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

Abcuro

Status and phase: Completed; Phase 1

Conditions: Inclusion Body Myositis

Treatments: Drug: ABC008

Study type: Interventional

Funder types: Industry

Identifiers

NCT04659031

ABC008-IBM-101

Details and patient eligibility

About

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

Full 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.

Enrollment: 19 patients

Sex: All

Ages: 40+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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19 participants in 9 patient groups

Cohort D1

Experimental group

Description:

Single Dose 0.1 mg / kg ABC008

Treatment:

Drug: ABC008

Cohort D2

Experimental group

Description:

Single Dose 0.5 mg / kg ABC008

Treatment:

Drug: ABC008

Cohort D3

Experimental group

Description:

Single Dose 2.0 mg / kg ABC008

Treatment:

Drug: ABC008

Cohort D4

Experimental group

Description:

Single Dose 5.0 mg / kg ABC008

Treatment:

Drug: ABC008

Cohort D5

Experimental group

Description:

X.X mg / kg ABC008

Treatment:

Drug: ABC008

Cohort 6

Experimental group

Description:

Single 2.0 mg / kg ABC008

Treatment:

Drug: ABC008

MAD Phase Cohort 1

Experimental group

Description:

Multiple Dose 0.1 mg / kg ABC008 every 8 weeks

Treatment:

Drug: ABC008

MAD Phase Cohort 2

Experimental group

Description:

Multiple Dose 0.5 mg / kg ABC008 every 8 weeks

Treatment:

Drug: ABC008

MAD Phase Cohort 3

Experimental group

Description:

Multiple Dose 2.0 mg / kg ABC008 every 8 weeks

Treatment:

Drug: ABC008