Abstract Details

Title INSPIRE-IBM: An NIH-funded, two-year, multicenter, observational study in inclusion body myositis (IBM)

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) S29 - Preclinical and Observational Studies of Neuromuscular Diseases (1:24 PM-1:36 PM)

Poster/Presentation
Number 003

Background A huge unmet need in IBM research is the lack of long-term true prospective longitudinal data on disease behavior and progression and the influence of the various blood biomarkers (NT5c1A antibodies and highly differentiated T lymphocytes) on disease behavior and progression.

Objective

To describe the INSPIRE-IBM multicenter, observational study

Design/Methods A 13-center observational prospective study was planned involving 150 patients with sporadic IBM. The study was funded by NIAMS/NIH and is scheduled to screen its first patient in October 2021. Primary eligibility criteria are diagnosis of IBM, fulfilling the ENMC 2011 criteria for clinicopathologically defined or clinically defined IBM, within 10 years of onset.

Results Each subject will be seen at 5 times point over a 2 year period, and will have serial collection of disease related data, including physical exams, outcome measures (IBMFRS, sIFA, quantitative muscle strength and grip strength testing, timed get up and go, pulmonary function tests, and fall and mobility device assessment). A number of patient reported outcome measures will also be obtained. Serial collection for serum and PBMCs will be done to assess for NT5c1A antibodies, select cytokines, and detailed immunophenotyping for blood based lymphocytes. RNA and DNA collection for future analyses will be done. A subset of 40 patients will also undergo fresh open muscle biopsy, along with concurrent blood collection, to assess muscle pathology and correlate blood markers with markers in muscle tissue.

Conclusions The study will provide the largest data to date on the natural history of IBM and will for the first time assess disease behavior and progression in relation to various disease biomarkers. At the end of the study a large repository of detailed patient data, PBMC, DNA, RNA and muscle tissue from highly characterized and phenotyped IBM patients will be available to assist future studies and drug development.

Authors/Disclosures
Marie Wencel, CCRP (University of California, Irvine) PRESENTER	Ms. Wencel has nothing to disclose.
Namita Goyal, MD, FAAN (University of California, Irvine)	Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx. Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Annexon. Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Abcuro. Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Fulcrum. Dr. Goyal has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for CSL Behring. Dr. Goyal has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Argenx. The institution of Dr. Goyal has received research support from Fulcrum. The institution of Dr. Goyal has received research support from Annexon. The institution of Dr. Goyal has received research support from Healey Platform. The institution of Dr. Goyal has received research support from Medicinova. The institution of Dr. Goyal has received research support from Transposon. The institution of Dr. Goyal has received research support from Abcuro. The institution of Dr. Goyal has received research support from Calico. The institution of Dr. Goyal has received research support from Clene. The institution of Dr. Goyal has received research support from Janssen. The institution of Dr. Goyal has received research support from PepGen. The institution of Dr. Goyal has received research support from PTC. The institution of Dr. Goyal has received research support from Roche. The institution of Dr. Goyal has received research support from Sanofi. Dr. Goyal has received publishing royalties from a publication relating to health care.
Tahseen Mozaffar, MD, FAAN (University of California Irvine)	Dr. Mozaffar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion Pharmaceuticals. Dr. Mozaffar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Argenx. Dr. Mozaffar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sanofi-Genzyme. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amicus. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Modis Therapeutics. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UCB/ Ra Pharmaceuticals. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Maze Therapeutics. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Spark Therapeutics. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for National Institutes of Health. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Argenx. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as an officer or member of the Board of Directors for Neuromuscular Disease Foundation. The institution of Dr. Mozaffar has received research support from NIH. The institution of Dr. Mozaffar has received research support from Muscular Dystrophy Association. The institution of Dr. Mozaffar has received research support from Sanofi-Genzyme. The institution of Dr. Mozaffar has received research support from Argenx. The institution of Dr. Mozaffar has received research support from Amicus Therapeutics. The institution of Dr. Mozaffar has received research support from Spark Therapeutics. The institution of Dr. Mozaffar has received research support from Audentes Therapeutics. The institution of Dr. Mozaffar has received research support from Cartesian. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Study Section Member with NIH.
	No disclosure on file