Sporadic Inclusion Body Myositis Natural History Study

Inclusion Body Myositis Clinical Trial

— INSPIRE-IBM  

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)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05046821
• Other study ID #: 20216779
• Secondary ID: R01AR078340-01
• Status: Active, not recruiting
• Start date: October 11, 2021
• Est. completion date: January 31, 2026

Study information

• Verified date: April 2024
• Source: University of California, Irvine
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 150
• Est. completion date: January 31, 2026
• Est. primary completion date: January 31, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 40 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Inclusion Body Myositis
• Myositis
• Myositis, Inclusion Body
• Sporadic Inclusion Body Myositis

Locations

Country	Name	City	State
United States	University of Colorado	Aurora	Colorado
United States	Johns Hopkins University	Baltimore	Maryland
United States	Brigham and Women's Hospital	Boston	Massachusetts
United States	Ohio State University	Columbus	Ohio
United States	Kansas University Medical Center	Fairway	Kansas
United States	Nerve and Muscle Center of Texas	Houston	Texas
United States	University of California, Los Angeles	Los Angeles	California
United States	University of Miami	Miami	Florida
United States	University of California, Irvine	Orange	California
United States	University of Pennsylvania	Philadelphia	Pennsylvania
United States	Oregon Health & Science University	Portland	Oregon
United States	Washington University in St. Louis	Saint Louis	Missouri
United States	University of Washington	Seattle	Washington

Sponsors (2)

Lead Sponsor	Collaborator
University of California, Irvine	National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	Timed Up and Go (TUG)	Stand up from a chair, walk 3 meters, sit back down in chair	Change from Baseline to Month 24
Primary	Frequency of immunophenotypic abnormalities in blood and muscle	Laboratory assessment	Change from Baseline to Month 24
Primary	Forced Vital Capacity (FVC) (sitting)	breathing test	Change from Baseline to Month 24
Secondary	Manual Muscle Testing	muscle strength testing	Change from Baseline to Month 24
Secondary	Hand Held Dynamometry	muscle strength testing	Change from Baseline to Month 24
Secondary	Sydney Swallow Questionnaire	swallow ability questionnaire	Change from Baseline to Month 24
Secondary	EAT-10 Questionnaire	swallow ability questionnaire	Change from Baseline to Month 24
Secondary	NIH PROMIS questionnaires	quality of life questionnaires	Change from Baseline to Month 24
Secondary	Forced Vital Capacity (supine)	breathing test done while lying down	Change from Baseline to Month 24
Secondary	Maximum Inspiratory Pressure/Maximum Expiratory Pressure	breathing tests	Change from Baseline to Month 24
Secondary	sporadic inclusion body myositis physical functioning assessment (sIFA)	functional ability questionnaire	Change from Baseline to Month 24