A Study to Evaluate the Efficacy and Safety of ABC008 for Inclusion Body Myositis

• ClinicalTrials.gov Identifier: NCT05721573
• Recruitment Status: Active, not recruiting
• First Posted: February 10, 2023
• Last Update Posted: April 5, 2024
• Sponsor: Abcuro, Inc.
• Collaborator: Syneos Health
• Information provided by (Responsible Party): Abcuro, Inc.

Tracking Information

• First Submitted Date: February 1, 2023
• First Posted Date: February 10, 2023
• Last Update Posted Date: April 5, 2024
• Actual Study Start Date: February 28, 2023
• Estimated Primary Completion Date: November 2025 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: March 8, 2023)

• Part A - To determine the safety and tolerability of recurrent dosing of ABC008 in subjects with IBM at 2 SC dose levels. [ Time Frame: From Baseline (week 0) through week 20. ]
  Safety as assessed by the incidence, type and severity of Treatment Emergent Adverse Events (TEAEs)
• Part B - To determine the efficacy of ABC008 in IBM at two SC dose levels as measured by IBM Functional Rating Scale (IBMFRS) at Week (W)76 [ Time Frame: From Baseline (week 0) through study completion, an average of 76 weeks ]
  Mean change in IBM Functional Rating Scale (IBMFRS)

Original Primary Outcome Measures (submitted: February 1, 2023)

• Part A - To determine the safety and tolerability of recurrent dosing of ABC008 in subjects with IBM at 2 SC dose levels. [ Time Frame: From Baseline (week 0) through study completion on average of 80 weeks. ]
  Safety as assessed by the incidence, type and severity of Treatment Emergent Adverse Events (TEAEs)
• Part B - To determine the efficacy of ABC008 in IBM at two SC dose levels as measured by IBM Functional Rating Scale (IBMFRS) at Week (W)76 [ Time Frame: From Baseline (week 0) through study completion, an average of 76 weeks ]
  Mean change in IBM Functional Rating Scale (IBMFRS)

Current Secondary Outcome Measures (submitted: February 1, 2023)

• Part A - Treatment Emergent Serious Adverse Events (TEASAEs) [ Time Frame: From Baseline (Day 1) through study completion, an average of 80 weeks. ]
  Incidence, type and severity of TEASAEs.
• Part A - Treatment Emergent Adverse Events (TEAEs) onset within 24 hours of Study Medication Administration. [ Time Frame: From Baseline (Day 1) through study completion, an average of 80 weeks. ]
  Incidence, type, and severity of TEAEs with onset within 24 hours from the start of any of study medication administration
• Part A - Treatment Emergent Adverse Events leading to study medication or study discontinuation. [ Time Frame: From Baseline (Day 1) through study completion, an average of 80 weeks. ]
  Incidence of TEAEs leading to study medication or study discontinuation
• Part A - Clinically significant changes in standard laboratory parameters, vital signs, and ECGs [ Time Frame: From Baseline (Day 1) through study completion, an average of 80 weeks. ]
  Incidence of clinically significant changes in standard laboratory parameters, vital signs, and ECGs
• Part A - Adverse Events of Special Interest (AESI) [ Time Frame: From Baseline (Day 1) through study completion, an average of 80 weeks. ]
  Incidence of AESIs.
• Part B - Manual Muscle Test 12 (MMT 12) [ Time Frame: From Baseline (Day 1) through study completion, an average of 76 weeks. ]
  Mean change in MMT 12
• Part B - Hand Grip Dynamometry [ Time Frame: From Baseline (Day 1) through study completion, an average of 76 weeks. ]
  Mean change in hand grip strength by dynamometry.
• Part B - Quadriceps Dynamometry [ Time Frame: From Baseline (Day 1) through study completion, an average of 76 weeks. ]
  Mean change in quadriceps strength by dynamometry.
• Part B - Modified Timed Up and Go (mTUG) [ Time Frame: From Baseline (Day 1) through study completion, an average of 76 weeks. ]
  Mean change in mTUG.

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Study to Evaluate the Efficacy and Safety of ABC008 for Inclusion Body Myositis
• Official Title: A Phase II/III Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of ABC008 in the Treatment of Subjects With Inclusion Body Myositis
• Brief Summary: A Phase II/III Randomized, Double-blind, Placebo-controlled, Multicenter Study to Determine the Efficacy and Safety of ABC008 in the Treatment of Subjects with Inclusion Body Myositis

Detailed Description

A Phase II/III Randomized, Double-blind, Placebo-controlled, Multicenter Study to Determine the Efficacy and Safety of ABC008 in the Treatment of Subjects with Inclusion Body Myositis Detailed Description: A Phase II/III Randomized, Double-blind, Placebo-controlled, Multicenter Study to Determine the Efficacy and Safety of ABC008 in the Treatment of Subjects with Inclusion Body Myositis Detailed Description: This is a Phase II/III randomized, double-blind, placebo-controlled, parallel multicenter study with 3 parts.

The study will include a sentinel cohort (Part A) of 30 subjects who will receive first three doses of the study drug. Safety data from subjects in the sentinel cohorts will be evaluated by a Data and Safety Monitoring Board (DSMB) before further dosing of the sentinel cohort, as well as initiation of enrollment in the double-blind safety and efficacy cohort (Part B). After completion of Part A or Part B, subjects have the option of enrolling in an open-label long-term extension study or progressing to the pharmacodynamics (PD) recovery cohort (Part C), to evaluate the recovery of the depletion of killer cell lectin-like receptor G1 (KLRG1)+ cells after the end of treatment with ABC008.

Efficacy, safety, HRQoL, and HRU assessments will be conducted. Blood samples will be obtained to evaluate the serum PK, PD, and immunogenicity of ABC008 throughout the study.

• Study Type: Interventional
• Study Phase: Phase 2; Phase 3

Study Design

Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description:

Double Blind

Primary Purpose: Treatment

• Condition: Inclusion Body Myositis

Intervention

Drug: ABC008

Given by subcutaneous injection

Study Arms

• Active Comparator: 0.5 mg/kg ABC008

  Part A - ABC008 N=12

  Part B - ABC008 N= 67

  Intervention: Drug: ABC008
• Active Comparator: 2.0 mg/kg ABC008

  Part A - ABC008 N=12

  Part B - ABC008 N= 67

  Intervention: Drug: ABC008
• Placebo Comparator: Placebo

  Part A - Placebo N= 6

  Part B - Placebo N= 67

  Intervention: Drug: ABC008

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Active, not recruiting
• Estimated Enrollment (submitted: February 1, 2023): 231
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: December 2025
• Estimated Primary Completion Date: November 2025 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Adult males and females age >40 years at the time of the first dose of study medication;
• Weight >40 and <150 kg;
• Diagnosis of either clinico-pathologically defined IBM, clinically defined IBM, or probable IBM according to the European Neuromuscular Centre (ENMC) IBM 2011 research diagnostic criteria (Rose et al., 2013). Documented histopathology results must be available prior to Baseline (Day 1) to confirm eligibility;
• Able to arise from a chair (with armrests), with use of their arms but without support from another person or device (e.g., cane, walking stick), at Screening and Baseline (Day 1);
• Able to walk 3 meters, turn around, walk back to the chair, and sit down, with or without assistive device. Once arisen from the chair, subject may use any walking device but cannot be supported by another person, furniture, or a wall;

Exclusion Criteria:

• Any other form of myositis or myopathy other than IBM, e.g., metabolic or drug-induced myopathy, drug-induced myositis, anti-synthetase syndrome, polymyositis or dermatomyositis, cancer-associated myositis (myositis diagnosed within 3 years, either before or after), myositis in overlap with another autoimmune disease (e.g., systemic lupus, systemic sclerosis, rheumatoid arthritis), or muscular dystrophy;
• Any condition, e.g., severe degenerative arthritis with limited range of motion, which precludes the ability to quantitate muscle strength or perform functional assessments (e.g., mTUG), in the Investigator's opinion;.
• Presence of another autoimmune or autoinflammatory disease other than indication under study, e.g., rheumatoid arthritis, psoriatic arthritis, axial spondyloarthropathy, inflammatory bowel disease, systemic lupus erythematosus. Subjects with Sjogren's syndrome, T-cell large granular lymphocyte leukemia (T-LGLL), or well-controlled thyroid disease are permitted;

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 40 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Australia, Belgium, Canada, France, Germany, United Kingdom, United States

Administrative Information

• NCT Number: NCT05721573
• Other Study ID Numbers: ABC008-IBM-201
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Abcuro, Inc.
• Original Responsible Party: Same as current
• Current Study Sponsor: Abcuro, Inc.
• Original Study Sponsor: Same as current
• Collaborators: Syneos Health
• Investigators: Not Provided
• PRS Account: Abcuro, Inc.
• Verification Date: April 2024