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

Cervical Dystonia Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04849988
• Other study ID #: ABP-19000
• Status: Completed
• Phase: Phase 2
• Start date: March 29, 2021
• Est. completion date: July 11, 2022

Study information

• Verified date: January 2024
• Source: AEON Biopharma, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 61
• Est. completion date: July 11, 2022
• Est. primary completion date: July 11, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 75 Years

Eligibility

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

Related Conditions & MeSH terms

• Cervical Dystonia
• Dystonia
• Dystonic Disorders
• Torticollis

Intervention

Drug:
• ABP-450
ABP-450 (prabotulinumtoxinA) contains a 900kDA botulinum toxin type-A complex produced by the bacterium Clostridium botulinum.
• Placebo
0.9% sodium chloride, sterile, unpreserved, USP/Ph.Eur.

Locations

Country	Name	City	State
United States	University of New Mexico	Albuquerque	New Mexico
United States	Emory University	Atlanta	Georgia
United States	Rush University Medical Center	Chicago	Illinois
United States	Cleveland Clinic Lou Ruvo Center for Brain Health	Cleveland	Ohio
United States	The Cleveland Clinic Foundation	Cleveland	Ohio
United States	Michigan State University	East Lansing	Michigan
United States	Quest Research Institute - Hunt - PPDS	Farmington Hills	Michigan
United States	Parkinson's and Movement Disorder Institute	Fountain Valley	California
United States	Neuro Pain Medical Center	Fresno	California
United States	Infinity Clinical Research LLC	Hollywood	Florida
United States	Baylor College of Medicine	Houston	Texas
United States	Loma Linda University	Loma Linda	California
United States	Veracity Neuroscience LLC	Memphis	Tennessee
United States	The Neurology Research Group	Miami	Florida
United States	Vanderbilt University Medical Center	Nashville	Tennessee
United States	The Orthopedic Foundation	New Albany	Ohio
United States	Arizona Neuroscience Research	Phoenix	Arizona
United States	Movement Disorder Center of Arizona	Scottsdale	Arizona
United States	New England Institute for Neurology and Headache	Stamford	Connecticut
United States	University of South Florida	Tampa	Florida
United States	Neurology One	Winter Park	Florida

Sponsors (2)

Lead Sponsor	Collaborator
AEON Biopharma, Inc.	PPD

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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