A Study to Investigate the Efficacy and Safety of NT 201 (Botulinum Toxin) Compared With Placebo for the Treatment of Adult Participants With Essential Tremor in the Arm

Merz Pharmaceuticals GmbH78 enrolled

Overview

The purpose of this study is to determine whether a single treatment with administration of NT 201 (botulinum toxin) is superior to placebo (no medicine) for one-sided treatment of essential tremor in the arm (Unilateral Period). Participants will be assigned to the treatment groups by chance and neither the participants nor the research staff who interact with them will know the allocation. The following treatment cycle will investigate the safety and tolerability of two-sided treatment with NT 201 (botulinum toxin) (Open Label Bliaterial Period). All participants will receive NT 201 treatment.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

Conditions

Essential Tremor of the Upper Limb

Interventions

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: \- Score of ≥ 2 (at least 1 cm tremor amplitude) in at least two out of three maneuvers of test item 4 (upper limb tremor) confirmed by an independent TETRAS expert by means of video assessment. Exclusion Criteria: * History or presence of day-to-day fluctuations in ET which would jeopardize meaningful tremor assessment over time, e.g. severe tremor on one day and minimal or no tremor on another day. * Other neurological signs, such as dystonia, ataxia, or parkinsonism, which in the judgment of the investigator could interfere with the ET diagnosis and/or assessment of ET in ULs. * Tremor types other than ET

Locations (14)

USF, Department of Neurology, Merz Investigational Site #0010020

Tampa, Florida, United States

University of Nebraska Medical Center, Merz Investigational Site #0010269

Omaha, Nebraska, United States

Cleveland Clinic Lou Ruvo Center for Brain Health, Merz Investigational Site #0010457

Las Vegas, Nevada, United States

Mount Sinai Medical Center, Merz Investigational Site #0010191

New York, New York, United States

Vanderbilt University Medical Center, Neuroscience Institute, Merz Investigational Site #0010206

Nashville, Tennessee, United States

Houston Methodist Neurological Institute, Merz Investigational Site #0010226

Houston, Texas, United States

Medstar Georgetown Neurology, Merz Investigational Site #0010231

McLean, Virginia, United States

UW Medical Center - Montlake, Merz Investigational Site #0010450

Seattle, Washington, United States

Selkirk Neurology, Merz Investigational Site #0010456

Spokane, Washington, United States

London Health Sciences Centre, Merz Investigational Site #0010087

London, Ontario, Canada

...and 4 more locations

Outcomes

Primary Outcomes

Change from study baseline to Week 6 in maximum tremor amplitude measurement at wrist level during the unilateral treatment period

Time frame: From baseline to week 6

Secondary Outcomes

Change from study baseline to Week 6 in the Essential Tremor Rating Assessment Scale [TETRAS] Performance dominant upper limb [UL] score

Time frame: From baseline to week 6

Change from study baseline to Week 6 in TETRAS Activities of Daily Living [ADL] UL score

Time frame: From baseline to week 6

Change from study baseline to Week 6 in TETRAS ADL Functional Impact score

Time frame: From baseline to week 6

Subject´s Global Impression of Change Scale [GICS] score of motor dominant UL at Week 6

Time frame: Week 6

Investigator´s Global Impression of Change Scale [GICS] score of motor dominant UL at Week 6

Time frame: Week 6

Change from Cycle 2 baseline to Week 6 in TETRAS Performance dominant UL score

Time frame: Week 24 to week 36

Change from Cycle 2 baseline to Week 6 in TETRAS Performance subscale score

Time frame: Week 24 to week 36

Change from Cycle 2 baseline to Week 6 in TETRAS ADL UL score

Time frame: Week 24 to week 36

Change from Cycle 2 baseline to Week 6 in TETRAS ADL Functional Impact score

Time frame: Week 24 to week 36

Incidence of treatment-emergent AEs [TEAEs] related to treatment

Time frame: From baseline to week 36