Inclusion Body Myositis
Conditions
Keywords
Inclusion Body Myositis, IBM
Brief summary
An open-label, ascending dose study for adult patients with Inclusion Body Myositis (IBM).
Detailed 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.
Interventions
DRUGABC008
ABC008
Sponsors
Abcuro, Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
40 Years to No maximum
Healthy volunteers
No
Inclusion criteria
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Assessment of Safety and Tolerability | 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
| Measure | Time frame | Description |
|---|---|---|
| Assessment of time to peak serum concentration (Tmax) | Day 1 and throughout the 24 weeks of follow up | Assess the time to peak serum concentration (Tmax) of a single dose of ABC008 |
| Assessment of terminal half-life (t½) | Day 1 | Assess the terminal half-life (t½) of ABC008 |
| Assessment of area under the concentration versus time curve from time zero to 24 hours post-dose (AUC0-24hr) | 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) |
| Assessment of apparent clearance (CL/F) | Day 1 and throughout the 24 weeks of follow up | Assessment of apparent clearance (CL/F) of a single dose of ABC008 |
| Assessment of apparent volume of distribution (Vz/F) | Day 1 and throughout the 24 weeks of follow up | Assessment of apparent volume of distribution (Vz/F) of a single dose of ABC008 |
| Characterization of changes in KLRG1 expressing lymphocytes | Day 1 and throughout the 24 weeks of follow up | Characterize changes in KLRG1 expressing lymphocytes |
| Qualitative assessment of [ 89Zr]Zr-Df-crefmirlimab | [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 |
| Assessment of peak serum concentration (Cmax) | Day 1 and throughout the 24 weeks of follow up | Assess the peak serum concentration (Cmax) of a single dose of ABC008 |
| Assessment of global distribution of [ 89Zr]Zr-Df-crefmirlimab uptake in lymphoid organs | [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 |
| Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab pre and post dosing of ABC008 | [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 |
| Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab, determined by standardized uptake value (SUV)-based quantitative analysis peak (SUVpeak) | [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) |
| Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab, determined by standardized uptake value (SUV)-based quantitative analysis mean (SUVmean) | [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) |
| Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab, determined by standardized uptake value (SUV)-based quantitative analysis SUV of diseased muscle | [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 |
| Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab, determined by standardized uptake value (SUV)-based quantitative analysis SUV reference tissue | [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 |
| Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab, determined by standardized uptake value (SUV)-based quantitative analysis maximum (SUVmax) | [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) |
| Assessment of global distribution of [ 89Zr]Zr-Df-crefmirlimab uptake in skeletal muscle | [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 |
Countries
Australia
Outcome results
None listed