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

Study Purpose

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

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Interventional
Eligible Ages 40 Years and Over
Gender All
More Inclusion & Exclusion 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;

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT05721573
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase 2/Phase 3
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Abcuro, Inc.
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

N/A
Principal Investigator Affiliation N/A
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Industry, Other
Overall Status Recruiting
Countries Australia, Belgium, Canada, France, Germany, United Kingdom, United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Inclusion Body Myositis
Study Website: View Trial Website
Additional Details

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.

Arms & Interventions

Arms

Active Comparator: 0.5 mg/kg ABC008

Part A - ABC008 N=12 Part B - ABC008 N= 67

Active Comparator: 2.0 mg/kg ABC008

Part A - ABC008 N=12 Part B - ABC008 N= 67

Placebo Comparator: Placebo

Part A - Placebo N= 6 Part B - Placebo N= 67

Interventions

Drug: - ABC008

Given by subcutaneous injection

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Neuromuscular Research Center, Phoenix, Arizona

Status

Recruiting

Address

Neuromuscular Research Center

Phoenix, Arizona, 85028

Site Contact

Lucia Rodriguez

[email protected]

480-314-1007

Irvine, California

Status

Recruiting

Address

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

Irvine, California, 92868

Site Contact

Christina Orizabal

[email protected]

714-456-8520

Keck Hosptial of USC, Los Angeles, California

Status

Recruiting

Address

Keck Hosptial of USC

Los Angeles, California, 90033

Site Contact

Salma Akhter

[email protected]

323-442-6221

UCLA Medical Center, Los Angeles, California

Status

Recruiting

Address

UCLA Medical Center

Los Angeles, California, 90095

Site Contact

Velazquez Denisse

[email protected]

310-825-3264

Stanford Neuroscience Medical Center, Palo Alto, California

Status

Recruiting

Address

Stanford Neuroscience Medical Center

Palo Alto, California, 94304

Site Contact

Emily Lien

[email protected]

650-407-7912

University of California, San Francisco, San Francisco, California

Status

Recruiting

Address

University of California, San Francisco

San Francisco, California, 94143

Site Contact

Hannah George

[email protected]

415-501-0671

Aurora, Colorado

Status

Recruiting

Address

University of Colorado Hospital Anschutz Outpatient Pavillion

Aurora, Colorado, 80045

Site Contact

Alexis Shepardson

[email protected]

303-724-7248

Yale School of Medicine, New Haven, Connecticut

Status

Recruiting

Address

Yale School of Medicine

New Haven, Connecticut, 06519

Site Contact

Danielle Carlson

[email protected]

612-594-4080

Mayo Clinic, Jacksonville, Florida

Status

Recruiting

Address

Mayo Clinic

Jacksonville, Florida, 32224

Site Contact

Megan Donahue

[email protected]

904-953-3647

Chicago, Illinois

Status

Recruiting

Address

Northwestern Memorial Hospital, Department of Neurology (Clinic)

Chicago, Illinois, 60611

Site Contact

Emma Schmidt

[email protected]

312-503-4362

University of Kansas Medical, Kansas City, Kansas

Status

Recruiting

Address

University of Kansas Medical

Kansas City, Kansas, 66160

Site Contact

Ali Russo

[email protected]

913-588-5000

Johns Hopkins Bayview Medical Center, Baltimore, Maryland

Status

Recruiting

Address

Johns Hopkins Bayview Medical Center

Baltimore, Maryland, 21224

Site Contact

Grazyna Purwin

[email protected]

410-550-3609

Baltimore, Maryland

Status

Recruiting

Address

Johns Hopkins University School of Medicine

Baltimore, Maryland, 21287

Site Contact

Simone Thomas

[email protected]

410-614-4188

Boston, Massachusetts

Status

Recruiting

Address

Neuromuscular Diagnostic Center - Massachusetts General Hospital

Boston, Massachusetts, 02114

Site Contact

Raissa Rodrigues

[email protected]

857-249-0620

Brigham and Womens Hospital, Boston, Massachusetts

Status

Recruiting

Address

Brigham and Womens Hospital

Boston, Massachusetts, 021158

Site Contact

Vivian Hua

[email protected]

617-525-6497

Mayo Clinic, Rochester, Minnesota

Status

Recruiting

Address

Mayo Clinic

Rochester, Minnesota, 55905

Site Contact

Jane Sultze

[email protected]

507-538-5523

Washington University School of Medicine, Saint Louis, Missouri

Status

Recruiting

Address

Washington University School of Medicine

Saint Louis, Missouri, 63110

Site Contact

Kristy Conlon

[email protected]

314-362-3681

University of Nebraska Medical Center, Omaha, Nebraska

Status

Recruiting

Address

University of Nebraska Medical Center

Omaha, Nebraska, 98198

Site Contact

Sheryl Houston

[email protected]

402-552-6233

Hospital for Special Surgery, New York, New York

Status

Recruiting

Address

Hospital for Special Surgery

New York, New York, 10021

Site Contact

Ryan Heise

[email protected]

212-774-2123

New York, New York

Status

Recruiting

Address

Columbia University Medical Center / The Neurological Institute of New York

New York, New York, 10032

Site Contact

Raisy Fayerman

[email protected]

212-305-6035

Duke Neurological Disorders Clinic -1L, Durham, North Carolina

Status

Recruiting

Address

Duke Neurological Disorders Clinic -1L

Durham, North Carolina, 27710

Site Contact

Tabitha Karatz

[email protected]

919-684-0083

Wake Forrest School of Medicine, Winston-Salem, North Carolina

Status

Recruiting

Address

Wake Forrest School of Medicine

Winston-Salem, North Carolina, 27157

Site Contact

Gina Miller

[email protected]

1 617 865 5079

Cleveland, Ohio

Status

Recruiting

Address

University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106

Site Contact

Ashley Hawkins

[email protected]

216-844-2327

Columbus, Ohio

Status

Recruiting

Address

The Ohio State University Wexner Medical Center

Columbus, Ohio, 43210

Site Contact

Kaci Eubanks

[email protected]

614-366-3427

Oregon Health & Science University, Portland, Oregon

Status

Recruiting

Address

Oregon Health & Science University

Portland, Oregon, 97239

Site Contact

Sonya Boyd

[email protected]

541-602-2287

Hershey, Pennsylvania

Status

Recruiting

Address

Penn State Health Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033

Site Contact

Heidi Runk

[email protected]

717-531-0003 #287177

University of Pennsylvania, Philadelphia, Pennsylvania

Status

Recruiting

Address

University of Pennsylvania

Philadelphia, Pennsylvania, 19104

Site Contact

Daniel Corr

[email protected]

215-662-2692

Pittsburgh, Pennsylvania

Status

Recruiting

Address

UPMC Arthritis and Autoimmunity Center, Falk Clinic

Pittsburgh, Pennsylvania, 15213

Site Contact

Carol Oriss

[email protected]

1 617 865 5079

Austin Neuromuscular Center, Austin, Texas

Status

Recruiting

Address

Austin Neuromuscular Center

Austin, Texas, 78759

Site Contact

Lauren Guilbeau

[email protected]

512-920-0140

Texas Neurology, Dallas, Texas

Status

Recruiting

Address

Texas Neurology

Dallas, Texas, 75206

Site Contact

Hanan Al Halawani

[email protected]

214-827-3610 #374

Nerve and Muscle Center of Texas, Houston, Texas

Status

Recruiting

Address

Nerve and Muscle Center of Texas

Houston, Texas, 77030

Site Contact

Amy Megerle

[email protected]

713-795-0033 #14

Virginia Commonwealth University, Henrico, Virginia

Status

Active, not recruiting

Address

Virginia Commonwealth University

Henrico, Virginia, 23233

Seattle, Washington

Status

Recruiting

Address

University of Washington Medical Center - Montlake

Seattle, Washington, 98195

Site Contact

Dani Dixon

[email protected]

206-685-2028

Medical College of Wisconsin, Milwaukee, Wisconsin

Status

Recruiting

Address

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226

Site Contact

Marie Mejaki

[email protected]

414-955-0624

International Sites

Royal North Shore Hospital, Saint Leonards, New South Wales, Australia

Status

Recruiting

Address

Royal North Shore Hospital

Saint Leonards, New South Wales, 2065

Site Contact

Sharon Leaver

[email protected]

1 617 865 5079

Royal Brisbane and Women's Hospital, Herston, Queensland, Australia

Status

Recruiting

Address

Royal Brisbane and Women's Hospital

Herston, Queensland, 4006

Site Contact

Susan Heggie

[email protected]

61-7-3646-1478

Nedlands, Western Australia, Australia

Status

Recruiting

Address

Perron Institute for Neurological and Translational Science

Nedlands, Western Australia, 6009

Site Contact

Susan Walters

[email protected]

61 412 169 436

AZ Sint-Lucas & Volkskliniek, Gent, Belgium

Status

Recruiting

Address

AZ Sint-Lucas & Volkskliniek

Gent, , 9000

Site Contact

Kathy Dekoning

[email protected]

32 9 224 65 38

Calgary, Alberta, Canada

Status

Recruiting

Address

Heritage Medical Research Clinic - University of Calgary

Calgary, Alberta, 3M 1M4

Site Contact

Carissa Wong

[email protected]

403-210-3835

Genge Partners Inc., Montréal, Quebec, Canada

Status

Recruiting

Address

Genge Partners Inc.

Montréal, Quebec, H4A 3T2

Site Contact

Julian Santorelli

[email protected]

514-906-3876 #6026

Hospital Pitie-Salpetriere - AP-HP, Paris, France

Status

Recruiting

Address

Hospital Pitie-Salpetriere - AP-HP

Paris, , 75013

Site Contact

Bahareh Ghanizadeh

[email protected]

33142165863

Berlin, Germany

Status

Recruiting

Address

Krankenhaus und Poliklinik Rüdersdorf GmbH

Berlin, , 15562

Site Contact

Juliane Lorenz

[email protected]

0049 33638-8-3113

University Hosptial Duesseldorf, Düsseldorf, Germany

Status

Recruiting

Address

University Hosptial Duesseldorf

Düsseldorf, , 40225

Site Contact

Marcia Gasis

[email protected]

0049-211-8117889

London, United Kingdom

Status

Recruiting

Address

University College London Hospitals NHS Foundation Trust, National Hospital for Neurology and Neurosurgery (NHNN)

London, , WC1N 3BG

Site Contact

Louise Germain

[email protected]

00442031086308

Salford, United Kingdom

Status

Recruiting

Address

Salford Royal Hospital, Northern Care Alliance NHS Foundation Trust

Salford, , M6 8HD

Site Contact

Kathryn Slevin

[email protected]

0044161 206 6077