Clinical trial NCT04766723: 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
Overall status: COMPLETED
Phase: PHASE2
Conditions: Essential Tremor of the Upper Limb
Lead sponsor: Merz Pharmaceuticals GmbH
Sponsor class: INDUSTRY
First posted: 2021-02-23
Enrollment: 78
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Change from study baseline to Week 6 in maximum tremor amplitude measurement at wrist level during the unilateral treatment period
timeFrame: From baseline to week 6
Secondary outcomes:
measure: Change from study baseline to Week 6 in the Essential Tremor Rating Assessment Scale [TETRAS] Performance dominant upper limb [UL] score
timeFrame: From baseline to week 6
measure: Change from study baseline to Week 6 in TETRAS Activities of Daily Living [ADL] UL score
timeFrame: From baseline to week 6
measure: Change from study baseline to Week 6 in TETRAS ADL Functional Impact score
timeFrame: From baseline to week 6
measure: Subject´s Global Impression of Change Scale [GICS] score of motor dominant UL at Week 6
timeFrame: Week 6
measure: Investigator´s Global Impression of Change Scale [GICS] score of motor dominant UL at Week 6
timeFrame: Week 6
measure: Change from Cycle 2 baseline to Week 6 in TETRAS Performance dominant UL score
timeFrame: Week 24 to week 36
measure: Change from Cycle 2 baseline to Week 6 in TETRAS Performance subscale score
timeFrame: Week 24 to week 36
measure: Change from Cycle 2 baseline to Week 6 in TETRAS ADL UL score
timeFrame: Week 24 to week 36
measure: Change from Cycle 2 baseline to Week 6 in TETRAS ADL Functional Impact score
timeFrame: Week 24 to week 36
measure: Incidence of treatment-emergent AEs [TEAEs] related to treatment
timeFrame: From baseline to week 36
Other outcomes:
Locations:
city: Tampa
country: United States
facility: USF, Department of Neurology, Merz Investigational Site #0010020
state: Florida
city: Omaha
country: United States
facility: University of Nebraska Medical Center, Merz Investigational Site #0010269
state: Nebraska
city: Las Vegas
country: United States
facility: Cleveland Clinic Lou Ruvo Center for Brain Health, Merz Investigational Site #0010457
state: Nevada
city: New York
country: United States
facility: Mount Sinai Medical Center, Merz Investigational Site #0010191
state: New York
city: Nashville
country: United States
facility: Vanderbilt University Medical Center, Neuroscience Institute, Merz Investigational Site #0010206
state: Tennessee
city: Houston
country: United States
facility: Houston Methodist Neurological Institute, Merz Investigational Site #0010226
state: Texas
city: McLean
country: United States
facility: Medstar Georgetown Neurology, Merz Investigational Site #0010231
state: Virginia
city: Seattle
country: United States
facility: UW Medical Center - Montlake, Merz Investigational Site #0010450
state: Washington
city: Spokane
country: United States
facility: Selkirk Neurology, Merz Investigational Site #0010456
state: Washington
city: London
country: Canada
facility: London Health Sciences Centre, Merz Investigational Site #0010087
state: Ontario
city: Krakow
country: Poland
facility: Specjalistyczne Gabinety, Merz Investigational Site #0480059
city: Lodz
country: Poland
facility: NeuroKlinika Gabinet Lekarski, Merz Investigational Site #0480101
city: Oświęcim
country: Poland
facility: Instytut Zdrowia Dr. Boczarska-Jedynak
city: Warsaw
country: Poland
facility: Mazowiecki Szpital Bródnowski, Merz Investigational Site #0480064
Study officials:
affiliation: Merz Pharmaceuticals GmbH
name: Merz Medical Expert
role: STUDY_DIRECTOR
References: