A Phase 1 Study of ABC008 in Ascending (Single Ascending Dose/Multiple Ascending Dose) Study in Patients With (IBM)

Inclusion Body Myositis Clinical Trial

Official title:

A Phase 1, Open-Label, Single and Multiple Ascending Dose Study of ABC008 in Adult Patients With Inclusion Body Myositis (IBM)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04659031
• Other study ID #: ABC008-IBM-101
• Status: Active, not recruiting
• Phase: Phase 1
• Start date: May 25, 2021
• Est. completion date: August 2025

Study information

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

Clinical Trial Summary

An open-label, ascending dose study for adult patients with Inclusion Body Myositis (IBM).

Description:

Participants who successfully complete the SAD EOT visit, and have no emerging safety issues, will be eligible to enroll in Part 2 (MAD). Eligible participants for the MAD part will have inclusion and exclusion criteria (same as those for Part 1) reviewed prior to dosing on MAD Day 1.

Participants who successfully complete the MAD EOT visit, and have no emerging safety issues, will be eligible to enrol in Part 3, MAD Extension.

After the final MAD visit (W48), participants will have the option to continue on to Part 3 MAD Extension.

For Part 3 (MAD Extension), participant dosing will be at 8-week intervals starting at Day 1. Duration of dosing in Part 3 will be up to approximately 80 weeks (18 months), or until a new long-term extension study has been initiated. The SMC will review all participant safety data approximately every 6 months while the Part 3 dosing continues.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 30
• Est. completion date: August 2025
• Est. primary completion date: March 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 40 Years and older

Eligibility

Key Inclusion Criteria:

• 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

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

Related Conditions & MeSH terms

• Inclusion Body Myositis
• Myositis
• Myositis, Inclusion Body

Intervention

Drug:
• ABC008
ABC008

Locations

Country	Name	City	State
Australia	Royal Adelaide Hospital	Adelaide	South Australia
Australia	Royal Brisbane	Herston
Australia	Perron Institute	Perth
Australia	Royal North Shore Hospital	Sydney

Sponsors (1)

Lead Sponsor	Collaborator
Abcuro, Inc.

Country where clinical trial is conducted

Australia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Assessment of Safety and Tolerability	Characterize the safety and tolerability profile of single (SAD) and multiple (MAD) escalating dose levels of ABC008 in IBM when administered subcutaneously (SC) as measured by the number and severity of treatment emergent adverse events, serious adverse events, and adverse events of special interest, number of dose limiting toxicities.	Through Study Completion an average of 28 weeks for SAD (Single Ascending Dose) phase and 52 weeks for MAD (Multiple Ascending Dose) phase]
Secondary	Assessment of peak serum concentration (Cmax)	Assess the peak serum concentration (Cmax) of a single dose of ABC008	Day 1 and throughout the 24 weeks of follow up
Secondary	Assessment of time to peak serum concentration (Tmax)	Assess the time to peak serum concentration (Tmax) of a single dose of ABC008	Day 1 and throughout the 24 weeks of follow up
Secondary	Assessment of terminal half-life (t½)	Assess the terminal half-life (t½) of ABC008	Day 1
Secondary	Assessment of area under the concentration versus time curve from time zero to 24 hours post-dose (AUC0-24hr)	Assess the area under the concentration versus time curve of a single dose of ABC008 from time zero to 24 hours post-dose (AUC0-24hr)	Day 1
Secondary	Assessment of apparent clearance (CL/F)	Assessment of apparent clearance (CL/F) of a single dose of ABC008	Day 1 and throughout the 24 weeks of follow up
Secondary	Assessment of apparent volume of distribution (Vz/F)	Assessment of apparent volume of distribution (Vz/F) of a single dose of ABC008	Day 1 and throughout the 24 weeks of follow up
Secondary	Characterization of changes in KLRG1 expressing lymphocytes	Characterize changes in KLRG1 expressing lymphocytes	Day 1 and throughout the 24 weeks of follow up
Secondary	Qualitative assessment of [ 89Zr]Zr-Df-crefmirlimab	Qualitative assessment of [ 89Zr]Zr-Df-crefmirlimab uptake in involved skeletal muscles including inflamed and non-inflamed sites as determined by using a visual scoring (VS) system for the time point assessed, the possible scores VS1-VS5	[Through Study Completion, avg. 48 weeks
Secondary	Assessment of global distribution of [ 89Zr]Zr-Df-crefmirlimab uptake in skeletal muscle	Assessment of global distribution of [ 89Zr]Zr-Df-crefmirlimab uptake in skeletal muscle; Pattern(s) of absolute and relative changes in uptake within various skeletal muscle groups; Homogenous/diffuse, Focal, Mixed, Other	[Through Study Completion, avg. 48 weeks
Secondary	Assessment of global distribution of [ 89Zr]Zr-Df-crefmirlimab uptake in lymphoid organs	Assessment of global distribution of [ 89Zr]Zr-Df-crefmirlimab uptake in lymphoid organs; Uptake and relative changes in uptake within lymphoid tissue including spleen and lymph nodes as well as other T-cell rich tissues such as bone marrow	[Through Study Completion, avg. 48 weeks
Secondary	Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab pre and post dosing of ABC008	Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab uptake and relative changes in uptake within inflamed muscle tissue through Positron Emission Tomography (PET)/computed tomography (CT) imaging pre- and post-dosing with ABC008	[Through Study Completion, avg. 48 weeks
Secondary	Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab, determined by standardized uptake value (SUV)-based quantitative analysis peak (SUVpeak)	Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab uptake in involved skeletal muscles including inflamed and non-inflamed sites, and measurement of magnitude of difference observations as determined by standardized uptake value (SUV)-based quantitative analysis peak (SUVpeak)	[Through Study Completion, avg. 48 weeks
Secondary	Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab, determined by standardized uptake value (SUV)-based quantitative analysis mean (SUVmean)	Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab uptake in involved skeletal muscles including inflamed and non-inflamed sites, and measurement of magnitude of difference observations as determined by standardized uptake value (SUV)-based quantitative analysis mean (SUVmean)	[Through Study Completion, avg. 48 weeks
Secondary	Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab, determined by standardized uptake value (SUV)-based quantitative analysis SUV of diseased muscle	Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab uptake in involved skeletal muscles including inflamed and non-inflamed sites, and measurement of magnitude of difference observations as determined by standardized uptake value (SUV)-based quantitative analysis SUV of diseased muscle	[Through Study Completion, avg. 48 weeks
Secondary	Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab, determined by standardized uptake value (SUV)-based quantitative analysis SUV reference tissue	Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab uptake in involved skeletal muscles including inflamed and non-inflamed sites, and measurement of magnitude of difference observations as determined by standardized uptake value (SUV)-based quantitative analysis SUV reference tissue	[Through Study Completion, avg. 48 weeks
Secondary	Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab, determined by standardized uptake value (SUV)-based quantitative analysis maximum (SUVmax)	Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab uptake in involved skeletal muscles including inflamed and non-inflamed sites, and measurement of magnitude of difference observations as determined by standardized uptake value (SUV)-based quantitative analysis maximum (SUVmax)	[Through Study Completion, avg. 48 weeks