A Pilot Efficacy and Safety Study of ST101 in Essential Tremor - Full Text View

Purpose

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.

Condition	Intervention	Phase
Essential Tremor	Drug: ST101 Drug: Placebo	Phase 2


Study Type:	Interventional
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

Resource links provided by NLM:

Genetics Home Reference related topics: essential tremor

MedlinePlus related topics: Tremor

U.S. FDA Resources

Further study details as provided by Sonexa Therapeutics, Inc.:

Primary Outcome Measures:

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.

Secondary Outcome Measures:

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.


Enrollment:	25
Study Start Date:	April 2011
Study Completion Date:	December 2011
Primary Completion Date:	December 2011 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: ST101 ST101 oval tablets	Drug: ST101 tablets for oral administration
Placebo Comparator: Placebo oval tablets to match ST101 tablet	Drug: Placebo placebo to match

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.

Eligibility


Ages Eligible for Study:	18 Years and older (Adult, Senior)
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01332695

Locations


United States, California

Los Angeles, California, United States

Sponsors and Collaborators

Sonexa Therapeutics, Inc.

More Information


Responsible Party:	Sonexa Therapeutics, Inc.
ClinicalTrials.gov Identifier:	NCT01332695 History of Changes
Other Study ID Numbers:	ST101-A002-203
Study First Received:	April 7, 2011
Last Updated:	January 4, 2012
Health Authority:	United States: Food and Drug Administration

Keywords provided by Sonexa Therapeutics, Inc.:


tremor WHIGET Fahn Tolosa Marin Scale

Additional relevant MeSH terms:


Tremor Essential Tremor Dyskinesias Neurologic Manifestations	Nervous System Diseases Signs and Symptoms Movement Disorders Central Nervous System Diseases

ClinicalTrials.gov processed this record on September 27, 2016