Clinical trial NCT04659031: 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, Single and Multiple Ascending Dose Study of ABC008 in Adult Patients With Inclusion Body Myositis (IBM)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Inclusion Body Myositis
Keywords: Inclusion Body Myositis
IBM
Lead sponsor: Abcuro, Inc.
Sponsor class: INDUSTRY
First posted: 2020-12-09
Enrollment: 19
Sex: ALL
Minimum age (years): 40
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility 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

Primary outcomes:
measure: Assessment of Safety and Tolerability
timeFrame: 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:
measure: Assessment of peak serum concentration (Cmax)
timeFrame: Day 1 and throughout the 24 weeks of follow up
measure: Assessment of time to peak serum concentration (Tmax)
timeFrame: Day 1 and throughout the 24 weeks of follow up
measure: Assessment of terminal half-life (t½)
timeFrame: Day 1
measure: Assessment of area under the concentration versus time curve from time zero to 24 hours post-dose (AUC0-24hr)
timeFrame: Day 1
measure: Assessment of apparent clearance (CL/F)
timeFrame: Day 1 and throughout the 24 weeks of follow up
measure: Assessment of apparent volume of distribution (Vz/F)
timeFrame: Day 1 and throughout the 24 weeks of follow up
measure: Characterization of changes in KLRG1 expressing lymphocytes
timeFrame: Day 1 and throughout the 24 weeks of follow up
measure: Qualitative assessment of [ 89Zr]Zr-Df-crefmirlimab
timeFrame: [Through Study Completion, avg. 48 weeks
measure: Assessment of global distribution of [ 89Zr]Zr-Df-crefmirlimab uptake in skeletal muscle
timeFrame: [Through Study Completion, avg. 48 weeks
measure: Assessment of global distribution of [ 89Zr]Zr-Df-crefmirlimab uptake in lymphoid organs
timeFrame: [Through Study Completion, avg. 48 weeks
measure: Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab pre and post dosing of ABC008
timeFrame: [Through Study Completion, avg. 48 weeks
measure: Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab, determined by standardized uptake value (SUV)-based quantitative analysis peak (SUVpeak)
timeFrame: [Through Study Completion, avg. 48 weeks
measure: Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab, determined by standardized uptake value (SUV)-based quantitative analysis mean (SUVmean)
timeFrame: [Through Study Completion, avg. 48 weeks
measure: Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab, determined by standardized uptake value (SUV)-based quantitative analysis SUV of diseased muscle
timeFrame: [Through Study Completion, avg. 48 weeks
measure: Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab, determined by standardized uptake value (SUV)-based quantitative analysis SUV reference tissue
timeFrame: [Through Study Completion, avg. 48 weeks
measure: Quantitative assessment of [ 89Zr]Zr-Df-crefmirlimab, determined by standardized uptake value (SUV)-based quantitative analysis maximum (SUVmax)
timeFrame: [Through Study Completion, avg. 48 weeks
Other outcomes:
Locations:
city: Adelaide
country: Australia
facility: Royal Adelaide Hospital
state: South Australia
city: Herston
country: Australia
facility: Royal Brisbane
city: Perth
country: Australia
facility: Perron Institute
city: Sydney
country: Australia
facility: Royal North Shore Hospital
Study officials:
References: