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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05721573




Registration number
NCT05721573
Ethics application status
Date submitted
1/02/2023
Date registered
10/02/2023

Titles & IDs
Public title
A Study to Evaluate the Efficacy and Safety of ABC008 for Inclusion Body Myositis
Scientific 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
Secondary ID [1] 0 0
ABC008-IBM-201
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Inclusion Body Myositis 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - ABC008

Active comparator: 0.5 mg/kg ABC008 - Part A - ABC008 N=12

Part B - ABC008 N= 67

Active comparator: 2.0 mg/kg ABC008 - Part A - ABC008 N=12

Part B - ABC008 N= 67

Placebo comparator: Placebo - Part A - Placebo N= 6

Part B - Placebo N= 67


Treatment: Drugs: ABC008
Given by subcutaneous injection
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Part A - To determine the safety and tolerability of recurrent dosing of ABC008 in subjects with IBM at 2 SC dose levels.
Timepoint [1] 0 0
From Baseline (week 0) through week 20.
Primary outcome [2] 0 0
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
Timepoint [2] 0 0
From Baseline (week 0) through study completion, an average of 76 weeks
Secondary outcome [1] 0 0
Part A - Treatment Emergent Serious Adverse Events (TEASAEs)
Timepoint [1] 0 0
From Baseline (Day 1) through study completion, an average of 80 weeks.
Secondary outcome [2] 0 0
Part A - Treatment Emergent Adverse Events (TEAEs) onset within 24 hours of Study Medication Administration.
Timepoint [2] 0 0
From Baseline (Day 1) through study completion, an average of 80 weeks.
Secondary outcome [3] 0 0
Part A - Treatment Emergent Adverse Events leading to study medication or study discontinuation.
Timepoint [3] 0 0
From Baseline (Day 1) through study completion, an average of 80 weeks.
Secondary outcome [4] 0 0
Part A - Clinically significant changes in standard laboratory parameters, vital signs, and ECGs
Timepoint [4] 0 0
From Baseline (Day 1) through study completion, an average of 80 weeks.
Secondary outcome [5] 0 0
Part A - Adverse Events of Special Interest (AESI)
Timepoint [5] 0 0
From Baseline (Day 1) through study completion, an average of 80 weeks.
Secondary outcome [6] 0 0
Part B - Manual Muscle Test 12 (MMT 12)
Timepoint [6] 0 0
From Baseline (Day 1) through study completion, an average of 76 weeks.
Secondary outcome [7] 0 0
Part B - Hand Grip Dynamometry
Timepoint [7] 0 0
From Baseline (Day 1) through study completion, an average of 76 weeks.
Secondary outcome [8] 0 0
Part B - Quadriceps Dynamometry
Timepoint [8] 0 0
From Baseline (Day 1) through study completion, an average of 76 weeks.
Secondary outcome [9] 0 0
Part B - Modified Timed Up and Go (mTUG)
Timepoint [9] 0 0
From Baseline (Day 1) through study completion, an average of 76 weeks.

Eligibility
Key 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;
Minimum age
40 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
28/02/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/12/2025
Actual
Sample size
Target
231
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,WA
Recruitment hospital [1] 0 0
Royal North Shore Hospital - Saint Leonards
Recruitment hospital [2] 0 0
Royal Brisbane and Women's Hospital - Herston
Recruitment hospital [3] 0 0
Perron Institute for Neurological and Translational Science - Nedlands
Recruitment postcode(s) [1] 0 0
2065 - Saint Leonards
Recruitment postcode(s) [2] 0 0
4006 - Herston
Recruitment postcode(s) [3] 0 0
6009 - Nedlands
Recruitment outside Australia
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United States of America
State/province [1] 0 0
Arizona
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United States of America
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California
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Colorado
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Connecticut
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Florida
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Illinois
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Kansas
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Maryland
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Massachusetts
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Minnesota
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Missouri
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Nebraska
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New York
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North Carolina
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Ohio
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Oregon
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Pennsylvania
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Texas
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Virginia
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Washington
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United States of America
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Wisconsin
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Belgium
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Gent
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Canada
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Alberta
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Canada
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Quebec
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France
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Paris
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Germany
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Berlin
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Germany
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Düsseldorf
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United Kingdom
State/province [28] 0 0
London
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United Kingdom
State/province [29] 0 0
Salford

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Abcuro, Inc.
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
Syneos Health
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05721573