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

Inclusion Body Myositis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05721573
• Other study ID #: ABC008-IBM-201
• Status: Active, not recruiting
• Phase: Phase 2/Phase 3
• Start date: February 28, 2023
• Est. completion date: December 2025

Study information

• Verified date: April 2024
• Source: Abcuro, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 231
• Est. completion date: December 2025
• Est. primary completion date: November 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 40 Years and older

Eligibility

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;

Related Conditions & MeSH terms

• Inclusion Body Myositis
• Myositis
• Myositis, Inclusion Body

Intervention

Drug:
• ABC008
Given by subcutaneous injection

Locations

Country	Name	City	State
Australia	Royal Brisbane and Women's Hospital	Herston	Queensland
Australia	Perron Institute for Neurological and Translational Science	Nedlands	Western Australia
Australia	Royal North Shore Hospital	Saint Leonards	New South Wales
Belgium	AZ Sint-Lucas & Volkskliniek	Gent
Canada	Heritage Medical Research Clinic - University of Calgary	Calgary	Alberta
Canada	Genge Partners Inc.	Montréal	Quebec
France	Hospital Pitie-Salpetriere - AP-HP	Paris
Germany	Krankenhaus und Poliklinik Rüdersdorf GmbH	Berlin
Germany	University Hosptial Duesseldorf	Düsseldorf
United Kingdom	University College London Hospitals NHS Foundation Trust, National Hospital for Neurology and Neurosurgery (NHNN)	London
United Kingdom	Salford Royal Hospital, Northern Care Alliance NHS Foundation Trust	Salford
United States	University of Colorado Hospital Anschutz Outpatient Pavillion	Aurora	Colorado
United States	Austin Neuromuscular Center	Austin	Texas
United States	Johns Hopkins Bayview Medical Center	Baltimore	Maryland
United States	Johns Hopkins University School of Medicine	Baltimore	Maryland
United States	Brigham and Womens Hospital	Boston	Massachusetts
United States	Neuromuscular Diagnostic Center - Massachusetts General Hospital	Boston	Massachusetts
United States	Northwestern Memorial Hospital, Department of Neurology (Clinic)	Chicago	Illinois
United States	University Hospitals Cleveland Medical Center	Cleveland	Ohio
United States	The Ohio State University Wexner Medical Center	Columbus	Ohio
United States	Texas Neurology	Dallas	Texas
United States	Duke Neurological Disorders Clinic -1L	Durham	North Carolina
United States	Virginia Commonwealth University	Henrico	Virginia
United States	Penn State Health Milton S. Hershey Medical Center	Hershey	Pennsylvania
United States	Nerve and Muscle Center of Texas	Houston	Texas
United States	University of California Irvine Medical Center (UCIMC) - Amyotrophic Lateral Sclerosis (ALS) and Neuromuscular Center	Irvine	California
United States	Mayo Clinic	Jacksonville	Florida
United States	University of Kansas Medical	Kansas City	Kansas
United States	Keck Hosptial of USC	Los Angeles	California
United States	UCLA Medical Center	Los Angeles	California
United States	Medical College of Wisconsin	Milwaukee	Wisconsin
United States	Yale School of Medicine	New Haven	Connecticut
United States	Columbia University Medical Center / The Neurological Institute of New York	New York	New York
United States	Hospital for Special Surgery	New York	New York
United States	University of Nebraska Medical Center	Omaha	Nebraska
United States	Stanford Neuroscience Medical Center	Palo Alto	California
United States	University of Pennsylvania	Philadelphia	Pennsylvania
United States	Neuromuscular Research Center	Phoenix	Arizona
United States	UPMC Arthritis and Autoimmunity Center, Falk Clinic	Pittsburgh	Pennsylvania
United States	Oregon Health & Science University	Portland	Oregon
United States	Mayo Clinic	Rochester	Minnesota
United States	Washington University School of Medicine	Saint Louis	Missouri
United States	University of California, San Francisco	San Francisco	California
United States	University of Washington Medical Center - Montlake	Seattle	Washington
United States	Wake Forrest School of Medicine	Winston-Salem	North Carolina

Sponsors (2)

Lead Sponsor	Collaborator
Abcuro, Inc.	Syneos Health

Countries where clinical trial is conducted

United States,  Australia,  Belgium,  Canada,  France,  Germany,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.
Primary	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
Secondary	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.
Secondary	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.
Secondary	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.
Secondary	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.
Secondary	Part A - Adverse Events of Special Interest (AESI)	Incidence of AESIs.	From Baseline (Day 1) through study completion, an average of 80 weeks.
Secondary	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.
Secondary	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.
Secondary	Part B - Quadriceps Dynamometry	Mean change in quadriceps strength by dynamometry.	From Baseline (Day 1) through study completion, an average of 76 weeks.
Secondary	Part B - Modified Timed Up and Go (mTUG)	Mean change in mTUG.	From Baseline (Day 1) through study completion, an average of 76 weeks.

External Links

•   Study Website