Sporadic Inclusion Body Myositis Natural History Study (INSPIRE-IBM)

Influence of NT5c1A Antibodies on Disease Progression, Clinical Phenotype and Blood and Muscle Biomarkers in Sporadic Inclusion Body Myositis - A Prospective Evaluation (INSPIRE-IBM Study)

This is a prospective natural history study on patients with clinically defined sIBM. Participants will be assessed every 6 months over two years (five visits total). We will include 150 participants, enrolled across 13 sites, with sporadic IBM, diagnosed according to established criteria.

Study Overview

• Status: Active, not recruiting
• Conditions: Inclusion Body Myositis; Sporadic Inclusion Body Myositis

Detailed Description

This is a prospective natural history study on patients with clinically defined sIBM. Participants will be assessed every 6 months over two years (five visits total). We will include 150 participants with sporadic IBM, diagnosed according to established criteria. Participants will require an routine serum sample via blood draw to evaluate for NT5c1A antibody status. This testing will be performed at Washington University School of Medicine in the Neuromuscular Laboratory. Investigators will be blinded to antibody status. A subset of participants (40) will undergo a muscle biopsy at the Baseline visit.

Aim 1. To determine for the first time whether NT5c1A antibodies mediate disease progression over a two-year interval in patients with sIBM.

We will perform a prospective, non-interventional, observational study on patients with sIBM with follow up and evaluations every 6 months over a two-year time frame. Primary analyses are: 1) the rates of disease progression and severity as measured by rates of decline in IBM Functional Rating Scale (IBMFRS) score and Timed Get Up and Go (TUG); 2) the presence or absence of serum antibodies to NT5c1A; and 3) the presence and frequency of variant T-cells in the serum and skeletal muscle.

Aim 2. To perform a detailed morphological, histochemical and immunohistochemical analysis of fresh muscle biopsy specimens obtained from a subset of patients with sIBM.

Aim 3. To characterize the distribution of "immunosenescent" lymphocytes in circulating blood from patients with sIBM.

Aim 4. To quantify the decline in the respiratory function of sIBM patients.

• Study Type: Observational
• Enrollment (Actual): 150

Contacts and Locations

Study Locations

• United States
  • California
    • Los Angeles, California, United States, 90095
      • University of California, Los Angeles
    • Orange, California, United States, 92868
      • University of California, Irvine
  • Colorado
    • Aurora, Colorado, United States, 80045
      • University of Colorado
  • Florida
    • Miami, Florida, United States, 33136
      • University of Miami
  • Kansas
    • Fairway, Kansas, United States, 66205
      • Kansas University Medical Center
  • Maryland
    • Baltimore, Maryland, United States, 21287
      • Johns Hopkins University
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Brigham and Women's Hospital
  • Missouri
    • Saint Louis, Missouri, United States, 63110
      • Washington University in St. Louis
  • Ohio
    • Columbus, Ohio, United States, 43221
      • Ohio State University
  • Oregon
    • Portland, Oregon, United States, 97239
      • Oregon Health & Science University
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • University of Pennsylvania
  • Texas
    • Houston, Texas, United States, 77030
      • Nerve and Muscle Center of Texas
  • Washington
    • Seattle, Washington, United States, 98195
      • University of Washington

Participation Criteria

Eligibility Criteria

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

• Sampling Method: Probability Sample

Study Population

Paticipants age 40 and older with sporadic IBM, diagnosed according to established criteria.

Description

Inclusion Criteria:

1. Ages 40 years and older
2. Fulfills ENMC 2011 criteria of clinically definite or probable sporadic Inclusion body myositis (sIBM)
3. Disease onset is within the past 10 years of the time of Baseline visit
4. Able to participate and comply with study related procedures
5. Able to provide written consent

Exclusion Criteria:

1. Current or very recent use (within last 6 months of the Baseline visit) of immunomodulation or immunosuppression therapy.
2. Current or very recent use (within last 90 days of the Baseline visit) of an investigational medication or therapy.
3. Co-existing significant medical or surgical conditions that, in the opinion of the investigator, will influence study participation or alter natural history.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Inclusion Body Myositis Functional Rating Scale (IBMFRS)	The IBMFRS is a 10 item questionnaire. Scores for each item range from 0 to 4. There is a total maximum score of 40 and minimum score of 0. The higher the score the better functional status of the person.	Change from Baseline to Month 24
Timed Up and Go (TUG)	Stand up from a chair, walk 3 meters, sit back down in chair	Change from Baseline to Month 24
Frequency of immunophenotypic abnormalities in blood and muscle	Laboratory assessment	Change from Baseline to Month 24
Forced Vital Capacity (FVC) (sitting)	breathing test	Change from Baseline to Month 24

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Manual Muscle Testing	muscle strength testing	Change from Baseline to Month 24
Hand Held Dynamometry	muscle strength testing	Change from Baseline to Month 24
Sydney Swallow Questionnaire	swallow ability questionnaire	Change from Baseline to Month 24
EAT-10 Questionnaire	swallow ability questionnaire	Change from Baseline to Month 24
NIH PROMIS questionnaires	quality of life questionnaires	Change from Baseline to Month 24
Forced Vital Capacity (supine)	breathing test done while lying down	Change from Baseline to Month 24
Maximum Inspiratory Pressure/Maximum Expiratory Pressure	breathing tests	Change from Baseline to Month 24
sporadic inclusion body myositis physical functioning assessment (sIFA)	functional ability questionnaire	Change from Baseline to Month 24

Collaborators and Investigators

• Sponsor: University of California, Irvine
• Collaborators: National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
• Investigators: Principal Investigator: Tahseen Mozaffar, MD FAAN, FANA, University of California, Irvine

Study record dates

Study Major Dates

• Study Start (Actual): October 11, 2021
• Primary Completion (Estimated): January 31, 2026
• Study Completion (Estimated): January 31, 2026

Study Registration Dates

• First Submitted: September 8, 2021
• First Submitted That Met QC Criteria: September 8, 2021
• First Posted (Actual): September 16, 2021

Study Record Updates

• Last Update Posted (Actual): April 9, 2024
• Last Update Submitted That Met QC Criteria: April 6, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Myositis; Myositis, Inclusion Body
• Other Study ID Numbers: 20216779; R01AR078340-01 (U.S. NIH Grant/Contract)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No