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

Sponsor
AbbVie (Industry)
Overall Status
Recruiting
CT.gov ID
NCT05216250
Collaborator
(none)
174
40
4
19.5
4.4
0.2

Study Details

Study Description

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 35 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.

Condition or Disease Intervention/Treatment Phase
  • Biological: BOTOX
  • Drug: Placebo for BOTOX
Phase 2

Study Design

Study Type:
Interventional
Anticipated Enrollment :
174 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose:
Treatment
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
Actual Study Start Date :
Apr 12, 2022
Anticipated Primary Completion Date :
Aug 15, 2023
Anticipated Study Completion Date :
Nov 28, 2023

Arms and Interventions

Arm Intervention/Treatment
Experimental: BOTOX/BOTOX Unilateral

Participants will receive BOTOX dose A in Cycle 1, BOTOX dose B in Cycle 2 and unilateral BOTOX in Cycle 3.

Biological: BOTOX
Intramuscular injection
Other Names:
  • Botulinum Toxin Type A
  • Experimental: BOTOX/BOTOX Bilateral

    Participants will receive BOTOX dose A in Cycle 1, BOTOX dose B in Cycle 2 and bilateral BOTOX in Cycle 3.

    Biological: BOTOX
    Intramuscular injection
    Other Names:
  • Botulinum Toxin Type A
  • Experimental: Placebo/BOTOX Unilateral

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

    Biological: BOTOX
    Intramuscular injection
    Other Names:
  • Botulinum Toxin Type A
  • Drug: Placebo for BOTOX
    Intramuscular injection

    Experimental: Placebo/BOTOX Bilateral

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

    Biological: BOTOX
    Intramuscular injection
    Other Names:
  • Botulinum Toxin Type A
  • Drug: Placebo for BOTOX
    Intramuscular injection

    Outcome Measures

    Primary Outcome Measures

    1. Change From Baseline in Tremor Disability Scale-Revised (TREDS-R) Total Score Across 7 Unilateral Items [Week 24]

      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.

    Secondary Outcome Measures

    1. Change From Baseline in TETRAS Activity of Daily Living (TETRAS ADL) Total Score Across 5 Unilateral Items [Week 24]

      TETRAS ADL is a clinical rating scale consisting of 10 items, with each item rated on a scale of 0 (normal) to 3 (severe - abnormal) measuring tremor's impact on ADL. The 5 tasks included in this subset will be feeding with a spoon, drinking from a glass, pouring, using keys, and writing.

    2. Change From Baseline in TETRAS Archimedes Spiral Score [Week 24]

      Tremor Research Group Essential Tremor Rating Assessment Scale (TETRAS) Archimedes Spiral is an item of the TETRAS performance subscale that is designed to assess tremor severity. During this test, participants are instructed to draw an unbroken spiral which is then scored from 0 to 4, with 1 being tremor is "barely visible" and 4 being "figure not recognizable."

    3. Change From Baseline in TETRAS Handwriting Score [Week 24]

      TETRAS Handwriting is an item of the TETRAS performance subscale that is designed to assess tremor severity. During this test, participants are instructed to write the standard sentence "This is a sample of my best handwriting" using the dominant hand only, which is then scored from 0 to 4 with 1 being tremor is "slight: untidy due to tremor that is barely visible" and 4 being "severe: completely illegible."

    4. Change From Baseline in Clinical Global Impression of Severity (CGI-S) [Week 24]

      CGI-S is a single item used to measure the clinician's impression of the current severity of the patient's essential tremor. The measure uses a 5-point rating scale where "0 = none, 1 = mild, 2 = moderate, 3 = severe, and 4 = very severe."

    5. Change From Baseline in Patient Global Impression of Severity (PGI-S) [Week 24]

      PGI-S is a single item used to measure the participant's impression of severity of their essential tremor symptoms. The measure uses a 5-point rating scale where 0 = none, 1 = mild, 2 = moderate, 3 = severe, and 4 = very severe.

    6. Change From Baseline in Essential Tremor Rating Assessment (TETRAS UL) Score [Week 24]

      TETRAS Upper Limb (UL) Scale is an item of the TETRAS performance subscale that is designed to assess upper limb tremor during three maneuvers: forward horizontal reach posture, lateral "wing beating" posture, and finger-nose-finger testing. Each maneuver is scored from 0 to 4, with 1 indicating a tremor amplitude that is "barely visible" and 4 indicating a tremor amplitude that is >= 20 cm.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years to 80 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:
    • Diagnosis of essential tremor in accordance with modified Tremor Investigation Group (TRIG) criteria as described in the protocol.

    • The Essential Tremor Rating Assessment Scale (TETRAS) upper limit (UL) score (0-4 scale) > 2 in the dominant limb on at least one of the 3 maneuvers.

    • TETRAS Archimedes spiral task score > 2 in the dominant limb.

    • TREDS-Revised Scale (1-4 scale) 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) (0-4 scale) 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.

    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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Accel Research Sites - Neurology and Neurodiagnostics of Alabama, LLC /ID# 240472 Hoover Alabama United States 35244-5700
    2 Barrow Neurological Institute /ID# 231731 Phoenix Arizona United States 85013-4407
    3 University of Arizona /ID# 239781 Tucson Arizona United States 85724
    4 Loma Linda University /ID# 230905 Loma Linda California United States 92354
    5 University of Colorado - Anschutz Medical Campus /ID# 239869 Aurora Colorado United States 80045-2527
    6 Ki Health Partners, LLC, dba, New England Institute for Clinical Research /ID# 238404 Stamford Connecticut United States 06905
    7 Parkinson's Disease and Movement Disorders Center of Boca Raton /ID# 231677 Boca Raton Florida United States 33486
    8 Coastal Clinical Research Specialists /ID# 240897 Fernandina Beach Florida United States 32034-4779
    9 Coastal Clinical Research Specialists /ID# 240471 Jacksonville Beach Florida United States 32250-1694
    10 CDC Research Institute LLC /ID# 242744 Port Saint Lucie Florida United States 34952-7539
    11 Charter Research - Winter Park /ID# 241568 Winter Park Florida United States 32792-6707
    12 Emory University / Emory Brain Health Center /ID# 231911 Atlanta Georgia United States 30329-2206
    13 Accel Research Site-NeuroStudies /ID# 239881 Decatur Georgia United States 30030-2627
    14 Univ Kansas Med Ctr /ID# 231166 Kansas City Kansas United States 66160
    15 Kansas Institute of Research /ID# 231623 Overland Park Kansas United States 66211-1363
    16 Tufts Medical Center /ID# 239929 Boston Massachusetts United States 02111-1552
    17 Lahey Hospital and Medical Center /ID# 233232 Burlington Massachusetts United States 01805
    18 Henry Ford Health Medical Center - Jackson /ID# 231400 Jackson Michigan United States 49201-1852
    19 University of Missouri /ID# 233815 Columbia Missouri United States 65203
    20 Icahn School of Medicine at Mount Sinai - The Mount Sinai Medical Center /ID# 231468 New York New York United States 10029
    21 UNC Hospitals Neurology Clinic - Chapel Hill /ID# 238857 Chapel Hill North Carolina United States 27517-4400
    22 Neurology - Triad /ID# 239806 Winston-Salem North Carolina United States 27103-6984
    23 Wake Forest Baptist Medical Center /ID# 233550 Winston-Salem North Carolina United States 27157-0001
    24 Dayton Center for Neurological Disorders /ID# 233737 Centerville Ohio United States 45459-3811
    25 The Orthopedic Foundation /ID# 232234 New Albany Ohio United States 43054-8167
    26 Oregon Health & Science University /ID# 231581 Portland Oregon United States 97239-3011
    27 Thomas Jefferson University /ID# 232614 Philadelphia Pennsylvania United States 19107-4414
    28 University of Pittsburgh MC /ID# 233735 Pittsburgh Pennsylvania United States 15260
    29 Medical University of South Carolina /ID# 233538 Charleston South Carolina United States 29425
    30 University of Texas Southwestern Medical Center /ID# 239770 Dallas Texas United States 75390-7208
    31 Texas Movement Disorder Specialists, PLLC /ID# 242524 Georgetown Texas United States 78628-4126
    32 Baylor College of Medicine - Baylor Medical Center /ID# 231624 Houston Texas United States 77030-3411
    33 Integrated Neurology Services - Falls Church /ID# 233545 Falls Church Virginia United States 22043-2367
    34 Marshfield Clinic - Marshfield /ID# 233191 Marshfield Wisconsin United States 54449
    35 Gamma Therapeutic Center /ID# 232547 Milwaukee Wisconsin United States 53228-1321
    36 Vancouver Coastal Health Authority - University of British Columbia /ID# 240356 Vancouver British Columbia Canada V6T 1Z9
    37 Centricity Research /ID# 240587 Halifax Nova Scotia Canada B3S 1N2
    38 Toronto Western Hospital /ID# 231587 Toronto Ontario Canada M5T 2S8
    39 Montreal Neurological Institut /ID# 232629 Montreal Quebec Canada H3A 2B4
    40 Greene Avenue Clinic /ID# 244376 Westmount Quebec Canada H3Z 2A4

    Sponsors and Collaborators

    • AbbVie

    Investigators

    • Study Director: ABBVIE INC., AbbVie

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    AbbVie
    ClinicalTrials.gov Identifier:
    NCT05216250
    Other Study ID Numbers:
    • M21-471
    • 2021-002191-39
    First Posted:
    Jan 31, 2022
    Last Update Posted:
    Aug 24, 2022
    Last Verified:
    Aug 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    Yes
    Plan to Share IPD:
    Yes
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Product Manufactured in and Exported from the U.S.:
    No
    Keywords provided by AbbVie
    Additional relevant MeSH terms:

    Study Results

    No Results Posted as of Aug 24, 2022