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

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

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

Study Overview

• Status: Active, not recruiting
• Conditions: Inclusion Body Myositis
• Intervention / Treatment: Drug: ABC008

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 Type: Interventional
• Enrollment (Estimated): 231
• Phase: Phase 2; Phase 3

Contacts and Locations

• Study Contact: Name: Armando Reyes; Phone Number: 1 617 865 5079; Email: IBM-201_clinicaltrial@abcuro.com

Study Locations

• Australia
  • New South Wales
    • Saint Leonards, New South Wales, Australia, 2065
      • Royal North Shore Hospital
  • Queensland
    • Herston, Queensland, Australia, 4006
      • Royal Brisbane and Women'S Hospital
  • Western Australia
    • Nedlands, Western Australia, Australia, 6009
      • Perron Institute for Neurological and Translational Science
• Belgium
  • Gent, Belgium, 9000
    • AZ Sint-Lucas & Volkskliniek
• Canada
  • Alberta
    • Calgary, Alberta, Canada, 3M 1M4
      • Heritage Medical Research Clinic - University Of Calgary
  • Quebec
    • Montréal, Quebec, Canada, H4A 3T2
      • Genge Partners Inc.
• France
  • Paris, France, 75013
    • Hospital Pitie-Salpetriere - AP-HP
• Germany
  • Berlin, Germany, 15562
    • Krankenhaus und Poliklinik Rüdersdorf GmbH
  • Düsseldorf, Germany, 40225
    • University Hosptial Duesseldorf
• United Kingdom
  • London, United Kingdom, WC1N 3BG
    • University College London Hospitals NHS Foundation Trust, National Hospital for Neurology and Neurosurgery (NHNN)
  • Salford, United Kingdom, M6 8HD
    • Salford Royal Hospital, Northern Care Alliance NHS Foundation Trust
• United States
  • Arizona
    • Phoenix, Arizona, United States, 85028
      • Neuromuscular Research Center
  • California
    • Irvine, California, United States, 92868
      • University of California Irvine Medical Center (UCIMC) - Amyotrophic Lateral Sclerosis (ALS) and Neuromuscular Center
    • Los Angeles, California, United States, 90095
      • UCLA Medical Center
    • Los Angeles, California, United States, 90033
      • Keck Hosptial of USC
    • Palo Alto, California, United States, 94304
      • Stanford Neuroscience Medical Center
    • San Francisco, California, United States, 94143
      • University of California, San Francisco
  • Colorado
    • Aurora, Colorado, United States, 80045
      • University of Colorado Hospital Anschutz Outpatient Pavillion
  • Connecticut
    • New Haven, Connecticut, United States, 06519
      • Yale School of Medicine
  • Florida
    • Jacksonville, Florida, United States, 32224
      • Mayo Clinic
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Northwestern Memorial Hospital, Department of Neurology (Clinic)
  • Kansas
    • Kansas City, Kansas, United States, 66160
      • University of Kansas Medical
  • Maryland
    • Baltimore, Maryland, United States, 21287
      • Johns Hopkins University School of Medicine
    • Baltimore, Maryland, United States, 21224
      • Johns Hopkins Bayview Medical Center
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Neuromuscular Diagnostic Center - Massachusetts General Hospital
    • Boston, Massachusetts, United States, 021158
      • Brigham and Womens Hospital
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic
  • Missouri
    • Saint Louis, Missouri, United States, 63110
      • Washington University School of Medicine
  • Nebraska
    • Omaha, Nebraska, United States, 98198
      • University Of Nebraska Medical Center
  • New York
    • New York, New York, United States, 10021
      • Hospital for Special Surgery
    • New York, New York, United States, 10032
      • Columbia University Medical Center / The Neurological Institute of New York
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Duke Neurological Disorders Clinic -1L
    • Winston-Salem, North Carolina, United States, 27157
      • Wake Forrest School of Medicine
  • Ohio
    • Cleveland, Ohio, United States, 44106
      • University Hospitals Cleveland Medical Center
    • Columbus, Ohio, United States, 43210
      • The Ohio State University Wexner Medical Center
  • Oregon
    • Portland, Oregon, United States, 97239
      • Oregon Health & Science University
  • Pennsylvania
    • Hershey, Pennsylvania, United States, 17033
      • Penn State Health Milton S. Hershey Medical Center
    • Philadelphia, Pennsylvania, United States, 19104
      • University of Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15213
      • UPMC Arthritis and Autoimmunity Center, Falk Clinic
  • Texas
    • Austin, Texas, United States, 78759
      • Austin Neuromuscular Center
    • Dallas, Texas, United States, 75206
      • Texas Neurology
    • Houston, Texas, United States, 77030
      • Nerve and Muscle Center of Texas
  • Virginia
    • Henrico, Virginia, United States, 23233
      • Virginia Commonwealth University
  • Washington
    • Seattle, Washington, United States, 98195
      • University of Washington Medical Center - Montlake
  • Wisconsin
    • Milwaukee, Wisconsin, United States, 53226
      • Medical College of Wisconsin

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 40 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: 0.5 mg/kg ABC008; Part A - ABC008 N=12 Part B - ABC008 N= 67	Drug: ABC008; Given by subcutaneous injection
Active Comparator: 2.0 mg/kg ABC008; Part A - ABC008 N=12 Part B - ABC008 N= 67	Drug: ABC008; Given by subcutaneous injection
Placebo Comparator: Placebo; Part A - Placebo N= 6 Part B - Placebo N= 67	Drug: ABC008; Given by subcutaneous injection

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Abcuro, Inc.
• Collaborators: Syneos Health

Publications and helpful links

Helpful Links

• Study Website

Study record dates

Study Major Dates

• Study Start (Actual): February 28, 2023
• Primary Completion (Estimated): November 1, 2025
• Study Completion (Estimated): December 1, 2025

Study Registration Dates

• First Submitted: February 1, 2023
• First Submitted That Met QC Criteria: February 1, 2023
• First Posted (Actual): February 10, 2023

Study Record Updates

• Last Update Posted (Actual): April 5, 2024
• Last Update Submitted That Met QC Criteria: April 4, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Keywords: Myositis; Neuromuscular Diseases; Muscular Diseases; Nervous System Disease; Inflammatory Myopathy
• Additional Relevant MeSH Terms: Nervous System Diseases; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Myositis; Myositis, Inclusion Body
• Other Study ID Numbers: ABC008-IBM-201

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No