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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05046821
Recruitment Status : Recruiting
First Posted : September 16, 2021
Last Update Posted : November 24, 2021
Sponsor:
Collaborator:
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Information provided by (Responsible Party):
University of California, Irvine

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.

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Study Type : Observational
Estimated 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 : November 15, 2024
Estimated Study Completion Date : November 15, 2024





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


Information from the National Library of Medicine

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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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05046821


Contacts
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Contact: Marie Wencel, BS (714) 456-2525 mwencel@hs.uci.edu
Contact: Jennifer Avelar (714) 506-2665 jbavelar@hs.uci.edu

Locations
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United States, California
University of California, Los Angeles Not yet recruiting
Los Angeles, California, United States, 90095
Contact: Denisse Velazquez       denissevelazquez@mednet.ucla.edu   
Principal Investigator: Perry Shieh, MD, PhD         
University of California, Irvine Recruiting
Orange, California, United States, 92868
Contact: Marie Wencel, BS    714-456-2525    mwencel@hs.uci.edu   
Principal Investigator: Namita Goyal, MD         
United States, Colorado
University of Colorado Not yet recruiting
Aurora, Colorado, United States, 80045
Contact: Diana Santacruz       diana.santacruz@cuanschutz.edu   
Principal Investigator: Matthew Wicklund, MD         
United States, Kansas
Kansas University Medical Center Recruiting
Fairway, Kansas, United States, 66205
Contact: Lillian Saavedra    913-945-9937    lsaavedra2@kumc.edu   
Principal Investigator: Mazen Dimachkie, MD         
United States, Maryland
Johns Hopkins University Not yet recruiting
Baltimore, Maryland, United States, 21287
Contact: Michaela Pletsch    410-614-4188    mpletsc2@jhu.edu   
Principal Investigator: Thomas E Lloyd, MD, PhD         
United States, Massachusetts
Brigham and Women's Hospital Not yet recruiting
Boston, Massachusetts, United States, 02115
Contact: Janet Orozco    617-834-6135    jorozco1@bwh.harvard.edu   
Principal Investigator: Anthony A Amato, MD         
United States, Missouri
Washington University in St. Louis Not yet recruiting
Saint Louis, Missouri, United States, 63110
Contact: Michelle Seiffert    314-273-7946    mseiffert@wustl.edu   
Principal Investigator: Conrad C Weihl, MD, PhD         
United States, Ohio
Ohio State University Recruiting
Columbus, Ohio, United States, 43221
Contact: Amy Bartlett       Amy.bartlett@osumc.edu   
Principal Investigator: Miriam Freimer, MD         
United States, Oregon
Oregon Health & Science University Not yet recruiting
Portland, Oregon, United States, 97239
Contact: Katie Norris    503-494-7394    norriska@ohsu.edu   
Principal Investigator: Nizar Chahin, MD         
United States, Pennsylvania
University of Pennsylvania Not yet recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Daniel Corr Moulton    267-593-9459    dancorr20@gmail.com   
Principal Investigator: Colin Quinn, MD         
United States, Texas
University of Texas Southwestern Not yet recruiting
Dallas, Texas, United States, 75390
Contact: Amy Conger    214-648-0214    amy.conger@utsouthwestern.edu   
Principal Investigator: Salman Bhai, MD         
Nerve and Muscle Center of Texas Not yet recruiting
Houston, Texas, United States, 77030
Contact: Kim Anyaegbu    843-416-8026    houneukim@msn.com   
Principal Investigator: Aziz Shaibani, MD         
United States, Washington
University of Washington Not yet recruiting
Seattle, Washington, United States, 98195
Contact: Corrie Moreau    206-685-2028    cmorea2@uw.edu   
Principal Investigator: Leo H Wang, MD, PhD         
Sponsors and Collaborators
University of California, Irvine
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Investigators
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Principal Investigator: Tahseen Mozaffar, MD FAAN, FANA University of California, Irvine
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Responsible Party: University of California, Irvine
ClinicalTrials.gov Identifier: NCT05046821    
Other Study ID Numbers: HS# 2021-6779
R01AR078340-01 ( U.S. NIH Grant/Contract )
First Posted: September 16, 2021    Key Record Dates
Last Update Posted: November 24, 2021
Last Verified: November 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Myositis
Myositis, Inclusion Body
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases