A Phase 2 Study to Evaluate the Safety and Efficacy of ABP-450 in the Treatment of Cervical Dystonia

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

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.

Study Overview

• Status: Completed
• Conditions: Cervical Dystonia
• Intervention / Treatment: Drug: ABP-450; Drug: Placebo

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.

• Study Type: Interventional
• Enrollment (Actual): 61
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Phoenix, Arizona, United States, 85032
      • Arizona Neuroscience Research
    • Scottsdale, Arizona, United States, 85258
      • Movement Disorder Center of Arizona
  • California
    • Fountain Valley, California, United States, 92708
      • Parkinson's and Movement Disorder Institute
    • Fresno, California, United States, 93710
      • Neuro Pain Medical Center
    • Loma Linda, California, United States, 92354
      • Loma Linda University
  • Connecticut
    • Stamford, Connecticut, United States, 06905
      • New England Institute for Neurology and Headache
  • Florida
    • Hollywood, Florida, United States, 33024
      • Infinity Clinical Research LLC
    • Miami, Florida, United States, 33176
      • The Neurology Research Group
    • Tampa, Florida, United States, 33613
      • University of South Florida
    • Winter Park, Florida, United States, 32792
      • Neurology One
  • Georgia
    • Atlanta, Georgia, United States, 30329
      • Emory University
  • Illinois
    • Chicago, Illinois, United States, 60612
      • Rush University Medical Center
  • Michigan
    • East Lansing, Michigan, United States, 48824
      • Michigan State University
    • Farmington Hills, Michigan, United States, 48334
      • Quest Research Institute - Hunt - PPDS
  • New Mexico
    • Albuquerque, New Mexico, United States, 87106
      • University of New Mexico
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • The Cleveland Clinic Foundation
    • Cleveland, Ohio, United States, 89106
      • Cleveland Clinic Lou Ruvo Center for Brain Health
    • New Albany, Ohio, United States, 43054
      • The Orthopedic Foundation
  • Tennessee
    • Memphis, Tennessee, United States, 38157
      • Veracity Neuroscience LLC
    • Nashville, Tennessee, United States, 37232
      • Vanderbilt University Medical Center
  • Texas
    • Houston, Texas, United States, 77030
      • Baylor College of Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: ABP-450 - Low Dose; ABP-450 Low Dose - Intramuscular injections into affected neck muscles.	Drug: ABP-450; ABP-450 (prabotulinumtoxinA) contains a 900kDA botulinum toxin type-A complex produced by the bacterium Clostridium botulinum.; Other Names: prabotulinumtoxinA
Experimental: ABP-450 - Medium Dose; ABP-450 Mid Dose - Intramuscular injections into affected neck muscles.	Drug: ABP-450; ABP-450 (prabotulinumtoxinA) contains a 900kDA botulinum toxin type-A complex produced by the bacterium Clostridium botulinum.; Other Names: prabotulinumtoxinA
Experimental: ABP-450 - High Dose; ABP-450 High Dose - Intramuscular injections into affected neck muscles.	Drug: ABP-450; ABP-450 (prabotulinumtoxinA) contains a 900kDA botulinum toxin type-A complex produced by the bacterium Clostridium botulinum.; Other Names: prabotulinumtoxinA
Placebo Comparator: Placebo; Placebo (0.9% saline, sterile, unpreserved, USP/Ph.Eur.) - Intramuscular injections into affected neck muscles.	Drug: Placebo; 0.9% sodium chloride, sterile, unpreserved, USP/Ph.Eur.; Other Names: saline; 0.9% sodium chloride

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Treatment-Related Serious Adverse Events	The primary safety endpoint will be the number of participants with treatment-related serious adverse events since the start of treatment by Treatment Group when dosed with placebo, ABP-450 (low dose), ABP-450 (medium d dose), or ABP-450 (high dose).	Baseline up to 20 Weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mean Change of the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS)-Total Score From Baseline to Week 4	The Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) scale is used to assess the severity of cervical Dystonia. The TWSTRS-total score has a minimum score of 0 and a maximum score of 85, where higher scores represent worse outcomes. It comprises 3 subscales, which are summated to get the total score: the Torticollis Severity Scale (minimum score of 0, maximum score of 35), the Disability Scale (minimum score of 0, maximum score of 30), and the Pain Scale (minimum score of 0, maximum score of 20).	Baseline to Week 4
Mean Change in the Subscale Score of Disability of the Baseline Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) From Baseline to Week 4	The Disability subscale of the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) is used to assess the severity of disability in cervical dystonia. The disability subscale has a minimum score of 0 and maximum score of 30, where higher scores represent worse outcomes.	Baseline to Week 4
Mean Change in the Subscale Score of Pain of the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) From Baseline to Week 4	The Pain subscale of the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) is used to assess the severity of pain in cervical dystonia. The pain subscale has a minimum score of 0 and maximum score of 20, where higher scores represent worse outcomes.	Baseline to Week 4
Mean Change in the Subscale Score of Severity of the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) From Baseline to Week 4	The Severity subscale of the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) is used to assess the severity of torticollis in cervical dystonia. The severity subscale has a minimum score of 0 and maximum score of 35, where higher scores represent worse outcomes.	Baseline to Week 4
Mean Change in Patient Global Impression of Change (PGI-C) From Baseline to Week 4	The PGI-C enables the patient to rate changes in their perception of their general health status over the duration of the assessment via a 7-point scale ranging from "much improved" to "much worse". The 7-point scale range is much improved (+3) to much worse (-3) with no change at "0". The mean change in the subject's assessment of the change in clinical status since the start of treatment measured by the Patients' Global Impression of Change (PGI-C) Scale was assessed by Treatment Group at Week 4.	Baseline to Week 4
Mean Change in Clinical Global Impression of Change (CGI-C) From Baseline to Week 4	The CGI-C enables the patient to rate changes in their perception of their general health status over the duration of the assessment via a 7-point scale ranging from "much improved" to "much worse". The 7-point scale range is much improved (+3) to much worse (-3) with no change at "0". The mean change from Baseline in the Clinical Global Impression of Change (CGI-C) Score was assessed by Treatment Group at Week 4.	Baseline to Week 4

Collaborators and Investigators

• Sponsor: AEON Biopharma, Inc.
• Collaborators: PPD
• Investigators: Principal Investigator: Joseph Jankovic, Baylor St. Luke's Medical Center; Principal Investigator: Cynthia Comella, MD, Rush University Medical Center

Study record dates

Study Major Dates

• Study Start (Actual): March 29, 2021
• Primary Completion (Actual): July 11, 2022
• Study Completion (Actual): July 11, 2022

Study Registration Dates

• First Submitted: April 14, 2021
• First Submitted That Met QC Criteria: April 14, 2021
• First Posted (Actual): April 20, 2021

Study Record Updates

• Last Update Posted (Estimated): February 14, 2024
• Last Update Submitted That Met QC Criteria: January 23, 2024
• Last Verified: January 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Movement Disorders; Dyskinesias; Dystonia; Dystonic Disorders; Torticollis
• Other Study ID Numbers: ABP-19000

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Individual Participant Data collected during the trial, after deidentification may be shared following review of the clinical study report by the FDA review division and if a decision is made to publish the results in a publication outside posting the results in clinicaltrials.gov.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No