Study of BOTOX Injections to Assess Change in Disease Symptoms in Adult Participants With Upper Limb Essential Tremor

NCT05216250 | Completed Phase 2 FDA Drug

• 2 other identifiers: M21-4712021-002191-39
• Study Type: interventional
• Target: 174
• Locations: 2 countries
• Sites: 40

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.

Conditions

Upper Limb Essential Tremor (UL ET)

Keywords

Upper Limb Essential Tremor; UL ET; BOTOX; Botulinum Toxin Type A

Outcome Measures

Primary Outcomes (2)

• Change From Baseline in Tremor Disability Scale-Revised (TREDS-R) Total Score Across 7 Unilateral Items

  Tremor Disability Scale-Revised (TREDS-R) is a scale to assess activities of daily living (ADL) through a self-questionnaire asking participants to rate how easy or difficult it is to perform a particular activity. The scale consists of 20 items with each item rated on a scale of 1 (able to do the activity without difficulty) to 4 (cannot do the activity by yourself). 7 items included in this subset will be: use a spoon to drink soup, hold a cup of tea, pour milk from a bottle or carton, dial a telephone, pick up your change in a shop, insert an electric plug into a socket, unlock your front door with a key.

  Week 24

• Number of Participants with Treatment Emergent Adverse Events (AEs)

  An AE is defined as any untoward medical occurrence in a patient or clinical investigation in which a participant is administered a pharmaceutical product which does not necessarily have a causal relationship with this treatment.

  Up to approximately 36 weeks

Secondary Outcomes (6)

• Change From Baseline in TETRAS Activity of Daily Living (TETRAS ADL) Total Score Across 5 Unilateral Items

  Week 24

• Change From Baseline in TETRAS Archimedes Spiral Score

  Week 24

• Change From Baseline in TETRAS Handwriting Score

  Week 24

• Change From Baseline in Clinical Global Impression of Severity (CGI-S)

  Week 24

• Change From Baseline in Patient Global Impression of Severity (PGI-S)

  Week 24

Study Arms (4)

BOTOX/BOTOX Unilateral

EXPERIMENTAL

Participants will receive BOTOX in Cycle 1 and Cycle 2 and unilateral BOTOX in Cycle 3

Biological: BOTOX

BOTOX/BOTOX Bilateral

EXPERIMENTAL

Participants will receive BOTOX in Cycle 1 and Cycle 2 and bilateral BOTOX in Cycle 3

Biological: BOTOX

Placebo/BOTOX Unilateral

EXPERIMENTAL

Participants will receive placebo in Cycle 1 and Cycle 2 followed by unilateral BOTOX in Cycle 3.

Biological: BOTOX; Drug: Placebo for BOTOX

Placebo/BOTOX Bilateral

EXPERIMENTAL

Participants will receive placebo in Cycle 1 and Cycle 2 followed by bilateral BOTOX in Cycle 3.

Biological: BOTOX; Drug: Placebo for BOTOX

Interventions

BOTOX BIOLOGICAL

Intramuscular injection

Also known as: Botulinum Toxin Type A

BOTOX/BOTOX Bilateral; BOTOX/BOTOX Unilateral; Placebo/BOTOX Bilateral; Placebo/BOTOX Unilateral

Placebo for BOTOX DRUG

Intramuscular injection

Placebo/BOTOX Bilateral; Placebo/BOTOX Unilateral

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• AbbVie: lead

Study Sites (40)

Accel Research Sites - Neurology and Neurodiagnostics of Alabama, LLC /ID# 240472

Hoover, Alabama, 35244-5700, United States

Location

Barrow Neurological Institute - Dignity Health St. Joseph's Hosp and Medical Ctr /ID# 231731

Phoenix, Arizona, 85013, United States

Location

University of Arizona /ID# 239781

Tucson, Arizona, 85724, United States

Location

Loma Linda University /ID# 230905

Loma Linda, California, 92354, United States

Location

University of Colorado - Anschutz Medical Campus /ID# 239869

Aurora, Colorado, 80045-2527, United States

Location

New England Institute for Clinical Research /ID# 238404

Stamford, Connecticut, 06905, United States

Location

Parkinson's Disease and Movement Disorders Center of Boca Raton /ID# 231677

Boca Raton, Florida, 33486, United States

Location

Coastal Clinical Research Specialists /ID# 240897

Fernandina Beach, Florida, 32034-4779, United States

Location

Coastal Clinical Research Specialists /ID# 240471

Jacksonville, Florida, 32205-8275, United States

Location

Charter Research - Winter Park /ID# 241568

Orlando, Florida, 32803-1839, United States

Location

CDC Research Institute LLC /ID# 242744

Port Saint Lucie, Florida, 34952-7539, United States

Location

Emory University / Emory Brain Health Center /ID# 231911

Atlanta, Georgia, 30329-2206, United States

Location

Accel Research Site-NeuroStudies /ID# 239881

Decatur, Georgia, 30030-2627, United States

Location

Univ Kansas Med Ctr /ID# 231166

Kansas City, Kansas, 66160, United States

Location

Kansas Institute of Research /ID# 231623

Overland Park, Kansas, 66211-1363, United States

Location

Tufts Medical Center /ID# 239929

Boston, Massachusetts, 02111-1552, United States

Location

Lahey Hospital & Medical Center /ID# 233232

Burlington, Massachusetts, 01805, United States

Location

Henry Ford Health Medical Center - Jackson /ID# 231400

Jackson, Michigan, 49201-1852, United States

Location

Dent Neurologic Institute - Amherst /ID# 255665

Amherst, New York, 14226, United States

Location

UNC Hospitals Neurology Clinic - Chapel Hill /ID# 238857

Chapel Hill, North Carolina, 27517-4400, United States

Location

Neurology - Triad /ID# 239806

Winston-Salem, North Carolina, 27103-6984, United States

Location

Atrium Health Wake Forest Baptist Medical Center /ID# 233550

Winston-Salem, North Carolina, 27157-0001, United States

Location

Dayton Center for Neurological Disorders /ID# 233737

Centerville, Ohio, 45459-3811, United States

Location

The Orthopedic Foundation /ID# 232234

New Albany, Ohio, 43054-8167, United States

Location

Oregon Health and Science University /ID# 231581

Portland, Oregon, 97239, United States

Location

Thomas Jefferson University Hospital /ID# 232614

Philadelphia, Pennsylvania, 19107, United States

Location

University of Pittsburgh MC /ID# 233735

Pittsburgh, Pennsylvania, 15260, United States

Location

Medical University of South Carolina /ID# 233538

Charleston, South Carolina, 29425, United States

Location

Texas Neurology /ID# 250428

Dallas, Texas, 75214, United States

Location

University of Texas Southwestern Medical Center /ID# 239770

Dallas, Texas, 75390-7208, United States

Location

Texas Movement Disorder Specialists, PLLC /ID# 242524

Georgetown, Texas, 78628-4126, United States

Location

Baylor College of Medicine /ID# 231624

Houston, Texas, 77030, United States

Location

Univ Texas HSC San Antonio /ID# 232615

San Antonio, Texas, 78229-3901, United States

Location

Integrated Neurology Services - Falls Church /ID# 233545

Falls Church, Virginia, 22043-2367, United States

Location

Gamma Therapeutic Center /ID# 232547

Greenfield, Wisconsin, 53228-1321, United States

Location

Marshfield Clinic - Marshfield /ID# 233191

Marshfield, Wisconsin, 54449, United States

Location

Vancouver Coastal Health Authority - University of British Columbia /ID# 240356

Vancouver, British Columbia, V6T 1Z9, Canada

Location

Centricity Research /ID# 240587

Halifax, Nova Scotia, B3S 1N2, Canada

Location

Toronto Western Hospital /ID# 231587

Toronto, Ontario, M5T 2S8, Canada

Location

Genge Partners /ID# 251546

Montreal, Quebec, H4A 3T2, Canada

Location

Related Links

• Related Info

MeSH Terms

Interventions

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

Study Officials

• ABBVIE INC.

  AbbVie

  STUDY DIRECTOR

Study Design

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

Study Record Dates

• First Submitted: October 1, 2021
• First Posted: January 31, 2022
• Study Start: April 12, 2022
• Primary Completion: February 3, 2025
• Study Completion: February 3, 2025
• Last Updated: January 20, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR
• Time Frame: For details on when studies are available for sharing, please refer to the link below.
• Access Criteria: Access to this clinical trial data can be requested by any qualified researchers who engage in rigorous, independent scientific research, and will be provided following review and approval of a research proposal and Statistical Analysis Plan (SAP) and execution of a Data Use Agreement (DUA). For more information on the process, or to submit a request, visit the following link.

Locations

US (36); CA (4)