Extension Study of ABP-19000 to Evaluate Safety and Efficacy of Repeat Treatments of ABP-450 in Cervical Dystonia

An Open-Label, Multicenter Study to Evaluate the Safety and Efficacy of Repeat Intramuscular ABP-450 (prabotulinumtoxinA) Injection for the Treatment of Cervical Dystonia

This Open-label Extension 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 42 sites in the United States from Phase 2 (ABP-19000) and Phase 3 (ABP-19001) trials and 29 sites in Europe from Phase 3 (ABP-19001) trial. Study subjects who had their initial dose of study drug in Phase 2 or Phase 3 trial studies, irrespective of treatment allocation, will be eligible to enroll in this OLE study.

Study Overview

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

Detailed Description

This Open-label Extension 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 42 sites in the United States from Phase 2 (ABP-19000) and Phase 3 (ABP-19001) trials and 29 sites in Europe from Phase 3 (ABP-19001) trial. Study subjects who had their initial dose of study drug in Phase 2 or Phase 3 trial studies, irrespective of treatment allocation, will be eligible to enroll in this OLE study. Study subjects will receive a predetermined dose of ABP-450 between the Low Dose and High Dose, based on the investigator's discretion and clinical judgment.

• Study Type: Interventional
• Enrollment (Actual): 52
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Phoenix, Arizona, United States, 85032
      • Arizona Neuroscience Research
  • 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
      • Brainstorm Research
    • 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: 16 years to 73 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Qualified for and had their initial dose of study drug in the ABP-19000 or ABP-19001 studies and for whom 8 weeks have elapsed between last treatment in the double-blind study (ie, EOS visit) and first treatment in the ABP-19002 OLE study.
2. Provided written informed consent to being treated for cervical dystonia with ABP-450.
3. Were a male or female patient between 18 and 75 years of age (inclusive) when they entered the ABP-19000 or ABP-19001 studies.
4. Have clinical diagnosis of cervical dystonia and need for injection, as determined by the investigator, with TWSTRS total score ≥20.
5. Entered the ABP-19000 or ABP-19001 study on a stable dose of medications (if any) used for focal dystonia treatment (eg, anticholinergics and benzodiazepines) for ≥3months prior to and expected throughout the duration of the study.
6. 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 tele visits.

Exclusion Criteria:

1. Have traumatic torticollis or tardive torticollis.
2. Have predominant retrocollis or anterocollis.
3. Have hypersensitivity to human serum albumin, sucrose, or botulinum toxin type A.
4. Have diagnosis of myasthenia gravis, Lambert-Eaton syndrome, amyotrophic lateral sclerosis, or any other significant neuromuscular disease that might interfere with the study.
5. Have marked limitation on passive range of motion that suggests contractures or other structural abnormality (eg, cervical contractures or cervical spine syndrome).
6. Have 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.
7. Current swallowing disorder of any origin (dysphagia scale ≥3, ie, severe, with swallowing difficulties and requiring a change in diet).
8. Participated in another interventional study during participation in this study.
9. Were a pregnant or lactating female, or female of child-bearing potential not willing to use an acceptable method of contraception (ie, intrauterine device, barrier methods with spermicide, or abstinence).
10. Would not benefit from treatment with ABP-450 for their cervical dystonia, in the investigator's opinion.
11. Viral or other active infection or any medical condition that, in the opinion of the investigator, classifies the patient as unsuitable for participation in the study or patients who do not seem to be in good general health at the time of Day 0 "rollover", and prior to any investigational study drug administration.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: ABP-450 - Between Low Dose and High Dose; ABP-450 Between Low Dose and High Dose - Intramuscular injections into affected neck muscles with investigator's determined dose within the range of low dose and high dose - 4 injection cycles at 3-month intervals.	Drug: ABP-450; ABP-450 (prabotulinumtoxinA) contains a 900kDA botulinum toxin type-A complex produced by the bacterium Clostridium botulinum.; Other Names: prabotulinumtoxinA

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of Treatment-related Serious Adverse Events	The primary safety endpoint will be the incidence of treatment-related serious adverse events since the start of treatment by Treatment Group when dosed with ABP-450 between Low Dose and High Dose.	Up to 52 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mean Change in Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) Total Score	The mean change in the total score of the TWSTRS since the start of treatment will be assessed by treatment group. The TWSTRS total score is a summation of the following subscales: Severity scale, Disability scale and Pain scale of 0 - 85 with a higher score representing a worse outcome. with a higher score representing a worse outcome.	Up to 52 weeks
Mean Change in the subscale score of severity of the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS)	The mean change in the subscale score of severity of the TWSTRS since the start of treatment will be assessed by Treatment Group. This subscale has a scale of 0 - 35 with the higher score representing the greater severity.	Up to 52 weeks
Mean Change in the subscale score of disability of the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) Spasmodic Torticollis Rating Scale (TWSTRS)	The mean change in the subscale score of disability of the TWSTRS since the start of treatment will be assessed by Treatment Group. This subscale has a scale of 0 - 30 with the higher score represents the greater disability.	Up to 52 weeks
Mean Change in the subscale score of pain of the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS)	The mean change in the subscale score of pain of the TWSTRS since the start of treatment will be assessed by Treatment Group. This subscale has a scale of 0 - 20 with the higher score represents the greater disability.	Up to 52 weeks
Mean Change in Patient Global Impression of Change (PGI-C)	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 with a 1-item scale ranging from "much better" to "much worse" with the higher score indicating worsening of symptoms will be assessed by Treatment Group.	Up to 52 weeks
Mean Change in Patient Global Impression of Severity (PGI-S)	The mean change in the subject's assessment of the severity of their condition since the start of treatment measured by the Patients' Global Impression of Severity (PGI-S) Scale with a 1-item scale ranging from "normal" to "severely ill" with the higher score indicating greater severity in illness will be assessed by Treatment Group.	Up to 52 weeks
Mean Change in Clinical Global Impression of Change (CGI-C)	The mean change from Baseline in the Clinical Global Impression of Change (CGI-C) Score will be assessed by Treatment Group. CGI-C is a clinical assessment with a 7-point scale ranging from "very much improved" to "very much worse".	Up to 52 weeks
Mean Change in Clinical Global Impression of Severity (CGI-S)	The mean change from Baseline in the Clinical Global Impression of Severity (CGI-S) Score will be assessed by Treatment Group. CGI-S is a clinical assessment of the patient's illness severity on a 7-point scale ranging from "normal" to "among the most extremely ill patients".	Up to 52 weeks
Time from the First ABP-450 Dose to the First Visit Without Efficacy	The time from first ABP-450 dose to the first visit (from Week 2) without efficacy with regards to TWSTRS total score ≥20 will be captured.	Up to 12 Weeks

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Actual): July 27, 2021
• Primary Completion (Actual): July 24, 2023
• Study Completion (Actual): July 24, 2023

Study Registration Dates

• First Submitted: April 29, 2021
• First Submitted That Met QC Criteria: April 29, 2021
• First Posted (Actual): May 4, 2021

Study Record Updates

• Last Update Posted (Estimated): December 12, 2023
• Last Update Submitted That Met QC Criteria: December 8, 2023
• Last Verified: December 1, 2023

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-19002

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