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

February 18, 2025 updated by: Abcuro, Inc.

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).

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

19

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • Herston, Australia
        • Royal Brisbane
      • Perth, Australia
        • Perron Institute
      • Sydney, Australia
        • Royal North Shore Hospital
    • South Australia
      • Adelaide, South Australia, Australia, 5000
        • Royal Adelaide Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

40 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cohort D1
Single Dose 0.1 mg / kg ABC008
Drug: ABC008
ABC008
Experimental: Cohort D2
Single Dose 0.5 mg / kg ABC008
Drug: ABC008
ABC008
Experimental: Cohort D3
Single Dose 2.0 mg / kg ABC008
Drug: ABC008
ABC008
Experimental: Cohort D4
Single Dose 5.0 mg / kg ABC008
Drug: ABC008
ABC008
Experimental: Cohort D5
X.X mg / kg ABC008
Drug: ABC008
ABC008
Experimental: Cohort 6
Single 2.0 mg / kg ABC008
Drug: ABC008
ABC008
Experimental: MAD Phase Cohort 1
Multiple Dose 0.1 mg / kg ABC008 every 8 weeks
Drug: ABC008
ABC008
Experimental: MAD Phase Cohort 2
Multiple Dose 0.5 mg / kg ABC008 every 8 weeks
Drug: ABC008
ABC008
Experimental: MAD Phase Cohort 3
Multiple Dose 2.0 mg / kg ABC008 every 8 weeks
Drug: ABC008
ABC008

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
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 Measures

Outcome Measure
Measure Description
Time Frame
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
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
Assess the time to peak serum concentration (Tmax) of a single dose of ABC008
Day 1 and throughout the 24 weeks of follow up
Assessment of terminal half-life (t½)
Time Frame: Day 1
Assess the terminal half-life (t½) of ABC008
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
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
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
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
Assessment of apparent volume of distribution (Vz/F) of a single dose of ABC008
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
Characterize changes in KLRG1 expressing lymphocytes
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
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
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
[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
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
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
[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 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
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)
[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 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
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
[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
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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Abcuro, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 25, 2021

Primary Completion (Actual)

January 10, 2025

Study Completion (Actual)

January 10, 2025

Study Registration Dates

First Submitted

November 25, 2020

First Submitted That Met QC Criteria

December 2, 2020

First Posted (Actual)

December 9, 2020

Study Record Updates

Last Update Posted (Actual)

March 25, 2025

Last Update Submitted That Met QC Criteria

February 18, 2025

Last Verified

June 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ABC008-IBM-101

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Inclusion Body Myositis

Clinical Trials on ABC008

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Singapore

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe