Influence of NT5C1A antibodies on disease progression, clinical phenotype and blood and muscle biomarkers in sporadic inclusion body myositis - a prospective evaluation

Active Project Grant Awarded Last action

Award ID
R01AR078340
Recipient
Awarding agency
Obligated
$13,884,199

Award facts

Completion date 93% complete
Originally
Funding
  • $11,585,227.00 Federal
  • $2,298,972.00 Non-federal
Place of performance
Orange, CA 92868, USA

Award details come from USAspending.gov.

Description

As the agency reported it.

Abstract
Sporadic inclusion body myositis (sibm) is a rare disorder of aging americans, causing asymmetric muscle weakness and severe disability and morbidity. IT is currently untreatable, and poorly understood. The prevalence of sibm is likely to increase as the proportion of the united states population above the age of 65 years continues to grow. A major barrier to clinical trials in sibm has been the lack of full understanding of the natural history of the disease. IT remains to be determined whether the rates of disease progression is uniform and whether the various biomarkers associated with sibm (anti-NT5C1A antibodies, variant t-cell populations) influence the natural history and disease behavior. Given the slow rate of disease progression, such observations cannot be made in the context of a routine clinical trial, and such studies need to be done as a separate stand-alone observational study. To address these unmet needs, we propose a prospective study with four specific aims. Aim 1: to determine for the first time whether C1A antibodies mediate disease progression over a two year interval in patients with sibm. 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. The significance of our proposed study is 1) to allow for a detailed characterization of the disease progression in sibm over a two-year period, and 2) to explore the relationship of a number of biomarkers associated with sibm, and their influence on disease behavior and disease progression. Upon completion of these aims, we will 1) understand the disease phenotype, including pattern of respiratory involvement, and disease progression in sibm better and understand the influence of serum antibodies to NT5C1A antibodies on the natural history and disease behavior; 2) define differences in serum variant t-cells and cytokine signatures in sibm patients and their influence on disease progression and behavior; and 3) understand muscle pathology and immune cell distribution in sibm patients and its relationship to NT5C1A antibodies. These findings may influence future trial design in sibm. Finally, we will have created a thirteen-site consortium of myositis treatment centers that will be ready to adopt quickly any future clinical trials aimed at changing the course of sibm.

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Subawards

Subrecipient Amount Awarded
The Johns Hopkins University No cost extension. The overall goal of this proposal is to perform a prospective natural history study on patients with clinically defined sIBM every 6 months… $0
The Regents Of The University Of Colorado Title: “Influence of NT5c1A antibodies on disease progression, clinical phenotype and blood and muscle biomarkers in sporadic Inclusion Body Myositis – A… $0
The Ohio State University The overall goal of this proposal is to perform a prospective natural history study on patients with clinically defined sIBM every 6 months over two years. To… $0
Brigham & Womens Hospital Inc. $0
University Of Miami The overall goal of this proposal is to perform a prospective natural history study on patients with clinically defined sIBM every 6 months over two years. To… $0
University Of Kansas Medical Center Research Institute, Inc. The overall goal of this proposal is to perform a prospective natural history study on patients with clinically defined sIBM every 6 months over two years. To… $0
The Washington University The overall goal of this proposal is to perform a prospective natural history study on patients with clinically defined sIBM every 6 months over two years. To… $0
The Trustees Of The University Of Pennsylvania The overall goal of this proposal is to perform a prospective natural history study on patients with clinically defined sIBM every 6 months over two years. To… $0
Oregon Health & Science University The overall goal of this proposal is to perform a prospective natural history study on patients with clinically defined sIBM every 6 months over two years. To… $0
University Of Rochester The University of Rochester's Clinical Trials Coordinating Center (CTCC) will act as the data coordinating center for this project. The CTCC has supported… $0
University Of Missouri System The overall goal of this proposal is to perform a prospective natural history study on patients with clinically defined sIBM every 6 months over two years. To… $7,043
University Of Rochester CTCC will act as the data coordinating center for this project. Initially, CTCC will help UCI study team develop the case report forms. $59,706
The Washington University Blood will be collected at three visits. Chemistry, CK, and serology tests will be performed at the site’s local laboratory. The Washington University in St.… $7,088
University Of Kansas Medical Center Research Institute, Inc. The overall goal of this proposal is to perform a prospective natural history study on patients with clinically defined sIBM every 6 months over two years. To… $26,824
The Johns Hopkins University The overall goal of this proposal is to perform a prospective natural history study on patients with clinically defined sIBM every 6 months over two years. To… $9,982
University Of Washington The overall goal of this proposal is to perform a prospective natural history study on patients with clinically defined sIBM every 6 months over two years. To… $10,030
Houston Neurocare, P.a. Title: “Influence of NT5c1A antibodies on disease progression, clinical phenotype and blood and muscle biomarkers in sporadic Inclusion Body Myositis… $7,524
The Trustees Of The University Of Pennsylvania Title: “Influence of NT5c1A antibodies on disease progression, clinical phenotype and blood and muscle biomarkers in sporadic Inclusion Body Myositis… $10,481
The Washington University The overall goal of this proposal is to perform a prospective natural history study on patients with clinically defined sIBM every 6 months over two years. To… $18,081
University Of Rochester The University of Rochester’s Clinical Trials Coordinating Center (CTCC) will act as the data coordinating center for this project. The CTCC has… $39,128
Oregon Health & Science University Scope of Work/Research Plan Summary For each of the participating sites (Main study and Muscle biopsy sub-study) Title: “Influence of NT5c1A antibodies on… $0
The Ohio State University Scope of Work/Research Plan Summary For each of the participating sites (Main study and Muscle biopsy sub-study) Title: “Influence of NT5c1A antibodies on… $10,062
Brigham & Womens Hospital Inc. The overall goal of this proposal is to perform a prospective natural history study on patients with clinically defined sIBM every 6 months over two years. To… -$20,568
The Johns Hopkins University The overall goal of this proposal is to perform a prospective natural history study on patients with clinically defined sIBM every 6 months over two years. To… $10,000
The Regents Of The University Of Colorado The overall goal of this proposal is to perform a prospective natural history study on patients with clinically defined sIBM every 6 months over two years. To… -$22,740
The Johns Hopkins University n'P_oveiall.qpalo.|_thisp.r_op_osal is.t_oj>efformApfpSj^vej?a«ujaL!XLSiO!YA«.Mgy_on PJlLQjl'.sjyil.ll.ç.[lDica.llY_ $10,664
The Trustees Of The University Of Pennsylvania Title: “Influence of NT5c1 A antibodies on disease progression, clinical phenotype and blood and muscle biomarkers in sporadic Inclusion Body Myositis - A… $0
Oregon Health & Science University Scope of Work/Research Plan Summary For each of the participating sites (Main study and Muscle biopsy sub-study) Title: “Influence of NT5c1A antibodies on… $0
The Ohio State University Scope of Work/Research Plan Summary For each of the participating sites (Main study and Muscle biopsy sub-study) Title: “Influence of NT5c1A antibodies on… $14,586
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Quick answers

Who received award R01AR078340?

R01AR078340 was awarded to University Of California Irvine by National Insititue of Arthritis and Musculoskeletal and Skin Diseases (National Institutes of Health) on April 1, 2021.

How much is award R01AR078340 worth?

$13,884,199 has been obligated on R01AR078340: $11,585,227 in federal funding and $2,298,972 in non-federal funding.

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