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

NCT05721573 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: ABC008-IBM-201
• Study Type: interventional
• Target: 272
• Locations: 7 countries
• Sites: 45

Brief Summary

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

Conditions

Inclusion Body Myositis

Keywords

Muscular Diseases; Inflammatory Myopathy; Myositis; Neuromuscular Diseases; Nervous System Disease

Outcome Measures

Primary Outcomes (9)

• Part A: Number of Participants With Treatment Emergent Adverse Events (TEAEs)

  An adverse event (AE) was defined as any untoward medical occurrence in any participant participating in a clinical investigation, regardless of the suspected cause. An AE was therefore any unfavorable and unintended sign, symptom, disease, concurrent illness, or clinically significant abnormal laboratory finding that emerged or worsened during the study. A TEAE was defined as an AE that first occurred or worsened after initiation of study drug.

  Up to Week 80

• Part A: Number of Participants With TEAEs by Severity

  A TEAE was defined as an AE that first occurred or worsened after initiation of study drug and was graded by severity per protocol-specified Common Terminology Criteria for Adverse Events (CTCAE) criteria: Grade 1, Mild (asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated); Grade 2, Moderate (minimal, local, or noninvasive intervention indicated; limited age-appropriate instrumental activities of daily living (ADL)); Grade 3, Severe or medically significant (medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; or limiting self-care ADL); Grade 4, Life-threatening (threatening consequences; urgent intervention indicated); and Grade 5, Death (death related to an AE).

  Up to Week 80

• Part A: Number of Participants With TEAEs Related to Study Drug

  An AE was defined as any untoward medical occurrence in any participant participating in a clinical investigation, regardless of the suspected cause. An AE was therefore can be any unfavorable and unintended sign, symptom, disease, concurrent illness, or clinically significant abnormal laboratory finding that emerged or worsened during the study. A TEAE was defined as an AE that first occurred or worsened after initiation of study drug. An AE with missing relationship to study drug was considered as related to study drug.

  Up to Week 80

• Part A: Mean Change From Baseline in the Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score at Week 76

  The IBMFRS was used as an ordinal rating scale to determine participant's assessments of their capabilities. It included 10 measures (swallowing, handwriting, cutting food and handling utensils, fine motor tasks, dressing, hygiene, turning in bed and adjusting covers, changing position from sitting to standing, walking, and climbing stairs) graded on a Likert scale (a psychometric tool) from 0 (being unable to perform) to 4 (normal). The sum of the 10 items provided a value between 0 and 40, with a higher score representing less functional limitation or better functional status. Baseline value was defined as the last non-missing valid value prior to first administration of study treatment (on Day 1). Change from Baseline was calculated as the data collected at post-randomization visits minus the Baseline.

  Baseline (Day 1) and at Week 76

• Part B: Number of Participants With TEAEs

  An AE was defined as any untoward medical occurrence in any participant participating in a clinical investigation, regardless of the suspected cause. An AE was therefore can be any unfavorable and unintended sign, symptom, disease, concurrent illness, or clinically significant abnormal laboratory finding that emerged or worsened during the study. A TEAE was defined as an AE that first occurred or worsened after initiation of study drug.

  Day 1 to Week 80

• Part B: Number of Participants With TEAEs by Severity

  A TEAE was defined as an AE that first occurred or worsened after initiation of study drug and was graded by severity per protocol-specified CTCAE criteria: Grade 1, Mild (asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated); Grade 2, Moderate (minimal, local, or noninvasive intervention indicated; limited age-appropriate instrumental ADL); Grade 3, Severe or medically significant (medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; or limiting self-care ADL); Grade 4, Life-threatening (threatening consequences; urgent intervention indicated); and Grade 5, Death (death related to an AE).

  Day 1 to Week 80

• Part B: Number of Participants With TEAEs Related to Study Drug

  An AE was defined as any untoward medical occurrence in any participant participating in a clinical investigation, regardless of the suspected cause. An AE was therefore can be any unfavorable and unintended sign, symptom, disease, concurrent illness, or clinically significant abnormal laboratory finding that emerged or worsened during the study. A TEAE was defined as an AE that first occurred or worsened after initiation of study drug. An AE with missing relationship to study drug was considered as related to study drug.

  Day 1 to Week 80

• Part B: Mean Change From Baseline in the IBMFRS Total Score at Week 76

  The IBMFRS was used as an ordinal rating scale to determine participant's assessments of their capabilities. It included 10 measures (swallowing, handwriting, cutting food and handling utensils, fine motor tasks, dressing, hygiene, turning in bed and adjusting covers, changing position from sitting to standing, walking, and climbing stairs) graded on a Likert scale (a psychometric tool) from 0 (being unable to perform) to 4 (normal). The sum of the 10 items provided a value between 0 and 40, with a higher score representing less functional limitation or better functional status. Baseline value was defined as the last non-missing valid value prior to first administration of study treatment (on Day 1). Change from Baseline was calculated as the data collected at post-randomization visits minus the Baseline.

  Baseline (Day 1) and Up to Week 76

• Part C: Time to PD Recovery

  Recovery was defined as the return of KLRG1+ cell counts toward baseline levels during the PD follow-up period.

  Up to Week 24

Secondary Outcomes (27)

• Part A and B: Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs)

  Up to Week 80

• Part A and B: Number of Participants With TEAEs With Onset Within 24 Hours From the Start of Any Study Medication Administration

  Within 24 hours following each dose, Up to Week 80

• Part A and B: Number of Participants With TEAEs Leading to Study Discontinuation

  Up to Week 80

• Part A and B: Change From Baseline in Hematology Parameters - Absolute Neutrophil Count, Basophils, Eosinophils, Leukocytes, Lymphocytes, Monocytes, Neutrophils, and Platelets

  Baseline (Day 1) to Week 76

• Part A and B: Change From Baseline in Hematology Parameter - Erythrocytes

  Baseline (Day 1) to Week 76

Study Arms (3)

0.5 mg/kg ABC008

ACTIVE COMPARATOR

Part A - ABC008 N=12 Part B - ABC008 N= 67

Drug: ABC008

2.0 mg/kg ABC008

ACTIVE COMPARATOR

Part A - ABC008 N=12 Part B - ABC008 N= 67

Drug: ABC008

Placebo

PLACEBO COMPARATOR

Part A - Placebo N= 6 Part B - Placebo N= 67

Drug: ABC008

Interventions

ABC008 DRUG

Given by subcutaneous injection

0.5 mg/kg ABC008; 2.0 mg/kg ABC008; Placebo

Eligibility Criteria

• Age: 40 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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;

You may not qualify if:

• 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;

Sponsors & Collaborators

• Abcuro, Inc.: lead
• Syneos Health: collaborator

Study Sites (45)

Neuromuscular Research Center

Phoenix, Arizona, 85028, United States

Location

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

Irvine, California, 92868, United States

Location

Keck Hosptial of USC

Los Angeles, California, 90033, United States

Location

UCLA Medical Center

Los Angeles, California, 90095, United States

Location

Stanford Neuroscience Medical Center

Palo Alto, California, 94304, United States

Location

University of California, San Francisco

San Francisco, California, 94143, United States

Location

University of Colorado Hospital Anschutz Outpatient Pavillion

Aurora, Colorado, 80045, United States

Location

Yale School of Medicine

New Haven, Connecticut, 06519, United States

Location

Mayo Clinic

Jacksonville, Florida, 32224, United States

Location

Northwestern Memorial Hospital, Department of Neurology (Clinic)

Chicago, Illinois, 60611, United States

Location

University of Kansas Medical

Kansas City, Kansas, 66160, United States

Location

Johns Hopkins Bayview Medical Center

Baltimore, Maryland, 21224, United States

Location

Johns Hopkins University School of Medicine

Baltimore, Maryland, 21287, United States

Location

Neuromuscular Diagnostic Center - Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Brigham and Womens Hospital

Boston, Massachusetts, 021158, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

University of Nebraska Medical Center

Omaha, Nebraska, 98198, United States

Location

Hospital for Special Surgery

New York, New York, 10021, United States

Location

Columbia University Medical Center / The Neurological Institute of New York

New York, New York, 10032, United States

Location

Duke Neurological Disorders Clinic -1L

Durham, North Carolina, 27710, United States

Location

Wake Forrest School of Medicine

Winston-Salem, North Carolina, 27157, United States

Location

University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106, United States

Location

The Ohio State University Wexner Medical Center

Columbus, Ohio, 43210, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

Penn State Health Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

UPMC Arthritis and Autoimmunity Center, Falk Clinic

Pittsburgh, Pennsylvania, 15213, United States

Location

Austin Neuromuscular Center

Austin, Texas, 78759, United States

Location

Texas Neurology

Dallas, Texas, 75206, United States

Location

Nerve and Muscle Center of Texas

Houston, Texas, 77030, United States

Location

Virginia Commonwealth University

Henrico, Virginia, 23233, United States

Location

University of Washington Medical Center - Montlake

Seattle, Washington, 98195, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Royal North Shore Hospital

Saint Leonards, New South Wales, 2065, Australia

Location

Royal Brisbane and Women's Hospital

Herston, Queensland, 4006, Australia

Location

Perron Institute for Neurological and Translational Science

Nedlands, Western Australia, 6009, Australia

Location

AZ Sint-Lucas & Volkskliniek

Ghent, 9000, Belgium

Location

Heritage Medical Research Clinic - University of Calgary

Calgary, Alberta, 3M 1M4, Canada

Location

Genge Partners Inc.

Montreal, Quebec, H4A 3T2, Canada

Location

Hospital Pitie-Salpetriere - AP-HP

Paris, 75013, France

Location

Krankenhaus und Poliklinik Rüdersdorf GmbH

Berlin, 15562, Germany

Location

University Hosptial Duesseldorf

Düsseldorf, 40225, Germany

Location

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

London, WC1N 3BG, United Kingdom

Location

Salford Royal Hospital, Northern Care Alliance NHS Foundation Trust

Salford, M6 8HD, United Kingdom

Location

Related Links

• Study Website

MeSH Terms

Conditions

Myositis, Inclusion Body; Muscular Diseases; Myositis; Neuromuscular Diseases; Nervous System Diseases

Condition Hierarchy (Ancestors)

Musculoskeletal Diseases

Results Point of Contact

• Title: Study Director
• Organization: Abcuro, Inc.

ibm201clinicaltrial@abcuro.com

617-865-5079

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Double Blind
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 1, 2023
• First Posted: February 10, 2023
• Study Start: February 28, 2023
• Primary Completion: November 6, 2025
• Study Completion: November 27, 2025
• Last Updated: October 1, 2026
• Results First Posted: October 1, 2026

Record last verified: 2026-09

Data Sharing

• IPD Sharing: Will not share

Locations

AU (3); US (34); GB (2); CA (2); DE (2); BE (1); FR (1)