A Study to Evaluate the Efficacy and Safety of ABC008 for Inclusion Body Myositis

NCT ID: NCT05721573

Last Updated: 2025-12-17

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2/PHASE3

Total Enrollment

231 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-02-28

Study Completion Date

2026-01-31

Brief Summary

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A Phase II/III Randomized, Double-blind, Placebo-controlled, Multicenter Study to Determine the Efficacy and Safety of ABC008 in the Treatment of Subjects with Inclusion Body Myositis

Detailed Description

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A Phase II/III Randomized, Double-blind, Placebo-controlled, Multicenter Study to Determine the Efficacy and Safety of ABC008 in the Treatment of Subjects with Inclusion Body Myositis Detailed Description: A Phase II/III Randomized, Double-blind, Placebo-controlled, Multicenter Study to Determine the Efficacy and Safety of ABC008 in the Treatment of Subjects with Inclusion Body Myositis Detailed Description: This is a Phase II/III randomized, double-blind, placebo-controlled, parallel multicenter study with 3 parts.

The study will include a sentinel cohort (Part A) of 30 subjects who will receive first three doses of the study drug. Safety data from subjects in the sentinel cohorts will be evaluated by a Data and Safety Monitoring Board (DSMB) before further dosing of the sentinel cohort, as well as initiation of enrollment in the double-blind safety and efficacy cohort (Part B). After completion of Part A or Part B, subjects have the option of enrolling in an open-label long-term extension study or progressing to the pharmacodynamics (PD) recovery cohort (Part C), to evaluate the recovery of the depletion of killer cell lectin-like receptor G1 (KLRG1)+ cells after the end of treatment with ABC008.

Efficacy, safety, HRQoL, and HRU assessments will be conducted. Blood samples will be obtained to evaluate the serum PK, PD, and immunogenicity of ABC008 throughout the study.

Conditions

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Inclusion Body Myositis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors
Double Blind

Study Groups

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0.5 mg/kg ABC008

Part A - ABC008 N=12

Part B - ABC008 N= 67

Group Type ACTIVE_COMPARATOR

ABC008

Intervention Type DRUG

Given by subcutaneous injection

2.0 mg/kg ABC008

Part A - ABC008 N=12

Part B - ABC008 N= 67

Group Type ACTIVE_COMPARATOR

ABC008

Intervention Type DRUG

Given by subcutaneous injection

Placebo

Part A - Placebo N= 6

Part B - Placebo N= 67

Group Type PLACEBO_COMPARATOR

ABC008

Intervention Type DRUG

Given by subcutaneous injection

Interventions

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ABC008

Given by subcutaneous injection

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Adult males and females age \>40 years at the time of the first dose of study medication;
* Weight \>40 and \<150 kg;
* Diagnosis of either clinico-pathologically defined IBM, clinically defined IBM, or probable IBM according to the European Neuromuscular Centre (ENMC) IBM 2011 research diagnostic criteria (Rose et al., 2013). Documented histopathology results must be available prior to Baseline (Day 1) to confirm eligibility;
* Able to arise from a chair (with armrests), with use of their arms but without support from another person or device (e.g., cane, walking stick), at Screening and Baseline (Day 1);
* Able to walk 3 meters, turn around, walk back to the chair, and sit down, with or without assistive device. Once arisen from the chair, subject may use any walking device but cannot be supported by another person, furniture, or a wall;

Exclusion Criteria

* Any other form of myositis or myopathy other than IBM, e.g., metabolic or drug-induced myopathy, drug-induced myositis, anti-synthetase syndrome, polymyositis or dermatomyositis, cancer-associated myositis (myositis diagnosed within 3 years, either before or after), myositis in overlap with another autoimmune disease (e.g., systemic lupus, systemic sclerosis, rheumatoid arthritis), or muscular dystrophy;
* Any condition, e.g., severe degenerative arthritis with limited range of motion, which precludes the ability to quantitate muscle strength or perform functional assessments (e.g., mTUG), in the Investigator's opinion;.
* Presence of another autoimmune or autoinflammatory disease other than indication under study, e.g., rheumatoid arthritis, psoriatic arthritis, axial spondyloarthropathy, inflammatory bowel disease, systemic lupus erythematosus. Subjects with Sjogren's syndrome, T-cell large granular lymphocyte leukemia (T-LGLL), or well-controlled thyroid disease are permitted;
Minimum Eligible Age

40 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Syneos Health

OTHER

Sponsor Role collaborator

Abcuro, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Neuromuscular Research Center

Phoenix, Arizona, United States

Site Status

University of California Irvine Medical Center (UCIMC) - Amyotrophic Lateral Sclerosis (ALS) and Neuromuscular Center

Irvine, California, United States

Site Status

Keck Hosptial of USC

Los Angeles, California, United States

Site Status

UCLA Medical Center

Los Angeles, California, United States

Site Status

Stanford Neuroscience Medical Center

Palo Alto, California, United States

Site Status

University of California, San Francisco

San Francisco, California, United States

Site Status

University of Colorado Hospital Anschutz Outpatient Pavillion

Aurora, Colorado, United States

Site Status

Yale School of Medicine

New Haven, Connecticut, United States

Site Status

Mayo Clinic

Jacksonville, Florida, United States

Site Status

Northwestern Memorial Hospital, Department of Neurology (Clinic)

Chicago, Illinois, United States

Site Status

University of Kansas Medical

Kansas City, Kansas, United States

Site Status

Johns Hopkins Bayview Medical Center

Baltimore, Maryland, United States

Site Status

Johns Hopkins University School of Medicine

Baltimore, Maryland, United States

Site Status

Neuromuscular Diagnostic Center - Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Brigham and Womens Hospital

Boston, Massachusetts, United States

Site Status

Mayo Clinic

Rochester, Minnesota, United States

Site Status

Washington University School of Medicine

St Louis, Missouri, United States

Site Status

University of Nebraska Medical Center

Omaha, Nebraska, United States

Site Status

Hospital for Special Surgery

New York, New York, United States

Site Status

Columbia University Medical Center / The Neurological Institute of New York

New York, New York, United States

Site Status

Duke Neurological Disorders Clinic -1L

Durham, North Carolina, United States

Site Status

Wake Forrest School of Medicine

Winston-Salem, North Carolina, United States

Site Status

University Hospitals Cleveland Medical Center

Cleveland, Ohio, United States

Site Status

The Ohio State University Wexner Medical Center

Columbus, Ohio, United States

Site Status

Oregon Health & Science University

Portland, Oregon, United States

Site Status

Penn State Health Milton S. Hershey Medical Center

Hershey, Pennsylvania, United States

Site Status

University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status

UPMC Arthritis and Autoimmunity Center, Falk Clinic

Pittsburgh, Pennsylvania, United States

Site Status

Austin Neuromuscular Center

Austin, Texas, United States

Site Status

Texas Neurology

Dallas, Texas, United States

Site Status

Nerve and Muscle Center of Texas

Houston, Texas, United States

Site Status

Virginia Commonwealth University

Henrico, Virginia, United States

Site Status

University of Washington Medical Center - Montlake

Seattle, Washington, United States

Site Status

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

Royal North Shore Hospital

Saint Leonards, New South Wales, Australia

Site Status

Royal Brisbane and Women's Hospital

Herston, Queensland, Australia

Site Status

Perron Institute for Neurological and Translational Science

Nedlands, Western Australia, Australia

Site Status

AZ Sint-Lucas & Volkskliniek

Ghent, , Belgium

Site Status

Heritage Medical Research Clinic - University of Calgary

Calgary, Alberta, Canada

Site Status

Genge Partners Inc.

Montreal, Quebec, Canada

Site Status

Hospital Pitie-Salpetriere - AP-HP

Paris, , France

Site Status

Krankenhaus und Poliklinik Rüdersdorf GmbH

Berlin, , Germany

Site Status

University Hosptial Duesseldorf

Düsseldorf, , Germany

Site Status

University College London Hospitals NHS Foundation Trust, National Hospital for Neurology and Neurosurgery (NHNN)

London, , United Kingdom

Site Status

Salford Royal Hospital, Northern Care Alliance NHS Foundation Trust

Salford, , United Kingdom

Site Status

Countries

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United States Australia Belgium Canada France Germany United Kingdom

Related Links

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http://abcuro.com

Study Website

Other Identifiers

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ABC008-IBM-201

Identifier Type: -

Identifier Source: org_study_id