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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT04659031




Registration number
NCT04659031
Ethics application status
Date submitted
25/11/2020
Date registered
9/12/2020
Date last updated
6/06/2024

Titles & IDs
Public title
A Phase 1 Study of ABC008 in Ascending (Single Ascending Dose/Multiple Ascending Dose) Study in Patients With (IBM)
Scientific title
A Phase 1, Open-Label, Single and Multiple Ascending Dose Study of ABC008 in Adult Patients With Inclusion Body Myositis (IBM)
Secondary ID [1] 0 0
ABC008-IBM-101
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

Experimental: Cohort D1 - Single Dose 0.1 mg / kg ABC008

Experimental: Cohort D2 - Single Dose 0.5 mg / kg ABC008

Experimental: Cohort D3 - Single Dose 2.0 mg / kg ABC008

Experimental: Cohort D4 - Single Dose 5.0 mg / kg ABC008

Experimental: Cohort D5 - X.X mg / kg ABC008

Experimental: Cohort 6 - Single 2.0 mg / kg ABC008

Experimental: MAD Phase Cohort 1 - Multiple Dose 0.1 mg / kg ABC008 every 8 weeks

Experimental: MAD Phase Cohort 2 - Multiple Dose 0.5 mg / kg ABC008 every 8 weeks

Experimental: MAD Phase Cohort 3 - Multiple Dose 2.0 mg / kg ABC008 every 8 weeks


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

Outcomes
Primary outcome [1] 0 0
Assessment of Safety and Tolerability
Timepoint [1] 0 0
Through Study Completion an average of 28 weeks for SAD (Single Ascending Dose) phase and 52 weeks for MAD (Multiple Ascending Dose) phase]
Secondary outcome [1] 0 0
Assessment of peak serum concentration (Cmax)
Timepoint [1] 0 0
Day 1 and throughout the 24 weeks of follow up
Secondary outcome [2] 0 0
Assessment of time to peak serum concentration (Tmax)
Timepoint [2] 0 0
Day 1 and throughout the 24 weeks of follow up
Secondary outcome [3] 0 0
Assessment of terminal half-life (t½)
Timepoint [3] 0 0
Day 1
Secondary outcome [4] 0 0
Assessment of area under the concentration versus time curve from time zero to 24 hours post-dose (AUC0-24hr)
Timepoint [4] 0 0
Day 1
Secondary outcome [5] 0 0
Assessment of apparent clearance (CL/F)
Timepoint [5] 0 0
Day 1 and throughout the 24 weeks of follow up
Secondary outcome [6] 0 0
Assessment of apparent volume of distribution (Vz/F)
Timepoint [6] 0 0
Day 1 and throughout the 24 weeks of follow up
Secondary outcome [7] 0 0
Characterization of changes in KLRG1 expressing lymphocytes
Timepoint [7] 0 0
Day 1 and throughout the 24 weeks of follow up
Secondary outcome [8] 0 0
Qualitative assessment of [ 89Zr]Zr-Df-crefmirlimab
Timepoint [8] 0 0
[Through Study Completion, avg. 48 weeks
Secondary outcome [9] 0 0
Assessment of global distribution of [ 89Zr]Zr-Df-crefmirlimab uptake in skeletal muscle
Timepoint [9] 0 0
[Through Study Completion, avg. 48 weeks
Secondary outcome [10] 0 0
Assessment of global distribution of [ 89Zr]Zr-Df-crefmirlimab uptake in lymphoid organs
Timepoint [10] 0 0
[Through Study Completion, avg. 48 weeks
Secondary outcome [11] 0 0
Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab pre and post dosing of ABC008
Timepoint [11] 0 0
[Through Study Completion, avg. 48 weeks
Secondary outcome [12] 0 0
Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab, determined by standardized uptake value (SUV)-based quantitative analysis peak (SUVpeak)
Timepoint [12] 0 0
[Through Study Completion, avg. 48 weeks
Secondary outcome [13] 0 0
Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab, determined by standardized uptake value (SUV)-based quantitative analysis mean (SUVmean)
Timepoint [13] 0 0
[Through Study Completion, avg. 48 weeks
Secondary outcome [14] 0 0
Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab, determined by standardized uptake value (SUV)-based quantitative analysis SUV of diseased muscle
Timepoint [14] 0 0
[Through Study Completion, avg. 48 weeks
Secondary outcome [15] 0 0
Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab, determined by standardized uptake value (SUV)-based quantitative analysis SUV reference tissue
Timepoint [15] 0 0
[Through Study Completion, avg. 48 weeks
Secondary outcome [16] 0 0
Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab, determined by standardized uptake value (SUV)-based quantitative analysis maximum (SUVmax)
Timepoint [16] 0 0
[Through Study Completion, avg. 48 weeks

Eligibility
Key inclusion criteria
Key

- Diagnosis of either clinico-pathologically defined IBM, clinically defined IBM, or
probable IBM according to the European Neuromuscular Center (ENMC) IBM 2011

- Able to arise from a chair (with or without armrests) without support from another
person or device

- Able to ambulate at least 20 feet / 6 meters with or without assistive device
Minimum age
40 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Taking > 7.5 mg prednisolone (or equivalent) or on intravenous immunoglobulin (IVIg)
or other immunosuppressants within the last 3 months. Topical, nasal, and ocular
corticosteroids are allowed unless they are being widely applied or the severity of
the underlying condition makes them unsuitable in the Investigator's opinion. Local
steroid injections are allowed

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised 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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 1
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
25/05/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/08/2025
Actual
Sample size
Target
30
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
SA
Recruitment hospital [1] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [2] 0 0
Royal Brisbane - Herston
Recruitment hospital [3] 0 0
Perron Institute - Perth
Recruitment hospital [4] 0 0
Royal North Shore Hospital - Sydney
Recruitment postcode(s) [1] 0 0
5000 - Adelaide
Recruitment postcode(s) [2] 0 0
- Herston
Recruitment postcode(s) [3] 0 0
- Perth
Recruitment postcode(s) [4] 0 0
- Sydney

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Abcuro, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
An open-label, ascending dose study for adult patients with Inclusion Body Myositis (IBM).
Trial website
https://clinicaltrials.gov/ct2/show/NCT04659031
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