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

Abcuro, Inc.19 enrolled

Overview

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

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

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Inclusion Body Myositis

Interventions

ABC008DRUG

ABC008

Eligibility

Sex: ALLMin age: 40 Years

Medical Language ↔ Plain English

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

Locations (4)

Royal Adelaide Hospital

Adelaide, South Australia, Australia

Royal Brisbane

Herston, Australia

Perron Institute

Perth, Australia

Royal North Shore Hospital

Sydney, Australia

Outcomes

Primary Outcomes

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.

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 Outcomes

Assessment of peak serum concentration (Cmax)

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

Time frame: 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

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

Assessment of terminal half-life (t½)

Assess the terminal half-life (t½) of ABC008

Time frame: 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)

Time frame: Day 1

Assessment of apparent clearance (CL/F)

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

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

Data from ClinicalTrials.gov

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Assessment of apparent volume of distribution (Vz/F)

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

Characterization of changes in KLRG1 expressing lymphocytes

Characterize changes in KLRG1 expressing lymphocytes

Time frame: 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