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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

Phase 2
Completed
Conditions
Essential Tremor of the Upper Limb
Interventions
Drug: NT 201
Drug: Placebo
Registration Number
NCT04766723
Lead Sponsor
Merz Pharmaceuticals GmbH
Brief Summary

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.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
78
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.
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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
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Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
PlaceboNT 201Unilateral Treatment Period (1 treatment cycle): subjects to receive unilateral intramuscular placebo injection into muscles of the upper limb. Open Label Bilateral Treatment Period (1 treatment cycle): subjects to receive bilateral intramuscular injection of NT 201 (130-165 units per arm) into muscles of the upper limbs.
NT 201 (IncobotulinumtoxinA, Xeomin)NT 201Unilateral Treatment Period (1 treatment cycle): subjects to receive unilateral intramuscular injection of NT 201 (130-165 units) into muscles of the upper limb. Open Label Bilateral Treatment Period (1 treatment cycle): subjects to receive bilateral intramuscular injection of NT 201 (130-165 units) into muscles of the upper limbs.
PlaceboPlaceboUnilateral Treatment Period (1 treatment cycle): subjects to receive unilateral intramuscular placebo injection into muscles of the upper limb. Open Label Bilateral Treatment Period (1 treatment cycle): subjects to receive bilateral intramuscular injection of NT 201 (130-165 units per arm) into muscles of the upper limbs.
Primary Outcome Measures
NameTimeMethod
Change from study baseline to Week 6 in maximum tremor amplitude measurement at wrist level during the unilateral treatment periodFrom baseline to week 6
Secondary Outcome Measures
NameTimeMethod
Change from study baseline to Week 6 in TETRAS ADL Functional Impact scoreFrom baseline to week 6
Investigator´s Global Impression of Change Scale [GICS] score of motor dominant UL at Week 6Week 6
Incidence of treatment-emergent AEs [TEAEs] related to treatmentFrom baseline to week 36
Change from study baseline to Week 6 in TETRAS Activities of Daily Living [ADL] UL scoreFrom baseline to week 6
Change from Cycle 2 baseline to Week 6 in TETRAS Performance subscale scoreWeek 24 to week 36
Change from Cycle 2 baseline to Week 6 in TETRAS Performance dominant UL scoreWeek 24 to week 36
Change from study baseline to Week 6 in the Essential Tremor Rating Assessment Scale [TETRAS] Performance dominant upper limb [UL] scoreFrom baseline to week 6
Change from Cycle 2 baseline to Week 6 in TETRAS ADL UL scoreWeek 24 to week 36
Change from Cycle 2 baseline to Week 6 in TETRAS ADL Functional Impact scoreWeek 24 to week 36
Subject´s Global Impression of Change Scale [GICS] score of motor dominant UL at Week 6Week 6

Trial Locations

Locations (14)

Mount Sinai Medical Center, Merz Investigational Site #0010191

🇺🇸

New York, New York, United States

Medstar Georgetown Neurology, Merz Investigational Site #0010231

🇺🇸

McLean, Virginia, United States

London Health Sciences Centre, Merz Investigational Site #0010087

🇨🇦

London, Ontario, Canada

Selkirk Neurology, Merz Investigational Site #0010456

🇺🇸

Spokane, Washington, United States

Specjalistyczne Gabinety, Merz Investigational Site #0480059

🇵🇱

Krakow, Poland

Houston Methodist Neurological Institute, Merz Investigational Site #0010226

🇺🇸

Houston, Texas, United States

UW Medical Center - Montlake, Merz Investigational Site #0010450

🇺🇸

Seattle, Washington, United States

Mazowiecki Szpital Bródnowski, Merz Investigational Site #0480064

🇵🇱

Warsaw, Poland

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

🇺🇸

Las Vegas, Nevada, United States

University of Nebraska Medical Center, Merz Investigational Site #0010269

🇺🇸

Omaha, Nebraska, United States

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

🇺🇸

Nashville, Tennessee, United States

Instytut Zdrowia Dr. Boczarska-Jedynak

🇵🇱

Oświęcim, Poland

USF, Department of Neurology, Merz Investigational Site #0010020

🇺🇸

Tampa, Florida, United States

NeuroKlinika Gabinet Lekarski, Merz Investigational Site #0480101

🇵🇱

Lodz, Poland

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