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

Phase 2

Active, not recruiting

• Conditions: Inclusion Body Myositis
• Interventions: Drug: ABC008

• Registration Number: NCT05721573
• Lead Sponsor: Abcuro, Inc.

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

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: 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.

Study Timeline

• Study First Submitted: 2023-02-01
• Study First Submitted QC: 2023-02-01
• Study First Posted: 2023-02-10
• Study Start: 2023-02-28
• Status Verified: 2024-04
• Last Update Submitted: 2024-04-04
• Last Update Posted: 2024-04-05
• Primary Completion: 2025-11
• Study Completion: 2025-12

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 231

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;

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	ABC008	Part A - Placebo N= 6 Part B - Placebo N= 67
0.5 mg/kg ABC008	ABC008	Part A - ABC008 N=12 Part B - ABC008 N= 67
2.0 mg/kg ABC008	ABC008	Part A - ABC008 N=12 Part B - ABC008 N= 67

Primary Outcome Measures

Name	Time	Method
Part B - To determine the efficacy of ABC008 in IBM at two SC dose levels as measured by IBM Functional Rating Scale (IBMFRS) at Week (W)76	From Baseline (week 0) through study completion, an average of 76 weeks	Mean change in IBM Functional Rating Scale (IBMFRS)
Part A - To determine the safety and tolerability of recurrent dosing of ABC008 in subjects with IBM at 2 SC dose levels.	From Baseline (week 0) through week 20.	Safety as assessed by the incidence, type and severity of Treatment Emergent Adverse Events (TEAEs)

Secondary Outcome Measures

Name	Time	Method
Part A - Clinically significant changes in standard laboratory parameters, vital signs, and ECGs	From Baseline (Day 1) through study completion, an average of 80 weeks.	Incidence of clinically significant changes in standard laboratory parameters, vital signs, and ECGs
Part B - Modified Timed Up and Go (mTUG)	From Baseline (Day 1) through study completion, an average of 76 weeks.	Mean change in mTUG.
Part B - Manual Muscle Test 12 (MMT 12)	From Baseline (Day 1) through study completion, an average of 76 weeks.	Mean change in MMT 12
Part A - Treatment Emergent Serious Adverse Events (TEASAEs)	From Baseline (Day 1) through study completion, an average of 80 weeks.	Incidence, type and severity of TEASAEs.
Part A - Treatment Emergent Adverse Events (TEAEs) onset within 24 hours of Study Medication Administration.	From Baseline (Day 1) through study completion, an average of 80 weeks.	Incidence, type, and severity of TEAEs with onset within 24 hours from the start of any of study medication administration
Part A - Treatment Emergent Adverse Events leading to study medication or study discontinuation.	From Baseline (Day 1) through study completion, an average of 80 weeks.	Incidence of TEAEs leading to study medication or study discontinuation
Part B - Hand Grip Dynamometry	From Baseline (Day 1) through study completion, an average of 76 weeks.	Mean change in hand grip strength by dynamometry.
Part B - Quadriceps Dynamometry	From Baseline (Day 1) through study completion, an average of 76 weeks.	Mean change in quadriceps strength by dynamometry.
Part A - Adverse Events of Special Interest (AESI)	From Baseline (Day 1) through study completion, an average of 80 weeks.	Incidence of AESIs.

Trial Locations

Locations (44)

UCLA Medical Center; 🇺🇸 Los Angeles, California, United States

Johns Hopkins Bayview Medical Center; 🇺🇸 Baltimore, Maryland, United States

University of Pennsylvania; 🇺🇸 Philadelphia, Pennsylvania, United States

UPMC Arthritis and Autoimmunity Center, Falk Clinic; 🇺🇸 Pittsburgh, Pennsylvania, United States

University Hospitals Cleveland Medical Center; 🇺🇸 Cleveland, Ohio, United States

Perron Institute for Neurological and Translational Science; 🇦🇺 Nedlands, Western Australia, Australia

Keck Hosptial of USC; 🇺🇸 Los Angeles, California, United States

Hospital for Special Surgery; 🇺🇸 New York, New York, United States

Hospital Pitie-Salpetriere - AP-HP; 🇫🇷 Paris, France

Penn State Health Milton S. Hershey Medical Center; 🇺🇸 Hershey, Pennsylvania, United States

Nerve and Muscle Center of Texas; 🇺🇸 Houston, Texas, United States

University Hosptial Duesseldorf; 🇩🇪 Düsseldorf, Germany

Krankenhaus und Poliklinik Rüdersdorf GmbH; 🇩🇪 Berlin, Germany

Salford Royal Hospital, Northern Care Alliance NHS Foundation Trust; 🇬🇧 Salford, United Kingdom

Neuromuscular Research Center; 🇺🇸 Phoenix, Arizona, United States

Neuromuscular Diagnostic Center - Massachusetts General Hospital; 🇺🇸 Boston, Massachusetts, United States

Mayo Clinic; 🇺🇸 Rochester, Minnesota, United States

University of Washington Medical Center - Montlake; 🇺🇸 Seattle, Washington, United States

Medical College of Wisconsin; 🇺🇸 Milwaukee, Wisconsin, United States

Duke Neurological Disorders Clinic -1L; 🇺🇸 Durham, North Carolina, United States

Brigham and Womens Hospital; 🇺🇸 Boston, Massachusetts, United States

University of Nebraska Medical Center; 🇺🇸 Omaha, Nebraska, United States

Oregon Health & Science University; 🇺🇸 Portland, Oregon, United States

University of California Irvine Medical Center (UCIMC) - Amyotrophic Lateral Sclerosis (ALS) and Neuromuscular Center; 🇺🇸 Irvine, California, United States

Stanford Neuroscience Medical Center; 🇺🇸 Palo Alto, California, United States

University of Kansas Medical; 🇺🇸 Kansas City, Kansas, United States

Northwestern Memorial Hospital, Department of Neurology (Clinic); 🇺🇸 Chicago, Illinois, United States

Washington University School of Medicine; 🇺🇸 Saint Louis, Missouri, United States

Columbia University Medical Center / The Neurological Institute of New York; 🇺🇸 New York, New York, United States

Texas Neurology; 🇺🇸 Dallas, Texas, United States

Royal North Shore Hospital; 🇦🇺 Saint Leonards, New South Wales, Australia

AZ Sint-Lucas & Volkskliniek; 🇧🇪 Gent, Belgium

Heritage Medical Research Clinic - University of Calgary; 🇨🇦 Calgary, Alberta, Canada

Genge Partners Inc.; 🇨🇦 Montréal, Quebec, Canada

Austin Neuromuscular Center; 🇺🇸 Austin, Texas, United States

Royal Brisbane and Women's Hospital; 🇦🇺 Herston, Queensland, Australia

University College London Hospitals NHS Foundation Trust, National Hospital for Neurology and Neurosurgery (NHNN); 🇬🇧 London, United Kingdom

The Ohio State University Wexner Medical Center; 🇺🇸 Columbus, Ohio, United States

Virginia Commonwealth University; 🇺🇸 Henrico, Virginia, United States

Johns Hopkins University School of Medicine; 🇺🇸 Baltimore, Maryland, United States

University of California, San Francisco; 🇺🇸 San Francisco, California, United States

University of Colorado Hospital Anschutz Outpatient Pavillion; 🇺🇸 Aurora, Colorado, United States

Yale School of Medicine; 🇺🇸 New Haven, Connecticut, United States

Wake Forrest School of Medicine; 🇺🇸 Winston-Salem, North Carolina, United States