Clinical trial NCT04849988: A Phase 2 Study to Evaluate the Safety and Efficacy of ABP-450 in the Treatment of Cervical Dystonia
Official title: A Phase 2, Randomized, Double-Blind, Multicenter, Placebo Controlled Study to Evaluate the Safety and Efficacy of Intramuscular ABP-450 (prabotulinumtoxinA) Injection for the Treatment of Cervical Dystonia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Cervical Dystonia
Lead sponsor: AEON Biopharma, Inc.
Sponsor class: INDUSTRY
Collaborators: name: PPD Development, LP
First posted: 2021-04-20
Enrollment: 61
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Approximately 60 patients will be randomized in a 1:1:1:1 ratio and receive one of the four treatments: ABP-450 low dose, ABP-450 mid dose, ABP-450 high dose, or placebo via intramuscular injection into affected neck muscles.
Primary purpose: TREATMENT

Brief summary: This Phase 2 trial will evaluate the safety and efficacy of ABP-450 for the treatment of cervical dystonia in adults. The study will enroll 60 patients across approximately 30 sites in the United States. Study subjects will be divided evenly across a low dose group, a medium dose group, a high dose group, and a placebo group for one treatment cycle.

Detailed description: This Phase 2 trial will evaluate the safety and efficacy of ABP-450 for the treatment of cervical dystonia in adults. The study will enroll 60 patients across approximately 30 sites in the United States. Study subjects will be divided evenly across a low dose, a medium dose group, a high dose and placebo group for one treatment cycle.

Eligibility criteria: Inclusion Criteria:

1. Male or female patients between 18 and 75 years of age (inclusive)
2. A clinical diagnosis of cervical dystonia (ie, spasmodic torticollis) defined by:

   * TWSTRS total score ≥20
   * TWSTRS severity score ≥10
   * TWSTRS disability score ≥3
   * TWSTRS pain score ≥1
3. On a stable dose of medications (if any) used for focal dystonia treatment (eg, anticholinergics and benzodiazepines) for at least 3 months prior to and expected throughout the study duration
4. For pre-treated patients only: Source documentation (eg, patient history) of the last 2 consecutive injection sessions with a botulinum toxin type A
5. For pre-treated patients only: At least 16 weeks must have passed between the last injection with botulinum toxin for cervical dystonia and baseline treatment (patients can be screened at Week 15 but cannot be enrolled until 16 weeks \[for Day 0 injection\])
6. Provided written informed consent to being treated for cervical dystonia with ABP-450
7. Stated willingness to comply with all study procedures, including attendance at the study center for all study visits as scheduled and have technological capabilities to have televisits

Exclusion Criteria:

1. Traumatic torticollis or tardive torticollis
2. Predominant retrocollis or anterocollis
3. Myotomy or denervation surgery in the affected muscles (eg, peripheral denervation and/or spinal cord stimulation)
4. Hypersensitivity to human serum albumin, sucrose, or botulinum toxin type A
5. Previous treatment for cervical dystonia with rimabotulinumtoxin B
6. Diagnosis of myasthenia gravis, Lambert-Eaton syndrome, amyotrophic lateral sclerosis, or any other significant neuromuscular disease that might interfere with the trial
7. Current swallowing disorder of any origin (dysphagia scale ≥3, ie, severe, with swallowing difficulties and requiring a change in diet)
8. Marked limitation on passive range of motion that suggests contractures or other structural abnormality, eg, cervical contractures or cervical spine syndrome
9. Treatment with botulinum toxins of any type for any indication other than cervical dystonia within 16 weeks prior to baseline and during the study
10. Medical or psychiatric conditions that may increase the risk associated with study participation or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the patient inappropriate for entry into this study
11. Participation in another interventional study during participation in this study
12. Pregnant or lactating females, or females of child-bearing potential not willing to use an acceptable method of contraception (ie, intrauterine device, barrier methods with spermicide, or abstinence)
13. For pre-treated patients only: The patient's most recent injection with botulinum toxin exceeding the number of units specified as follows:

    * OnabotulinumtoxinA (BOTOX®): \>300 units
    * IncobotulinumtoxinA (Xeomin®): \>300 units
    * AbobotulinumtoxinA (Dysport®): \>750 units

Primary outcomes:
measure: Number of Participants With Treatment-Related Serious Adverse Events
timeFrame: Baseline up to 20 Weeks
Secondary outcomes:
measure: Mean Change of the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS)-Total Score From Baseline to Week 4
timeFrame: Baseline to Week 4
measure: Mean Change in the Subscale Score of Disability of the Baseline Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) From Baseline to Week 4
timeFrame: Baseline to Week 4
measure: Mean Change in the Subscale Score of Pain of the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) From Baseline to Week 4
timeFrame: Baseline to Week 4
measure: Mean Change in the Subscale Score of Severity of the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) From Baseline to Week 4
timeFrame: Baseline to Week 4
measure: Mean Change in Patient Global Impression of Change (PGI-C) From Baseline to Week 4
timeFrame: Baseline to Week 4
measure: Mean Change in Clinical Global Impression of Change (CGI-C) From Baseline to Week 4
timeFrame: Baseline to Week 4
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Arizona Neuroscience Research
state: Arizona
city: Scottsdale
country: United States
facility: Movement Disorder Center of Arizona
state: Arizona
city: Fountain Valley
country: United States
facility: Parkinson's and Movement Disorder Institute
state: California
city: Fresno
country: United States
facility: Neuro Pain Medical Center
state: California
city: Loma Linda
country: United States
facility: Loma Linda University
state: California
city: Stamford
country: United States
facility: New England Institute for Neurology and Headache
state: Connecticut
city: Hollywood
country: United States
facility: Infinity Clinical Research LLC
state: Florida
city: Miami
country: United States
facility: The Neurology Research Group
state: Florida
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: Winter Park
country: United States
facility: Neurology One
state: Florida
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: East Lansing
country: United States
facility: Michigan State University
state: Michigan
city: Farmington Hills
country: United States
facility: Quest Research Institute - Hunt - PPDS
state: Michigan
city: Albuquerque
country: United States
facility: University of New Mexico
state: New Mexico
city: Cleveland
country: United States
facility: The Cleveland Clinic Foundation
state: Ohio
city: Cleveland
country: United States
facility: Cleveland Clinic Lou Ruvo Center for Brain Health
state: Ohio
city: New Albany
country: United States
facility: The Orthopedic Foundation
state: Ohio
city: Memphis
country: United States
facility: Veracity Neuroscience LLC
state: Tennessee
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
Study officials:
affiliation: Rush University Medical Center
name: Cynthia Comella, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Baylor St. Luke's Medical Center
name: Joseph Jankovic
role: PRINCIPAL_INVESTIGATOR
References: