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

• ClinicalTrials.gov Identifier: NCT05046821
• Recruitment Status: Active, not recruiting
• First Posted: September 16, 2021
• Last Update Posted: April 9, 2024
• Sponsor: University of California, Irvine
• Collaborator: National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
• Information provided by (Responsible Party): Tahseen Mozaffar, University of California, Irvine

Study Description

Brief Summary:

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.

Condition or disease
Sporadic Inclusion Body Myositis; 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 Design

• Study Type: Observational
• Actual Enrollment: 150 participants
• Observational Model: Case-Only
• Time Perspective: Prospective
• Official Title: 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)
• Actual Study Start Date: October 11, 2021
• Estimated Primary Completion Date: January 31, 2026
• Estimated Study Completion Date: January 31, 2026

Groups and Cohorts

Outcome Measures

Primary Outcome Measures :

1. Inclusion Body Myositis Functional Rating Scale (IBMFRS) [ Time Frame: Change from Baseline to Month 24 ]
   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.
2. Timed Up and Go (TUG) [ Time Frame: Change from Baseline to Month 24 ]
   Stand up from a chair, walk 3 meters, sit back down in chair
3. Frequency of immunophenotypic abnormalities in blood and muscle [ Time Frame: Change from Baseline to Month 24 ]
   Laboratory assessment
4. Forced Vital Capacity (FVC) (sitting) [ Time Frame: Change from Baseline to Month 24 ]
   breathing test

Secondary Outcome Measures :

1. Manual Muscle Testing [ Time Frame: Change from Baseline to Month 24 ]
   muscle strength testing
2. Hand Held Dynamometry [ Time Frame: Change from Baseline to Month 24 ]
   muscle strength testing
3. Sydney Swallow Questionnaire [ Time Frame: Change from Baseline to Month 24 ]
   swallow ability questionnaire
4. EAT-10 Questionnaire [ Time Frame: Change from Baseline to Month 24 ]
   swallow ability questionnaire
5. NIH PROMIS questionnaires [ Time Frame: Change from Baseline to Month 24 ]
   quality of life questionnaires
6. Forced Vital Capacity (supine) [ Time Frame: Change from Baseline to Month 24 ]
   breathing test done while lying down
7. Maximum Inspiratory Pressure/Maximum Expiratory Pressure [ Time Frame: Change from Baseline to Month 24 ]
   breathing tests
8. sporadic inclusion body myositis physical functioning assessment (sIFA) [ Time Frame: Change from Baseline to Month 24 ]
   functional ability questionnaire

Biospecimen Retention:   Samples With DNA

DNA and RNA will be isolated from blood and muscle samples and stored for future research

Eligibility Criteria

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

Study Population

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

Criteria

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.

Contacts and Locations

Locations

United States, California

University of California, Los Angeles

Los Angeles, California, United States, 90095

University of California, Irvine

Orange, California, United States, 92868

United States, Colorado

University of Colorado

Aurora, Colorado, United States, 80045

United States, Florida

University of Miami

Miami, Florida, United States, 33136

United States, Kansas

Kansas University Medical Center

Fairway, Kansas, United States, 66205

United States, Maryland

Johns Hopkins University

Baltimore, Maryland, United States, 21287

United States, Massachusetts

Brigham and Women's Hospital

Boston, Massachusetts, United States, 02115

United States, Missouri

Washington University in St. Louis

Saint Louis, Missouri, United States, 63110

United States, Ohio

Ohio State University

Columbus, Ohio, United States, 43221

United States, Oregon

Oregon Health & Science University

Portland, Oregon, United States, 97239

United States, Pennsylvania

University of Pennsylvania

Philadelphia, Pennsylvania, United States, 19104

United States, Texas

Nerve and Muscle Center of Texas

Houston, Texas, United States, 77030

United States, Washington

University of Washington

Seattle, Washington, United States, 98195

Sponsors and Collaborators

University of California, Irvine

National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

Investigators

• Principal Investigator: Tahseen Mozaffar, MD FAAN, FANA; University of California, Irvine

More Information

• Responsible Party: Tahseen Mozaffar, Professor of Neurology, University of California, Irvine
• ClinicalTrials.gov Identifier: NCT05046821
• Other Study ID Numbers: 20216779; R01AR078340-01 ( U.S. NIH Grant/Contract )
• First Posted: September 16, 2021
• Last Update Posted: April 9, 2024
• Last Verified: April 2024

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Undecided

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

Additional relevant MeSH terms:

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