NCT04659031

Brief Summary

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

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
19

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started May 2021

Typical duration for phase_1

Geographic Reach
1 country

4 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 25, 2020

Completed
14 days until next milestone

First Posted

Study publicly available on registry

December 9, 2020

Completed
6 months until next milestone

Study Start

First participant enrolled

May 25, 2021

Completed
3.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 10, 2025

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 10, 2025

Completed
Last Updated

February 20, 2025

Status Verified

June 1, 2024

Enrollment Period

3.6 years

First QC Date

November 25, 2020

Last Update Submit

February 18, 2025

Conditions

Inclusion Body Myositis

Keywords

Inclusion Body MyositisIBM

Outcome Measures

Primary Outcomes (1)

  • 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.

    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 (16)

  • Assessment of peak serum concentration (Cmax)

    Day 1 and throughout the 24 weeks of follow up

  • Assessment of time to peak serum concentration (Tmax)

    Day 1 and throughout the 24 weeks of follow up

  • Assessment of terminal half-life (t½)

    Day 1

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

    Day 1

  • Assessment of apparent clearance (CL/F)

    Day 1 and throughout the 24 weeks of follow up

  • +11 more secondary outcomes

Study Arms (9)

Cohort D1

EXPERIMENTAL

Single Dose 0.1 mg / kg ABC008

Drug: ABC008

Cohort D2

EXPERIMENTAL

Single Dose 0.5 mg / kg ABC008

Drug: ABC008

Cohort D3

EXPERIMENTAL

Single Dose 2.0 mg / kg ABC008

Drug: ABC008

Cohort D4

EXPERIMENTAL

Single Dose 5.0 mg / kg ABC008

Drug: ABC008

Cohort D5

EXPERIMENTAL

X.X mg / kg ABC008

Drug: ABC008

Cohort 6

EXPERIMENTAL

Single 2.0 mg / kg ABC008

Drug: ABC008

MAD Phase Cohort 1

EXPERIMENTAL

Multiple Dose 0.1 mg / kg ABC008 every 8 weeks

Drug: ABC008

MAD Phase Cohort 2

EXPERIMENTAL

Multiple Dose 0.5 mg / kg ABC008 every 8 weeks

Drug: ABC008

MAD Phase Cohort 3

EXPERIMENTAL

Multiple Dose 2.0 mg / kg ABC008 every 8 weeks

Drug: ABC008

Interventions

ABC008DRUG

ABC008

Cohort 6Cohort D1Cohort D2Cohort D3Cohort D4Cohort D5MAD Phase Cohort 1MAD Phase Cohort 2MAD Phase Cohort 3

Eligibility Criteria

Age40 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Royal Adelaide Hospital

Adelaide, South Australia, 5000, Australia

Location

Royal Brisbane

Herston, Australia

Location

Perron Institute

Perth, Australia

Location

Royal North Shore Hospital

Sydney, Australia

Location

MeSH Terms

Conditions

Myositis, Inclusion Body

Condition Hierarchy (Ancestors)

MyositisMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System Diseases

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 25, 2020

First Posted

December 9, 2020

Study Start

May 25, 2021

Primary Completion

January 10, 2025

Study Completion

January 10, 2025

Last Updated

February 20, 2025

Record last verified: 2024-06

Locations

AU(4)