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

• ClinicalTrials.gov Identifier: NCT05721573
• Recruitment Status: Recruiting
• First Posted: February 10, 2023
• Last Update Posted: June 5, 2023
• Sponsor: Abcuro, Inc.
• Collaborator: Syneos Health
• Information provided by (Responsible Party): Abcuro, Inc.

Study Description

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

Condition or disease	Intervention/treatment	Phase
Inclusion Body Myositis	Drug: ABC008	Phase 2; Phase 3

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 Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 231 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Masking Description: Double Blind
• Primary Purpose: Treatment
• 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
• Actual Study Start Date: February 28, 2023
• Estimated Primary Completion Date: November 2025
• Estimated Study Completion Date: December 2025

Arms and Interventions

Arm	Intervention/treatment
Active Comparator: 0.5 mg/kg ABC008; Part A - ABC008 N=12 Part B - ABC008 N= 67	Drug: ABC008; Given by subcutaneous injection
Active Comparator: 2.0 mg/kg ABC008; Part A - ABC008 N=12 Part B - ABC008 N= 67	Drug: ABC008; Given by subcutaneous injection
Placebo Comparator: Placebo; Part A - Placebo N= 6 Part B - Placebo N= 67	Drug: ABC008; Given by subcutaneous injection

Outcome Measures

Primary Outcome Measures :

1. 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)
2. 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)

Secondary Outcome Measures :

1. 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.
2. 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
3. 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
4. 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
5. 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.
6. 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
7. 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.
8. 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.
9. 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.

Eligibility Criteria

• Ages Eligible for Study: 40 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

• Adult males and females age >40 years at the time of the first dose of study medication;
• Weight >40 and <115 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;

Contacts and Locations

Contacts

Contact: Armando Reyes; 1 617 865 5079; IBM-201_clinicaltrial@abcuro.com

Locations

United States, Arizona

Neuromuscular Research Center; Recruiting

Phoenix, Arizona, United States, 85028

Contact: Lucia Rodriguez 480-314-1007 Lrodriguez@nrcaz.com

Principal Investigator: Kumaraswamy Sivakumar, MD

United States, California

University of California Irvine Medical Center (UCIMC) - Amyotrophic Lateral Sclerosis (ALS) and Neuromuscular Center; Recruiting

Irvine, California, United States, 92868

Contact: Lauren Harris 714-509-2661 lnharri1@hs.uci.edu

Principal Investigator: Namita A Goyal, MD

Keck Hosptial of USC; Recruiting

Los Angeles, California, United States, 90033

Contact: Salma Akhter 323-442-6221 salma.akhter@med.usc.edu

Principal Investigator: Said Beydoun, MD

United States, Connecticut

Yale School of Medicine; Recruiting

New Haven, Connecticut, United States, 06519

Contact: Danielle Carlson 612-594-4080 danielle.carlson@yale.edu

Principal Investigator: Bhaskar Roy, MD

United States, Massachusetts

Neuromuscular Diagnostic Center - Massachusetts General Hospital; Recruiting

Boston, Massachusetts, United States, 02114

Contact: Raissa Rodrigues 508-808-2510 rnrodrigues@bwh.harvard.edu

Principal Investigator: Paloma Gonzalez-Perez, MD

Brigham and Womens Hospital; Recruiting

Boston, Massachusetts, United States, 021158

Contact: Anna Friedman 508-808-2510 afriedman12@bwh.harvard.edu

Principal Investigator: Anthony Amato, MD

United States, New York

Hospital for Special Surgery; Recruiting

New York, New York, United States, 10021

Contact: Aliza Bloostein 212-774-2123 bloosteina@hss.edu

Principal Investigator: David R Fernandez, MD

United States, Pennsylvania

University of Pennsylvania; Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contact: Daniel Corr 215-662-2692 daniel.corr@pennmedicine.upenn.edu

Principal Investigator: Colin Quinn, MD

United States, Texas

Austin Neuromuscular Center; Recruiting

Austin, Texas, United States, 78759

Contact: Lauren Guilbeau 512-920-0140 lauren@austinneuromuscle.com

Principal Investigator: Yessar M Hussain, MD

Texas Neurology; Recruiting

Dallas, Texas, United States, 75206

Contact: Hanan Al Halawani 214-827-3610 ext 374 halhalawani@texasneurology.com

Principal Investigator: Daragh Heitzman, MD

Nerve and Muscle Center of Texas; Recruiting

Houston, Texas, United States, 77030

Contact: Amy Megerle 713-795-0033 ext 14 houneuamy@msn.com

Principal Investigator: Aziz I Shaibani, MD

United States, Virginia

Virginia Commonwealth University; Recruiting

Henrico, Virginia, United States, 23233

Contact: Kimberly Claytor 804-828-0856 kimberly.claytor@vcuhealth.org

Principal Investigator: Kelly Gwathmey, MD

Sponsors and Collaborators

Abcuro, Inc.

Syneos Health

More Information

Additional Information:

Study Website 

• Responsible Party: Abcuro, Inc.
• ClinicalTrials.gov Identifier: NCT05721573
• Other Study ID Numbers: ABC008-IBM-201
• First Posted: February 10, 2023
• Last Update Posted: June 5, 2023
• Last Verified: June 2023

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

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

Keywords provided by Abcuro, Inc.:

Muscular Diseases; Inflammatory Myopathy; Myositis; Neuromuscular Diseases; Nervous System Disease

Additional relevant MeSH terms:

Myositis; Myositis, Inclusion Body; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases