Skip to main content
OpenTrials
Completed

NCT Number: NCT04659031

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

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

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Inclusion Body Myositis Muscular Diseases Musculoskeletal Diseases Myositis Myositis, Inclusion Body Nervous System Diseases Neuromuscular Diseases

Age range

40 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Royal Adelaide Hospital, Adelaide, South Australia, Australia

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

ABC008

Drug

ABC008

Primary outcomes

  1. Assessment of Safety and Tolerability

    Time frame: Through Study Completion an average of 28 weeks for SAD (Single Ascending Dose) phase and 52 weeks for MAD (Multiple Ascending Dose) phase]

    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.

Secondary outcomes

  1. Assessment of peak serum concentration (Cmax)

    Time frame: Day 1 and throughout the 24 weeks of follow up

    Assess the peak serum concentration (Cmax) of a single dose of ABC008

  2. Assessment of time to peak serum concentration (Tmax)

    Time frame: Day 1 and throughout the 24 weeks of follow up

    Assess the time to peak serum concentration (Tmax) of a single dose of ABC008

  3. Assessment of terminal half-life (t½)

    Time frame: Day 1

    Assess the terminal half-life (t½) of ABC008

  4. Assessment of area under the concentration versus time curve from time zero to 24 hours post-dose (AUC0-24hr)

    Time frame: Day 1

    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)

  5. Assessment of apparent clearance (CL/F)

    Time frame: Day 1 and throughout the 24 weeks of follow up

    Assessment of apparent clearance (CL/F) of a single dose of ABC008

  6. Assessment of apparent volume of distribution (Vz/F)

    Time frame: Day 1 and throughout the 24 weeks of follow up

    Assessment of apparent volume of distribution (Vz/F) of a single dose of ABC008

  7. Characterization of changes in KLRG1 expressing lymphocytes

    Time frame: Day 1 and throughout the 24 weeks of follow up

    Characterize changes in KLRG1 expressing lymphocytes

  8. Qualitative assessment of [ 89Zr]Zr-Df-crefmirlimab

    Time frame: [Through Study Completion, avg. 48 weeks

    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

  9. Assessment of global distribution of [ 89Zr]Zr-Df-crefmirlimab uptake in skeletal muscle

    Time frame: [Through Study Completion, avg. 48 weeks

    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

  10. Assessment of global distribution of [ 89Zr]Zr-Df-crefmirlimab uptake in lymphoid organs

    Time frame: [Through Study Completion, avg. 48 weeks

    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

  11. Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab pre and post dosing of ABC008

    Time frame: [Through Study Completion, avg. 48 weeks

    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

  12. Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab, determined by standardized uptake value (SUV)-based quantitative analysis peak (SUVpeak)

    Time frame: [Through Study Completion, avg. 48 weeks

    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)

  13. Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab, determined by standardized uptake value (SUV)-based quantitative analysis mean (SUVmean)

    Time frame: [Through Study Completion, avg. 48 weeks

    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)

  14. Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab, determined by standardized uptake value (SUV)-based quantitative analysis SUV of diseased muscle

    Time frame: [Through Study Completion, avg. 48 weeks

    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

  15. Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab, determined by standardized uptake value (SUV)-based quantitative analysis SUV reference tissue

    Time frame: [Through Study Completion, avg. 48 weeks

    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

  16. Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab, determined by standardized uptake value (SUV)-based quantitative analysis maximum (SUVmax)

    Time frame: [Through Study Completion, avg. 48 weeks

    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)

Sponsors and collaborators

Lead sponsor

Abcuro, Inc.

Industry

Registry information

Official study title

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

Important dates

Study start
2021
Primary completion
2025
Study completion
2025
First posted
Dec 9, 2020
Registry last updated
Feb 20, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.