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

Phase 2

Completed

• Conditions: Cervical Dystonia
• Interventions: Drug: ABP-450; Drug: Placebo

• Registration Number: NCT04849988
• Lead Sponsor: AEON Biopharma, Inc.

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.

Study Timeline

• Study Start: 2021-03-29
• Study First Submitted: 2021-04-14
• Study First Submitted QC: 2021-04-14
• Study First Posted: 2021-04-20
• Primary Completion: 2022-07-11
• Study Completion: 2022-07-11
• Results First Submitted: 2023-08-08
• Status Verified: 2024-01
• Results First Submitted QC: 2024-01-23
• Last Update Submitted: 2024-01-23
• Results First Posted: 2024-02-14
• Last Update Posted: 2024-02-14

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 61

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
ABP-450 - Low Dose	ABP-450	ABP-450 Low Dose - Intramuscular injections into affected neck muscles.
ABP-450 - Medium Dose	ABP-450	ABP-450 Mid Dose - Intramuscular injections into affected neck muscles.
ABP-450 - High Dose	ABP-450	ABP-450 High Dose - Intramuscular injections into affected neck muscles.
Placebo	Placebo	Placebo (0.9% saline, sterile, unpreserved, USP/Ph.Eur.) - Intramuscular injections into affected neck muscles.

Primary Outcome Measures

Name	Time	Method
Number of Participants With Treatment-Related Serious Adverse Events	Baseline up to 20 Weeks	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).

Secondary Outcome Measures

Name	Time	Method
Mean Change in Clinical Global Impression of Change (CGI-C) From Baseline to Week 4	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.
Mean Change of the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS)-Total Score From Baseline to Week 4	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).
Mean Change in the Subscale Score of Disability of the Baseline Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) From Baseline to Week 4	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.
Mean Change in the Subscale Score of Severity of the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) From Baseline to Week 4	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.
Mean Change in Patient Global Impression of Change (PGI-C) From Baseline to Week 4	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.
Mean Change in the Subscale Score of Pain of the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) From Baseline to Week 4	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.

Trial Locations

Locations (21)

Rush University Medical Center; 🇺🇸 Chicago, Illinois, United States

Neuro Pain Medical Center; 🇺🇸 Fresno, California, United States

Parkinson's and Movement Disorder Institute; 🇺🇸 Fountain Valley, California, United States

Neurology One; 🇺🇸 Winter Park, Florida, United States

Emory University; 🇺🇸 Atlanta, Georgia, United States

Quest Research Institute - Hunt - PPDS; 🇺🇸 Farmington Hills, Michigan, United States

Cleveland Clinic Lou Ruvo Center for Brain Health; 🇺🇸 Cleveland, Ohio, United States

Baylor College of Medicine; 🇺🇸 Houston, Texas, United States

The Neurology Research Group; 🇺🇸 Miami, Florida, United States

Michigan State University; 🇺🇸 East Lansing, Michigan, United States

The Cleveland Clinic Foundation; 🇺🇸 Cleveland, Ohio, United States

Vanderbilt University Medical Center; 🇺🇸 Nashville, Tennessee, United States

Arizona Neuroscience Research; 🇺🇸 Phoenix, Arizona, United States

New England Institute for Neurology and Headache; 🇺🇸 Stamford, Connecticut, United States

Movement Disorder Center of Arizona; 🇺🇸 Scottsdale, Arizona, United States

Loma Linda University; 🇺🇸 Loma Linda, California, United States

Infinity Clinical Research LLC; 🇺🇸 Hollywood, Florida, United States

The Orthopedic Foundation; 🇺🇸 New Albany, Ohio, United States

University of New Mexico; 🇺🇸 Albuquerque, New Mexico, United States

Veracity Neuroscience LLC; 🇺🇸 Memphis, Tennessee, United States

University of South Florida; 🇺🇸 Tampa, Florida, United States