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

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Primary Purpose

Status

Recruiting

Phase

Phase 1

Locations

Australia

Study Type

Interventional

Intervention

ABC008

About this trial

This is an interventional treatment trial for Inclusion Body Myositis focused on measuring Inclusion Body Myositis, IBM

Eligibility Criteria

40 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Royal Adelaide Hospital
Royal BrisbaneRecruiting
Perron InstituteRecruiting
Royal North Shore Hospital

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm 6

Arm 7

Arm 8

Arm 9

Arm Type

Experimental

Arm Label

Cohort D1

Cohort D2

Cohort D3

Cohort D4

Cohort D5

Cohort 6

MAD Phase Cohort 1

MAD Phase Cohort 2

MAD Phase Cohort 3

Arm Description

Single Dose 0.1 mg / kg ABC008

Single Dose 0.5 mg / kg ABC008

Single Dose 2.0 mg / kg ABC008

Single Dose 5.0 mg / kg ABC008

X.X mg / kg ABC008

Single 2.0 mg / kg ABC008

Multiple Dose 0.1 mg / kg ABC008 every 8 weeks

Multiple Dose 0.5 mg / kg ABC008 every 8 weeks

Multiple Dose 2.0 mg / kg ABC008 every 8 weeks

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Assessment of peak serum concentration (Cmax)

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

Assessment of time to peak serum concentration (Tmax)

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

Assessment of terminal half-life (t½)

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)

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)

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

Assessment of apparent volume of distribution (Vz/F)

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

Characterization of changes in KLRG1 expressing lymphocytes

Characterize changes in KLRG1 expressing lymphocytes

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

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

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

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

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)

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)

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

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

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)

Full Information

NCT ID

NCT04659031

First Posted

November 25, 2020

Last Updated

April 28, 2023

Sponsor

Abcuro, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT04659031

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

April 2023

Overall Recruitment Status

Recruiting

Study Start Date

May 25, 2021 (Actual)

Primary Completion Date

March 2024 (Anticipated)

Study Completion Date

May 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Abcuro, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

No

Studies a U.S. FDA-regulated Device Product

No

Data Monitoring Committee

No

5. Study Description

Brief Summary

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Inclusion Body Myositis

Keywords

Inclusion Body Myositis, IBM

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

30 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Cohort D1

Arm Type

Experimental

Arm Description

Single Dose 0.1 mg / kg ABC008

Arm Title

Cohort D2

Arm Type

Experimental

Arm Description

Single Dose 0.5 mg / kg ABC008

Arm Title

Cohort D3

Arm Type

Experimental

Arm Description

Single Dose 2.0 mg / kg ABC008

Arm Title

Cohort D4

Arm Type

Experimental

Arm Description

Single Dose 5.0 mg / kg ABC008

Arm Title

Cohort D5

Arm Type

Experimental

Arm Description

X.X mg / kg ABC008

Arm Title

Cohort 6

Arm Type

Experimental

Arm Description

Single 2.0 mg / kg ABC008

Arm Title

MAD Phase Cohort 1

Arm Type

Experimental

Arm Description

Multiple Dose 0.1 mg / kg ABC008 every 8 weeks

Arm Title

MAD Phase Cohort 2

Arm Type

Experimental

Arm Description

Multiple Dose 0.5 mg / kg ABC008 every 8 weeks

Arm Title

MAD Phase Cohort 3

Arm Type

Experimental

Arm Description

Multiple Dose 2.0 mg / kg ABC008 every 8 weeks

Intervention Type

Drug

Intervention Name(s)

ABC008

Intervention Description

ABC008

Primary Outcome Measure Information:

Title

Assessment of Safety and Tolerability

Description

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.

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]

Secondary Outcome Measure Information:

Title

Assessment of peak serum concentration (Cmax)

Description

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

Time Frame

Day 1 and throughout the 24 weeks of follow up

Title

Assessment of time to peak serum concentration (Tmax)

Description

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

Time Frame

Day 1 and throughout the 24 weeks of follow up

Title

Assessment of terminal half-life (t½)

Description

Assess the terminal half-life (t½) of ABC008

Time Frame

Day 1

Title

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

Description

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)

Time Frame

Day 1

Title

Assessment of apparent clearance (CL/F)

Description

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

Time Frame

Day 1 and throughout the 24 weeks of follow up

Title

Assessment of apparent volume of distribution (Vz/F)

Description

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

Time Frame

Day 1 and throughout the 24 weeks of follow up

Title

Characterization of changes in KLRG1 expressing lymphocytes

Description

Characterize changes in KLRG1 expressing lymphocytes

Time Frame

Day 1 and throughout the 24 weeks of follow up

Title

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

Description

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

Time Frame