study of Essential Tremor (WHIGET) Rating Scale [ Time Frame: Baseline, Week 3, Week 5, Week 8 ] [ Designated as safety issue: No ]The WHIGET Rating Scale is a 6-item scale. Ratings are performed for the right and left side for each activity. Each subject is videotaped in the same manner performing set activities, which are then rated. Rated tasks involve fine motor skills. Each item is scored by an independent, central, blinded reader, and the item scores are summed to provide a total score. Total score range is from 0 to 48, with an increase in score reflecting an increase in ET severity.
- Functional Disabilities Scale [ Time Frame: Screening, Baseline, Weeks 3, 5, 8 ] [ Designated as safety issue: No ]An 8-Item functional scale that assesses the following functional domains: Speaking/Phonation, Feeding (other than liquids), Bringing Liquids to Mouth, Hygiene, Dressing, Writing, Working, and Social Activities. Higher scores indicate worsening of disease.
- Clinical Global Impression of Change [ Time Frame: Week 3, Week 8 ] [ Designated as safety issue: No ]A 7-point Likert scale with Marked Worsening to Marked Improvement at the extremes, and Unchanged in the center. Scores range from -3 to +3 wherein negative scores indicate worsening, while positive scores indicate improvement.
- Subject Clinical Global Impression of Change [ Time Frame: Week 3, Week 8 ] [ Designated as safety issue: No ]A global visual analog scale (VAS) which is a 100 mm horizontal line on which the subject indicates their overall assessment of response to study drug by making a mark on the horizontal line.
|Study Start Date:||April 2011|
|Study Completion Date:||December 2011|
|Primary Completion Date:||December 2011 (Final data collection date for primary outcome measure)|
ST101 oval tablets
tablets for oral administration
Placebo Comparator: Placebo
oval tablets to match ST101 tablet
placebo to match
Essential tremor (ET) is a slowly progressive, chronic neurological disorder characterized by a 4- to 12-Hertz tremor, which can involve the arms, head, voice and lower extremities. Although the hallmark of the disorder is thought to be the kinetic tremor, ET patients usually also manifest a postural tremor as well. Kinetic tremor (tremor with voluntary movements) may interfere with fine motor skills (e.g., writing, eating, drinking from a cup, pouring liquids); and depending upon its severity, may result in significant disability and diminished quality of daily living. Essential tremor represents an area of unmet medical need and this study is designed as a safety, tolerability and proof-of-concept investigation of the ability of ST101 to improve tremor. In this 2-stage study, subjects will be randomly assigned to either ST101 or placebo in Stage 1, and will receive the alternate treatment in Stage 2.