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Abstract Number: 0441

A Randomized, Double-blind, Placebo-controlled Study of Arimoclomol in Patients with Inclusion Body Myositis

Pedro Machado1, Richard Barohn2, Michael McDermott3, Thomas Blaetter4, Tom Lloyd5, Aziz Shaibani6, Miriam Freimer7, Anthony Amato8, Emma Ciafaloni3, Sarah Jones9, Tahseen Mozaffar10, Summer Gibson11, Matthew Wicklund12, Todd Levine13, Claus Sundgreen4, Tim Carstensen4, Karen Bonefeld4, Anders Jørgensen4, Karina Phonekeo4, Andrew Heim14, Laura Herbelin14, Michael Hanna15 and Mazen Dimachkie14, 1Centre for Rheumatology & Department of Neuromuscular Diseases, University College London, London, United Kingdom, 2University of Missouri - Columbia, Columbia, MO, 3University of Rochester, Rochester, NY, 4Orphazyme A/S, Copenhagen, Denmark, 5Johns Hopkins University, Baltimore, MD, 6Nerve and Musle Center, Houston, TX, 7Ohio State University, Columbus, OH, 8Brigham and Women's Hospital, Boston, MA, 9University of Virginia, Charlottesville, VA, 10University of California Irvine, Irvine, CA, 11University of Utah, Salt Lake City, UT, 12University of Colorado, Denver, CO, 13Phoenix Neurological Associates, Phoenix, AZ, 14University of Kansas Medical Center, Kansas City, KS, 15University College London, London, United Kingdom

Meeting: ACR Convergence 2021

Keywords: clinical trial, Myopathies, Myositis, Outcome measures, Randomized Trial

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Session Information

Date: Saturday, November 6, 2021

Session Title: Abstracts: Muscle Biology, Myositis & Myopathies (0441–0444)

Session Type: Abstract Session

Session Time: 9:00AM-9:15AM

Background/Purpose: Inclusion body myositis (IBM) is the most common idiopathic inflammatory myopathy occurring in patients over the age of 45 years. Since immune suppression has not been effective, modulating the cytoprotective “heat shock response” (HSR) represents a candidate therapeutic approach targeting both inflammation and degeneration. In a pilot study, arimoclomol, an amplifier of the HSR, was safe and well tolerated with some trends suggesting efficacy at 8 months in subjects with IBM. Our aim is to present the efficacy and safety/tolerability data from a phase 2/3 randomized controlled trial of arimoclomol in IBM (NCT02753530).

Methods: In this international multicenter, double-blind, placebo-controlled trial, subjects were randomized (1:1) to receive either arimoclomol citrate 400 mg or matching placebo capsules three times a day (1,200 mg/day) for 20 months. The primary outcome measure was the change from baseline to Month 20 in the IBM Functional Rating Scale (IBMFRS) total score. Hierarchically ordered key secondary outcome measures included hand grip strength (strongest hand), Modified Time Up and Go, Manual Muscle Testing (24 muscles), 6-minute walk test distance, and the Short-Form 36 health survey. Other outcome measures included patient and clinician impressions, and other measures of muscle strength and function. Drug safety and tolerability were evaluated.

Results: One hundred fifty-two IBM subjects fulfilling ENMC 2011 criteria were randomized with mean age 67.2 years (SD 8.1), mostly men (76%), mean disease duration 98 months (SD 58), and mean baseline IBMFRS of 27.4 (SD 4.6). The IBMFRS declined by a mean of 3.25 points with arimoclomol vs. 2.26 points with placebo over 20 months (p=0.11). Secondary efficacy outcome measures did not show any statistically significant treatment group differences. Most frequently reported AEs observed with higher incidence in arimoclomol group were gastrointestinal disorders (54.8% vs. 39.7%). Patients receiving arimoclomol were more likely to discontinue treatment due to AEs (17.8% vs. 5.1%). The relative frequency of serious AEs was comparable in the two treatment arms (arimoclomol 15.1% vs. placebo 23.1%). Elevated transaminases were reported in the first three months and were more frequently observed with arimoclomol than with placebo (15.4% vs. 6.4%).

Conclusion: This trial did not demonstrate a benefit of arimoclomol in IBM with respect to its primary and secondary efficacy endpoints.


Disclosures: P. Machado, Abbvie, 6, BMS, 6, Celgene, 6, Eli Lilly, 2, Janssen, 2, MSD, 6, Galapagos, 6, Novartis, 2, 6, Pfizer, 6, Roche, 6, UCB, 2, 6, Orphazyme, 5, 6; R. Barohn, Orphazyme, 2, Orphazyme, 5; M. McDermott, Orphazyme, 5; T. Blaetter, Orphazyme, 3; T. Lloyd, Orphazyme, 5; A. Shaibani, Orphazyme, 5; M. Freimer, Orphazyme, 5; A. Amato, Orphazyme, 5; E. Ciafaloni, Orphazyme, 5; S. Jones, Orphazyme, 5; T. Mozaffar, None; S. Gibson, Orphazyme, 5; M. Wicklund, Orphazyme, 5; T. Levine, None; C. Sundgreen, Orphazyme, 3; T. Carstensen, Orphazyme, 3; K. Bonefeld, Orphazyme, 3; A. Jørgensen, Orphazyme, 3; K. Phonekeo, Orphazyme, 3; A. Heim, Orphazyme, 5; L. Herbelin, Orphazyme, 2, 5; M. Hanna, Orphazyme, 2, 5; M. Dimachkie, Octapharma, 2, 5, CSL-Behring, 2, 5, Orphazyme, 2, 5, Kezar, 1, 2, 5, UCB, 1, 2, 5, Shire Takeda, 2, 5, Bristol-Myers Squibb, 5, Corbus, 5, FDA/OOPD, 5, IMACS, 4.

To cite this abstract in AMA style:

Machado P, Barohn R, McDermott M, Blaetter T, Lloyd T, Shaibani A, Freimer M, Amato A, Ciafaloni E, Jones S, Mozaffar T, Gibson S, Wicklund M, Levine T, Sundgreen C, Carstensen T, Bonefeld K, Jørgensen A, Phonekeo K, Heim A, Herbelin L, Hanna M, Dimachkie M. A Randomized, Double-blind, Placebo-controlled Study of Arimoclomol in Patients with Inclusion Body Myositis [abstract]. Arthritis Rheumatol. 2021; 73 (suppl 9). https://acrabstracts.org/abstract/a-randomized-double-blind-placebo-controlled-study-of-arimoclomol-in-patients-with-inclusion-body-myositis/. Accessed January 27, 2023.
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