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

NCT04849988 | Completed Phase 2 Results FDA Drug

• 1 other identifier: ABP-19000
• Study Type: interventional
• Target: 61
• Locations: 1 country
• Sites: 21

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.

Conditions

Cervical Dystonia

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (6)

• Mean Change of the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS)-Total Score From Baseline to Week 4

  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

  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

  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

  Baseline to Week 4

• Mean Change in Patient Global Impression of Change (PGI-C) From Baseline to Week 4

  Baseline to Week 4

Study Arms (4)

ABP-450 - Low Dose

EXPERIMENTAL

ABP-450 Low Dose - Intramuscular injections into affected neck muscles.

Drug: ABP-450

ABP-450 - Medium Dose

EXPERIMENTAL

ABP-450 Mid Dose - Intramuscular injections into affected neck muscles.

Drug: ABP-450

ABP-450 - High Dose

EXPERIMENTAL

ABP-450 High Dose - Intramuscular injections into affected neck muscles.

Drug: ABP-450

Placebo

PLACEBO COMPARATOR

Placebo (0.9% saline, sterile, unpreserved, USP/Ph.Eur.) - Intramuscular injections into affected neck muscles.

Drug: Placebo

Interventions

ABP-450 DRUG

ABP-450 (prabotulinumtoxinA) contains a 900kDA botulinum toxin type-A complex produced by the bacterium Clostridium botulinum.

Also known as: prabotulinumtoxinA

ABP-450 - High Dose; ABP-450 - Low Dose; ABP-450 - Medium Dose

Placebo DRUG

0.9% sodium chloride, sterile, unpreserved, USP/Ph.Eur.

Also known as: 0.9% sodium chloride, saline

Placebo

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female patients between 18 and 75 years of age (inclusive)
• 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
• 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
• For pre-treated patients only: Source documentation (eg, patient history) of the last 2 consecutive injection sessions with a botulinum toxin type A
• 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 \[for Day 0 injection\])
• Provided written informed consent to being treated for cervical dystonia with ABP-450
• 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

You may not qualify if:

• Traumatic torticollis or tardive torticollis
• Predominant retrocollis or anterocollis
• Myotomy or denervation surgery in the affected muscles (eg, peripheral denervation and/or spinal cord stimulation)
• Hypersensitivity to human serum albumin, sucrose, or botulinum toxin type A
• Previous treatment for cervical dystonia with rimabotulinumtoxin B
• Diagnosis of myasthenia gravis, Lambert-Eaton syndrome, amyotrophic lateral sclerosis, or any other significant neuromuscular disease that might interfere with the trial
• Current swallowing disorder of any origin (dysphagia scale ≥3, ie, severe, with swallowing difficulties and requiring a change in diet)
• Marked limitation on passive range of motion that suggests contractures or other structural abnormality, eg, cervical contractures or cervical spine syndrome
• Treatment with botulinum toxins of any type for any indication other than cervical dystonia within 16 weeks prior to baseline and during the study
• 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
• Participation in another interventional study during participation in this study
• 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)
• 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

Sponsors & Collaborators

• AEON Biopharma, Inc.: lead
• PPD Development, LP: collaborator

Study Sites (21)

Arizona Neuroscience Research

Phoenix, Arizona, 85032, United States

Location

Movement Disorder Center of Arizona

Scottsdale, Arizona, 85258, United States

Location

Parkinson's and Movement Disorder Institute

Fountain Valley, California, 92708, United States

Location

Neuro Pain Medical Center

Fresno, California, 93710, United States

Location

Loma Linda University

Loma Linda, California, 92354, United States

Location

New England Institute for Neurology and Headache

Stamford, Connecticut, 06905, United States

Location

Infinity Clinical Research LLC

Hollywood, Florida, 33024, United States

Location

The Neurology Research Group

Miami, Florida, 33176, United States

Location

University of South Florida

Tampa, Florida, 33613, United States

Location

Neurology One

Winter Park, Florida, 32792, United States

Location

Emory University

Atlanta, Georgia, 30329, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

Michigan State University

East Lansing, Michigan, 48824, United States

Location

Quest Research Institute - Hunt - PPDS

Farmington Hills, Michigan, 48334, United States

Location

University of New Mexico

Albuquerque, New Mexico, 87106, United States

Location

The Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

Location

Cleveland Clinic Lou Ruvo Center for Brain Health

Cleveland, Ohio, 89106, United States

Location

The Orthopedic Foundation

New Albany, Ohio, 43054, United States

Location

Veracity Neuroscience LLC

Memphis, Tennessee, 38157, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Torticollis

Interventions

prabotulinumtoxin A; Sodium Chloride

Condition Hierarchy (Ancestors)

Dystonia; Dyskinesias; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds

Results Point of Contact

• Title: Dana Testa, PharmD
• Organization: AEON Biopharma, Inc.

dt@aeonbiopharma.com

949-354-6499

Study Officials

• Cynthia Comella, MD

  Rush University Medical Center

  PRINCIPAL INVESTIGATOR

• Joseph Jankovic

  Baylor St. Luke's Medical Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: The investigator, study nurse/other study personnel, and patients will be blinded to the treatment group. An appropriately trained person will reconstitute investigational product, fill masked syringes and provide them to the blinded investigator, but will not perform any assessments with the patient.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: April 14, 2021
• First Posted: April 20, 2021
• Study Start: March 29, 2021
• Primary Completion: July 11, 2022
• Study Completion: July 11, 2022
• Last Updated: February 14, 2024
• Results First Posted: February 14, 2024

Record last verified: 2024-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (21)