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

NCT04766723 | Completed Phase 2 Results FDA Drug

• 2 other identifiers: M6020110692021-001988-24
• Study Type: interventional
• Target: 78
• Locations: 3 countries
• Sites: 14

Brief Summary

The purpose of this study is to determine whether a single treatment with NT 201 (botulinum toxin) is superior to placebo (no medicine) for one-sided treatment of essential tremor in the motor-dominant 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 Bilateral Period). All participants will receive NT 201 treatment.

Conditions

Essential Tremor of the Upper Limb

Outcome Measures

Primary Outcomes (1)

• Unilateral Treatment Period: Change From Baseline to Week 6 in Maximum Tremor Amplitude of the Wrist

  TremorTek kinematic analytic investigational device is combination of computer software and wearable movement sensors that allows to collect motion data when placed on individual's arm to quantify tremor in various muscle groups that impact shoulder, elbow, wrist. This assessment system was used to measure maximum angular tremor amplitude at wrist of injected limb (unit: degrees). Angular tremor amplitude was measure of tremor severity. Reduction of maximum angular tremor amplitude at wrist of injected limb represented tremor improvement.

  Baseline up to Week 6

Secondary Outcomes (10)

• Unilateral Treatment Period: Change From Baseline to Week 6 in the Essential Tremor Rating Assessment Scale (TETRAS) Performance Dominant Upper Limb (UL) Score as Assessed by Investigator

  Baseline up to Week 6

• Unilateral Treatment Period: Change From Baseline to Week 6 in the TETRAS Activities of Daily Living (ADL) UL Score

  Baseline up to Week 6

• Unilateral Treatment Period: Change From Baseline to Week 6 in TETRAS ADL Functional Impact Score

  Baseline up to Week 6

• Unilateral Treatment Period: Subject's Global Impression of Change Scale (GICS) Score of Motor-dominant UL at Week 6

  Week 6

• Unilateral Treatment Period: Investigator's GICS Score of Motor-dominant UL at Week 6

  Week 6

Study Arms (2)

NT 201 (IncobotulinumtoxinA, Xeomin)

EXPERIMENTAL

Unilateral Treatment Period (1 treatment cycle): subjects to receive unilateral intramuscular injection of NT 201 (130-165 units) into muscles of the motor-dominant 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 both upper limbs.

Drug: NT 201

Placebo

PLACEBO COMPARATOR

Unilateral Treatment Period (1 treatment cycle): subjects to receive unilateral intramuscular placebo injection into muscles of the motor-dominant 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 both upper limbs.

Drug: NT 201; Drug: Placebo

Interventions

NT 201 DRUG

Active ingredient: Clostridium Botulinum neurotoxin Type A free from complexing proteins. Solution for injection prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl).

Also known as: Incobotulinumtoxin A, IncobotulinumtoxinA, Xeomin, Botulinum toxin type A (150 kiloDalton), free from complexing proteins

NT 201 (IncobotulinumtoxinA, Xeomin); Placebo

Placebo DRUG

Solution for injection prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl).

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• \- Score of ≥ 2 (at least 1 cm tremor amplitude) in at least two out of three maneuvers of test item 4 (upper limb tremor) of the TETRAS performances scale confirmed by an independent TETRAS expert by means of video assessment.

You may not qualify if:

• 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

Sponsors & Collaborators

• Merz Pharmaceuticals GmbH: lead

Study Sites (14)

USF, Department of Neurology, Merz Investigational Site #0010020

Tampa, Florida, 33612, United States

Location

University of Nebraska Medical Center, Merz Investigational Site #0010269

Omaha, Nebraska, 68131, United States

Location

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

Las Vegas, Nevada, 89106, United States

Location

Mount Sinai Medical Center, Merz Investigational Site #0010191

New York, New York, 10029, United States

Location

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

Nashville, Tennessee, 37232, United States

Location

Houston Methodist Neurological Institute, Merz Investigational Site #0010226

Houston, Texas, 77030, United States

Location

Medstar Georgetown Neurology, Merz Investigational Site #0010231

McLean, Virginia, 22101, United States

Location

UW Medical Center - Montlake, Merz Investigational Site #0010450

Seattle, Washington, 98195, United States

Location

Selkirk Neurology, Merz Investigational Site #0010456

Spokane, Washington, 99202, United States

Location

London Health Sciences Centre, Merz Investigational Site #0010087

London, Ontario, N6A 5A5, Canada

Location

Specjalistyczne Gabinety, Merz Investigational Site #0480059

Krakow, 30-539, Poland

Location

NeuroKlinika Gabinet Lekarski, Merz Investigational Site #0480101

Lodz, 90-640, Poland

Location

Instytut Zdrowia Dr. Boczarska-Jedynak

Oświęcim, 32-600, Poland

Location

Mazowiecki Szpital Bródnowski, Merz Investigational Site #0480064

Warsaw, 03-242, Poland

Location

MeSH Terms

Interventions

incobotulinumtoxinA; Botulinum Toxins, Type A

Intervention Hierarchy (Ancestors)

Botulinum Toxins; Metalloendopeptidases; Endopeptidases; Peptide Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes; Metalloproteases; Bacterial Proteins; Proteins; Amino Acids, Peptides, and Proteins; Bacterial Toxins; Toxins, Biological; Biological Factors

Results Point of Contact

• Title: Public Disclosure Manager
• Organization: Merz Therapeutics GmbH

clinicaltrials@merz.de

+49 69 1503 1

Study Officials

• Merz Medical Expert

  Merz Pharmaceuticals GmbH

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 19, 2021
• First Posted: February 23, 2021
• Study Start: February 24, 2021
• Primary Completion: April 26, 2023
• Study Completion: November 21, 2023
• Last Updated: April 28, 2026
• Results First Posted: April 28, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

PL (4); US (9); CA (1)