Study of BOTOX Injections to Assess Change in Disease Symptoms in Adult Participants With Upper Limb Essential Tremor
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 |
---|---|---|
|
Phase 2 |
Study Design
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:
|
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:
|
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:
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:
Drug: Placebo for BOTOX
Intramuscular injection
|
Outcome Measures
Primary Outcome Measures
- 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
- 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.
- 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."
- 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."
- 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."
- 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.
- 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
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.
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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.
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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 | |
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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.- M21-471
- 2021-002191-39