Inclusion Body MyositisPhase 1NCT04659031
A Phase 1 Study of ABC008 in Ascending (Single Ascending Dose/Multiple Ascending Dose) Study in Patients With (IBM)
A Phase 1 study testing ABC008 in people with Inclusion Body Myositis, run by Abcuro, Inc. at 4 sites in Australia.
Trial summary
An open-label, ascending dose study for adult patients with Inclusion Body Myositis (IBM). Read more about this study
- Who is it for?
- Ages 40 years and older
- Conditions the study is for
- Inclusion Body Myositis
- What is being tested?
- ABC008
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 4 sitesAustralia
- Study dates
- 25 May 2021to 10 Jan 2025
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 40 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
4 sites in 1 country.
Australia4 sites
Royal Adelaide Hospital
Adelaide, South Australia, 5000
Royal Brisbane
Herston
Perron Institute
Perth
Royal North Shore Hospital
Sydney
About this study
Official title: A Phase 1, Open-Label, Single and Multiple Ascending Dose Study of ABC008 in Adult Patients With Inclusion Body Myositis (IBM)
An open-label, ascending dose study for adult patients with Inclusion Body Myositis (IBM).
Read the 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.
- Conditions
- Inclusion Body Myositis
- Being tested
- ABC008
- Keywords
- Inclusion Body Myositis, IBM
- Medical subject headings
- Myositis, Inclusion Body
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ABC008Drug
ABC008
| Study group | Role | What they receive |
|---|---|---|
| Cohort D1 | Experimental | Single Dose 0.1 mg / kg ABC008 Receives: Drug: ABC008 |
| Cohort D2 | Experimental | Single Dose 0.5 mg / kg ABC008 Receives: Drug: ABC008 |
| Cohort D3 | Experimental | Single Dose 2.0 mg / kg ABC008 Receives: Drug: ABC008 |
| Cohort D4 | Experimental | Single Dose 5.0 mg / kg ABC008 Receives: Drug: ABC008 |
| Cohort D5 | Experimental | X.X mg / kg ABC008 Receives: Drug: ABC008 |
| Cohort 6 | Experimental | Single 2.0 mg / kg ABC008 Receives: Drug: ABC008 |
| MAD Phase Cohort 1 | Experimental | Multiple Dose 0.1 mg / kg ABC008 every 8 weeks Receives: Drug: ABC008 |
| MAD Phase Cohort 2 | Experimental | Multiple Dose 0.5 mg / kg ABC008 every 8 weeks Receives: Drug: ABC008 |
| MAD Phase Cohort 3 | Experimental | Multiple Dose 2.0 mg / kg ABC008 every 8 weeks Receives: Drug: ABC008 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Through Study Completion an average of 28 weeks for SAD (Single Ascending Dose) phase and 52 weeks for MAD (Multiple Ascending Dose) phase]
Secondary outcomes (16)
Assessment of peak serum concentration (Cmax)
Assess the peak serum concentration (Cmax) of a single dose of ABC008
Measured: Day 1 and throughout the 24 weeks of follow up
Assessment of time to peak serum concentration (Tmax)
Assess the time to peak serum concentration (Tmax) of a single dose of ABC008
Measured: Day 1 and throughout the 24 weeks of follow up
Assessment of terminal half-life (t½)
Assess the terminal half-life (t½) of ABC008
Measured: Day 1
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)
Measured: Day 1
Assessment of apparent clearance (CL/F)
Assessment of apparent clearance (CL/F) of a single dose of ABC008
Measured: Day 1 and throughout the 24 weeks of follow up
Assessment of apparent volume of distribution (Vz/F)
Assessment of apparent volume of distribution (Vz/F) of a single dose of ABC008
Measured: Day 1 and throughout the 24 weeks of follow up
Characterization of changes in KLRG1 expressing lymphocytes
Characterize changes in KLRG1 expressing lymphocytes
Measured: Day 1 and throughout the 24 weeks of follow up
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
Measured: [Through Study Completion, avg. 48 weeks
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
Measured: [Through Study Completion, avg. 48 weeks
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
Measured: [Through Study Completion, avg. 48 weeks
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
Measured: [Through Study Completion, avg. 48 weeks
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)
Measured: [Through Study Completion, avg. 48 weeks
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)
Measured: [Through Study Completion, avg. 48 weeks
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
Measured: [Through Study Completion, avg. 48 weeks
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
Measured: [Through Study Completion, avg. 48 weeks
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)
Measured: [Through Study Completion, avg. 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Dec 2020
- Study start25 May 2021
- Primary completion10 Jan 2025
- Study completion10 Jan 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Inclusion Body MyositisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Abcuro, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04659031
- Sponsor's protocol ID
- ABC008-IBM-101
- Status last verified
- Jun 2024
- First submitted
- 25 Nov 2020
- Last updated
- 20 Feb 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 20 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Open-Label, Single and Multiple Ascending Dose Study of ABC008 in Adult Patients With Inclusion Body Myositis (IBM)
- ClinicalTrials.gov ID
- NCT04659031
- Acronym / sponsor ID
- ABC008-IBM-101
- Conditions
- Inclusion Body Myositis
- Keywords
- Inclusion Body Myositis, IBM
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- Abcuro, Inc.Industry (pharma, biotech, devices)
- Study start
- 25 May 2021 (actual)
- Primary completion
- 10 Jan 2025 (actual)
- Study completion
- 10 Jan 2025 (actual)
- First posted
- 9 Dec 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 9
| Arm | Type | Receives |
|---|---|---|
| Cohort D1 | Experimental | Drug: ABC008Single Dose 0.1 mg / kg ABC008 |
| Cohort D2 | Experimental | Drug: ABC008Single Dose 0.5 mg / kg ABC008 |
| Cohort D3 | Experimental | Drug: ABC008Single Dose 2.0 mg / kg ABC008 |
| Cohort D4 | Experimental | Drug: ABC008Single Dose 5.0 mg / kg ABC008 |
| Cohort D5 | Experimental | Drug: ABC008X.X mg / kg ABC008 |
| Cohort 6 | Experimental | Drug: ABC008Single 2.0 mg / kg ABC008 |
| MAD Phase Cohort 1 | Experimental | Drug: ABC008Multiple Dose 0.1 mg / kg ABC008 every 8 weeks |
| MAD Phase Cohort 2 | Experimental | Drug: ABC008Multiple Dose 0.5 mg / kg ABC008 every 8 weeks |
| MAD Phase Cohort 3 | Experimental | Drug: ABC008Multiple Dose 2.0 mg / kg ABC008 every 8 weeks |
Who may qualify
- Ages
- 40 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (1 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ABC008ABC008 | Drug | Cohort 6, Cohort D1, Cohort D2, Cohort D3, Cohort D4, Cohort D5, MAD Phase Cohort 1, MAD Phase Cohort 2, MAD Phase Cohort 3 |
Primary outcome
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.
16 secondary outcomes
Assessment of peak serum concentration (Cmax)
Time frame: Day 1 and throughout the 24 weeks of follow up
Assessment of time to peak serum concentration (Tmax)
Time frame: Day 1 and throughout the 24 weeks of follow up
Assessment of terminal half-life (t½)
Time frame: Day 1
Assessment of area under the concentration versus time curve from time zero to 24 hours post-dose (AUC0-24hr)
Time frame: Day 1
Assessment of apparent clearance (CL/F)
Time frame: Day 1 and throughout the 24 weeks of follow up
Assessment of apparent volume of distribution (Vz/F)
Time frame: Day 1 and throughout the 24 weeks of follow up
Characterization of changes in KLRG1 expressing lymphocytes
Time frame: Day 1 and throughout the 24 weeks of follow up
Qualitative assessment of [ 89Zr]Zr-Df-crefmirlimab
Time frame: [Through Study Completion, avg. 48 weeks
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 lymphoid organs
Time frame: [Through Study Completion, avg. 48 weeks
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, 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, 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, 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, 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, determined by standardized uptake value (SUV)-based quantitative analysis maximum (SUVmax)
Time frame: [Through Study Completion, avg. 48 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Feb 2025.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ABC008 in people with Inclusion Body Myositis, run by Abcuro, Inc. at 4 sites in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 20 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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]). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Feb 2025.