Back to resultsA Pilot Efficacy and Safety Study of ST101 in Essential Tremor
Primary Purpose
Essential Tremor
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
ST101
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Essential Tremor focused on measuring tremor, WHIGET, Fahn Tolosa Marin Scale
Eligibility Criteria
Inclusion Criteria:
- Diagnosis of definite or probable essential tremor (ET) as defined by the Tremor Investigational Group with involvement of the hands and arms of at least 2 years duration
- Tremor severity score of >/= 2 in at least one upper extremity on the Fahn-Tolosa-Marin Tremor Rating Scale
- Subject who is accessible by telephone
- Subject who can comply with study visits, study drug compliance, and study procedures.
Exclusion Criteria:
- Subject whose tremor is adequately controlled on a current treatment
- Subject who is pregnant, plans to become pregnant during the study or within 2 months of completing the study or who is presently lactating or breast-feeding
- Subject with other medical conditions that may cause or explain subject's tremor
- Subject with a recent history of hematologic/oncologic disorders
- Subject taking medication(s) that might produce tremor or interfere with the evaluation of tremor
- Subject who has had surgical intervention for their tremor in the past (e.g., ablative thalamotomy or gamma knife thalamotomy), or has had a botulinum toxin injection in the 6 months prior to screening
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
ST101
Placebo
Arm Description
ST101 oval tablets
oval tablets to match ST101 tablet
Outcomes
Primary Outcome Measures
Washington Heights-Inwood Genetic Study of Essential Tremor (WHIGET) Rating Scale
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.
Secondary Outcome Measures
Functional Disabilities Scale
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
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
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.
Full Information
NCT ID
NCT01332695
First Posted
April 7, 2011
Last Updated
January 4, 2012
Sponsor
Sonexa Therapeutics, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT01332695
Brief Title
A Pilot Efficacy and Safety Study of ST101 in Essential Tremor
Official Title
A Randomized, Double-Blind, Placebo-Controlled, Cross-Over, Safety, Tolerability and Proof of Concept Study of ST101 for Essential Tremor
Study Type
Interventional
2. Study Status
Record Verification Date
January 2012
Overall Recruitment Status
Completed
Study Start Date
April 2011 (undefined)
Primary Completion Date
December 2011 (Actual)
Study Completion Date
December 2011 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Sonexa Therapeutics, Inc.
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
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.
Detailed Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Essential Tremor
Keywords
tremor, WHIGET, Fahn Tolosa Marin Scale
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Crossover Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
25 (Actual)
8. Arms, Groups, and Interventions
Arm Title
ST101
Arm Type
Experimental
Arm Description
ST101 oval tablets
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
oval tablets to match ST101 tablet
Intervention Type
Drug
Intervention Name(s)
ST101
Intervention Description
tablets for oral administration
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
placebo to match
Primary Outcome Measure Information:
Title
Washington Heights-Inwood Genetic Study of Essential Tremor (WHIGET) Rating Scale
Description
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.
Time Frame
Baseline, Week 3, Week 5, Week 8
Secondary Outcome Measure Information:
Title
Functional Disabilities Scale
Description
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.
Time Frame
Screening, Baseline, Weeks 3, 5, 8
Title
Clinical Global Impression of Change
Description
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.
Time Frame
Week 3, Week 8
Title
Subject Clinical Global Impression of Change
Description
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.
Time Frame
Week 3, Week 8
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Diagnosis of definite or probable essential tremor (ET) as defined by the Tremor Investigational Group with involvement of the hands and arms of at least 2 years duration
Tremor severity score of >/= 2 in at least one upper extremity on the Fahn-Tolosa-Marin Tremor Rating Scale
Subject who is accessible by telephone
Subject who can comply with study visits, study drug compliance, and study procedures.
Exclusion Criteria:
Subject whose tremor is adequately controlled on a current treatment
Subject who is pregnant, plans to become pregnant during the study or within 2 months of completing the study or who is presently lactating or breast-feeding
Subject with other medical conditions that may cause or explain subject's tremor
Subject with a recent history of hematologic/oncologic disorders
Subject taking medication(s) that might produce tremor or interfere with the evaluation of tremor
Subject who has had surgical intervention for their tremor in the past (e.g., ablative thalamotomy or gamma knife thalamotomy), or has had a botulinum toxin injection in the 6 months prior to screening
Facility Information:
City
Los Angeles
State/Province
California
Country
United States
12. IPD Sharing Statement
Learn more about this trial
A Pilot Efficacy and Safety Study of ST101 in Essential Tremor
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