Clinical trial NCT05216250: Study of BOTOX Injections to Assess Change in Disease Symptoms in Adult Participants With Upper Limb Essential Tremor
Official title: A Phase 2 Multicenter, Randomized, Double-blind, Placebo-controlled Study of BOTOX® (Botulinum Toxin Type A) Purified Neurotoxin Complex for the Treatment of Upper Limb Essential Tremor
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Upper Limb Essential Tremor (UL ET)
Keywords: Upper Limb Essential Tremor
UL ET
BOTOX
Botulinum Toxin Type A
Lead sponsor: AbbVie
Sponsor class: INDUSTRY
First posted: 2022-01-31
Enrollment: 174
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Upper limb essential tremor (UL ET) is a movement disorder characterized by postural and/or kinetic tremor. It can cause difficulty with everyday tasks such as writing, pouring, and eating, and patients also experience associated social embarrassment. This study will assess how safe and effective BOTOX is in treating UL ET. Adverse events and change in disease activity will be evaluated.

BOTOX is an investigational drug being developed for the treatment of UL ET. Participants are randomly assigned to 1 of the 4 groups, called treatment arms. Each group receives different treatment. There is 1 in 2 chance that participants will be assigned to placebo. Around 174 participants, aged 18 to 80 years with UL ET will be enrolled in approximately 40 sites in North America.

Participants will receive BOTOX or placebo injections in Cycle 1 and Cycle 2. In Cycle 3, participants will receive unilateral or bilateral BOTOX injections. Each cycle is 12 weeks.

There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires.


Eligibility criteria: Inclusion Criteria:

* Diagnosis of essential tremor in accordance with modified Tremor Investigation Group (TRIG) criteria as described in the protocol.
* TREDS-Revised Scale (1-4 scale, whole numbers) unilateral score of \>= 3 for the Tremor Disability Scale (TREDS) on any of the 7 unilateral items; no more than a single item score of 1 among the 7 unilateral items in the dominant limb.
* TETRAS activities of daily living (ADL) (recorded on a 0-4 scale, whole numbers) minimum score of \>= 3 on any of the 5 unilateral items; no more than a single item score of \<=1 among the 5 unilateral items in the dominant limb.
* At least one of the following criteria must also be met:

  * TETRAS UL score (0-4 scale, with 0.5 increments) of \> 2 in the dominant limb on at least one of the 3 maneuvers OR
  * TETRAS Archimedes spiral task score (0-4 scale, with 0.5 increments) of \> 2 in the dominant limb

Exclusion Criteria:

* Any uncontrolled clinically significant medical condition other than the one under study.
* Any medical condition that may put the participant at increased risk with exposure to BOTOX Purified Neurotoxin Complex.

Primary outcomes:
measure: Change From Baseline in Tremor Disability Scale-Revised (TREDS-R) Total Score Across 7 Unilateral Items
timeFrame: Week 24
measure: Number of Participants with Treatment Emergent Adverse Events (AEs)
timeFrame: Up to approximately 36 weeks
Secondary outcomes:
measure: Change From Baseline in TETRAS Activity of Daily Living (TETRAS ADL) Total Score Across 5 Unilateral Items
timeFrame: Week 24
measure: Change From Baseline in TETRAS Archimedes Spiral Score
timeFrame: Week 24
measure: Change From Baseline in TETRAS Handwriting Score
timeFrame: Week 24
measure: Change From Baseline in Clinical Global Impression of Severity (CGI-S)
timeFrame: Week 24
measure: Change From Baseline in Patient Global Impression of Severity (PGI-S)
timeFrame: Week 24
measure: Change From Baseline in Essential Tremor Rating Assessment (TETRAS UL) Score
timeFrame: Week 24
Other outcomes:
Locations:
city: Hoover
country: United States
facility: Accel Research Sites - Neurology and Neurodiagnostics of Alabama, LLC /ID# 240472
state: Alabama
city: Phoenix
country: United States
facility: Barrow Neurological Institute - Dignity Health St. Joseph's Hosp and Medical Ctr /ID# 231731
state: Arizona
city: Tucson
country: United States
facility: University of Arizona /ID# 239781
state: Arizona
city: Loma Linda
country: United States
facility: Loma Linda University /ID# 230905
state: California
city: Aurora
country: United States
facility: University of Colorado - Anschutz Medical Campus /ID# 239869
state: Colorado
city: Stamford
country: United States
facility: New England Institute for Clinical Research /ID# 238404
state: Connecticut
city: Boca Raton
country: United States
facility: Parkinson's Disease and Movement Disorders Center of Boca Raton /ID# 231677
state: Florida
city: Fernandina Beach
country: United States
facility: Coastal Clinical Research Specialists /ID# 240897
state: Florida
city: Jacksonville
country: United States
facility: Coastal Clinical Research Specialists /ID# 240471
state: Florida
city: Orlando
country: United States
facility: Charter Research - Winter Park /ID# 241568
state: Florida
city: Port Saint Lucie
country: United States
facility: CDC Research Institute LLC /ID# 242744
state: Florida
city: Atlanta
country: United States
facility: Emory University / Emory Brain Health Center /ID# 231911
state: Georgia
city: Decatur
country: United States
facility: Accel Research Site-NeuroStudies /ID# 239881
state: Georgia
city: Kansas City
country: United States
facility: Univ Kansas Med Ctr /ID# 231166
state: Kansas
city: Overland Park
country: United States
facility: Kansas Institute of Research /ID# 231623
state: Kansas
city: Boston
country: United States
facility: Tufts Medical Center /ID# 239929
state: Massachusetts
city: Burlington
country: United States
facility: Lahey Hospital & Medical Center /ID# 233232
state: Massachusetts
city: Jackson
country: United States
facility: Henry Ford Health Medical Center - Jackson /ID# 231400
state: Michigan
city: Amherst
country: United States
facility: Dent Neurologic Institute - Amherst /ID# 255665
state: New York
city: Chapel Hill
country: United States
facility: UNC Hospitals Neurology Clinic - Chapel Hill /ID# 238857
state: North Carolina
city: Winston-Salem
country: United States
facility: Neurology - Triad /ID# 239806
state: North Carolina
city: Winston-Salem
country: United States
facility: Atrium Health Wake Forest Baptist Medical Center /ID# 233550
state: North Carolina
city: Centerville
country: United States
facility: Dayton Center for Neurological Disorders /ID# 233737
state: Ohio
city: New Albany
country: United States
facility: The Orthopedic Foundation /ID# 232234
state: Ohio
city: Portland
country: United States
facility: Oregon Health and Science University /ID# 231581
state: Oregon
city: Philadelphia
country: United States
facility: Thomas Jefferson University Hospital /ID# 232614
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh MC /ID# 233735
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina /ID# 233538
state: South Carolina
city: Dallas
country: United States
facility: Texas Neurology /ID# 250428
state: Texas
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center /ID# 239770
state: Texas
city: Georgetown
country: United States
facility: Texas Movement Disorder Specialists, PLLC /ID# 242524
state: Texas
city: Houston
country: United States
facility: Baylor College of Medicine /ID# 231624
state: Texas
city: San Antonio
country: United States
facility: Univ Texas HSC San Antonio /ID# 232615
state: Texas
city: Falls Church
country: United States
facility: Integrated Neurology Services - Falls Church /ID# 233545
state: Virginia
city: Greenfield
country: United States
facility: Gamma Therapeutic Center /ID# 232547
state: Wisconsin
city: Marshfield
country: United States
facility: Marshfield Clinic - Marshfield /ID# 233191
state: Wisconsin
city: Vancouver
country: Canada
facility: Vancouver Coastal Health Authority - University of British Columbia /ID# 240356
state: British Columbia
city: Halifax
country: Canada
facility: Centricity Research /ID# 240587
state: Nova Scotia
city: Toronto
country: Canada
facility: Toronto Western Hospital /ID# 231587
state: Ontario
city: Montreal
country: Canada
facility: Genge Partners /ID# 251546
state: Quebec
Study officials:
affiliation: AbbVie
name: ABBVIE INC.
role: STUDY_DIRECTOR
References: