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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

Primary Purpose

Essential Tremor of the Upper Limb

Status
Active
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
NT 201
Placebo
Sponsored by
Merz Pharmaceuticals GmbH
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Essential Tremor of the Upper Limb

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • USF, Department of Neurology, Merz Investigational Site #0010020
  • University of Nebraska Medical Center, Merz Investigational Site #0010269
  • Cleveland Clinic Lou Ruvo Center for Brain Health, Merz Investigational Site #0010457
  • Mount Sinai Medical Center, Merz Investigational Site #0010191
  • Vanderbilt University Medical Center, Neuroscience Institute, Merz Investigational Site #0010206
  • Houston Methodist Neurological Institute, Merz Investigational Site #0010226
  • Medstar Georgetown Neurology, Merz Investigational Site #0010231
  • UW Medical Center - Montlake, Merz Investigational Site #0010450
  • Selkirk Neurology, Merz Investigational Site #0010456
  • London Health Sciences Centre, Merz Investigational Site #0010087
  • Specjalistyczne Gabinety, Merz Investigational Site #0480059
  • NeuroKlinika Gabinet Lekarski, Merz Investigational Site #0480101
  • Instytut Zdrowia Dr. Boczarska-Jedynak
  • Mazowiecki Szpital Bródnowski, Merz Investigational Site #0480064

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

NT 201 (IncobotulinumtoxinA, Xeomin)

Placebo

Arm Description

Unilateral 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.

Unilateral 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.

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Change from study baseline to Week 6 in the Essential Tremor Rating Assessment Scale [TETRAS] Performance dominant upper limb [UL] score
Change from study baseline to Week 6 in TETRAS Activities of Daily Living [ADL] UL score
Change from study baseline to Week 6 in TETRAS ADL Functional Impact score
Subject´s Global Impression of Change Scale [GICS] score of motor dominant UL at Week 6
Investigator´s Global Impression of Change Scale [GICS] score of motor dominant UL at Week 6
Change from Cycle 2 baseline to Week 6 in TETRAS Performance dominant UL score
Change from Cycle 2 baseline to Week 6 in TETRAS Performance subscale score
Change from Cycle 2 baseline to Week 6 in TETRAS ADL UL score
Change from Cycle 2 baseline to Week 6 in TETRAS ADL Functional Impact score
Incidence of treatment-emergent AEs [TEAEs] related to treatment

Full Information

First Posted
February 19, 2021
Last Updated
October 18, 2023
Sponsor
Merz Pharmaceuticals GmbH
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1. Study Identification

Unique Protocol Identification Number
NCT04766723
Brief Title
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
Official Title
Prospective, Randomized, Double-blind, Placebo-controlled, Parallel-group Trial With an Open-label Period to Investigate the Efficacy and Safety of NT 201 in the Unilateral and Bilateral Treatment of Essential Tremor of the Upper Limb
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
February 24, 2021 (Actual)
Primary Completion Date
April 26, 2023 (Actual)
Study Completion Date
November 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Merz Pharmaceuticals GmbH

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Essential Tremor of the Upper Limb

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
78 (Actual)

8. Arms, Groups, and Interventions

Arm Title
NT 201 (IncobotulinumtoxinA, Xeomin)
Arm Type
Experimental
Arm Description
Unilateral 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.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Unilateral 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.
Intervention Type
Drug
Intervention Name(s)
NT 201
Other Intervention Name(s)
Incobotulinumtoxin A, IncobotulinumtoxinA, Xeomin, Botulinum toxin type A (150 kiloDalton), free from complexing proteins
Intervention Description
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).
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Solution for injection prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl).
Primary Outcome Measure Information:
Title
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 Outcome Measure Information:
Title
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
Title
Change from study baseline to Week 6 in TETRAS Activities of Daily Living [ADL] UL score
Time Frame
From baseline to week 6
Title
Change from study baseline to Week 6 in TETRAS ADL Functional Impact score
Time Frame
From baseline to week 6
Title
Subject´s Global Impression of Change Scale [GICS] score of motor dominant UL at Week 6
Time Frame
Week 6
Title
Investigator´s Global Impression of Change Scale [GICS] score of motor dominant UL at Week 6
Time Frame
Week 6
Title
Change from Cycle 2 baseline to Week 6 in TETRAS Performance dominant UL score
Time Frame
Week 24 to week 36
Title
Change from Cycle 2 baseline to Week 6 in TETRAS Performance subscale score
Time Frame
Week 24 to week 36
Title
Change from Cycle 2 baseline to Week 6 in TETRAS ADL UL score
Time Frame
Week 24 to week 36
Title
Change from Cycle 2 baseline to Week 6 in TETRAS ADL Functional Impact score
Time Frame
Week 24 to week 36
Title
Incidence of treatment-emergent AEs [TEAEs] related to treatment
Time Frame
From baseline to week 36

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Merz Medical Expert
Organizational Affiliation
Merz Pharmaceuticals GmbH
Official's Role
Study Director
Facility Information:
Facility Name
USF, Department of Neurology, Merz Investigational Site #0010020
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
Facility Name
University of Nebraska Medical Center, Merz Investigational Site #0010269
City
Omaha
State/Province
Nebraska
ZIP/Postal Code
68131
Country
United States
Facility Name
Cleveland Clinic Lou Ruvo Center for Brain Health, Merz Investigational Site #0010457
City
Las Vegas
State/Province
Nevada
ZIP/Postal Code
89106
Country
United States
Facility Name
Mount Sinai Medical Center, Merz Investigational Site #0010191
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
Vanderbilt University Medical Center, Neuroscience Institute, Merz Investigational Site #0010206
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
Houston Methodist Neurological Institute, Merz Investigational Site #0010226
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Medstar Georgetown Neurology, Merz Investigational Site #0010231
City
McLean
State/Province
Virginia
ZIP/Postal Code
22101
Country
United States
Facility Name
UW Medical Center - Montlake, Merz Investigational Site #0010450
City
Seattle
State/Province
Washington
ZIP/Postal Code
98195
Country
United States
Facility Name
Selkirk Neurology, Merz Investigational Site #0010456
City
Spokane
State/Province
Washington
ZIP/Postal Code
99202
Country
United States
Facility Name
London Health Sciences Centre, Merz Investigational Site #0010087
City
London
State/Province
Ontario
ZIP/Postal Code
N6A 5A5
Country
Canada
Facility Name
Specjalistyczne Gabinety, Merz Investigational Site #0480059
City
Krakow
ZIP/Postal Code
30-539
Country
Poland
Facility Name
NeuroKlinika Gabinet Lekarski, Merz Investigational Site #0480101
City
Lodz
ZIP/Postal Code
90-640
Country
Poland
Facility Name
Instytut Zdrowia Dr. Boczarska-Jedynak
City
Oświęcim
ZIP/Postal Code
32-600
Country
Poland
Facility Name
Mazowiecki Szpital Bródnowski, Merz Investigational Site #0480064
City
Warsaw
ZIP/Postal Code
03-242
Country
Poland

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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