Clinical trial NCT05046821: Sporadic Inclusion Body Myositis Natural History Study
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)
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Sporadic Inclusion Body Myositis
Inclusion Body Myositis
Lead sponsor: University of California, Irvine
Sponsor class: OTHER
Collaborators: name: National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
First posted: 2021-09-16
Enrollment: 150
Sex: ALL
Minimum age (years): 40
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Paticipants age 40 and older with sporadic IBM, diagnosed according to established criteria.

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.

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.

Eligibility 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.

Primary outcomes:
measure: Inclusion Body Myositis Functional Rating Scale (IBMFRS)
timeFrame: Change from Baseline to Month 24
measure: Timed Up and Go (TUG)
timeFrame: Change from Baseline to Month 24
measure: Frequency of immunophenotypic abnormalities in blood and muscle
timeFrame: Change from Baseline to Month 24
measure: Forced Vital Capacity (FVC) (sitting)
timeFrame: Change from Baseline to Month 24
Secondary outcomes:
measure: Manual Muscle Testing
timeFrame: Change from Baseline to Month 24
measure: Hand Held Dynamometry
timeFrame: Change from Baseline to Month 24
measure: Sydney Swallow Questionnaire
timeFrame: Change from Baseline to Month 24
measure: EAT-10 Questionnaire
timeFrame: Change from Baseline to Month 24
measure: NIH PROMIS questionnaires
timeFrame: Change from Baseline to Month 24
measure: Forced Vital Capacity (supine)
timeFrame: Change from Baseline to Month 24
measure: Maximum Inspiratory Pressure/Maximum Expiratory Pressure
timeFrame: Change from Baseline to Month 24
measure: sporadic inclusion body myositis physical functioning assessment (sIFA)
timeFrame: Change from Baseline to Month 24
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: University of California, Los Angeles
state: California
city: Orange
country: United States
facility: University of California, Irvine
state: California
city: Aurora
country: United States
facility: University of Colorado
state: Colorado
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Fairway
country: United States
facility: Kansas University Medical Center
state: Kansas
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
city: Boston
country: United States
facility: Brigham and Women's Hospital
state: Massachusetts
city: Saint Louis
country: United States
facility: Washington University in St. Louis
state: Missouri
city: Columbus
country: United States
facility: Ohio State University
state: Ohio
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: Houston
country: United States
facility: Nerve and Muscle Center of Texas
state: Texas
city: Seattle
country: United States
facility: University of Washington
state: Washington
Study officials:
affiliation: University of California, Irvine
name: Tahseen Mozaffar, MD FAAN, FANA
role: PRINCIPAL_INVESTIGATOR
References: