Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
152 participants
INTERVENTIONAL
2017-08-16
2021-01-11
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
DOUBLE
Study Groups
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Arimoclomol
248 mg arimoclomol base (equivalent to 400 mg arimoclomol citrate) 3 times daily
Arimoclomol
2 capsules (2 x 124 mg arimoclomol base; equivalent to 2 x 200 mg arimoclomol citrate) taken 3 times daily during breakfast, early afternoon, and at bedtime
Placebo
248 mg matching placebo 3 times daily
Placebo
2 matched placebo capsules taken 3 times daily during breakfast, early afternoon, and at bedtime
Interventions
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Arimoclomol
2 capsules (2 x 124 mg arimoclomol base; equivalent to 2 x 200 mg arimoclomol citrate) taken 3 times daily during breakfast, early afternoon, and at bedtime
Placebo
2 matched placebo capsules taken 3 times daily during breakfast, early afternoon, and at bedtime
Eligibility Criteria
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Inclusion Criteria
* Demonstrate being able to arise from a chair without support from another person or device.
* Able to ambulate at least 20 ft/6 meters with or without assistive device. Once arisen from the chair, participant may use any walking device, i.e. walker/frame, can, crutches, or braces. They cannot be supported by another person and cannot use furniture or wall for support.
* Age at onset of weakness \>45 years.
* Body weight of \>= 40 kg.
* Able to give informed consent.
Exclusion Criteria
* Presence of any of the following on routine blood screening: White blood cells (WBC) \<3000; platelets \< 100,000; hematocrit \<30%; blood urea nitrogen (BUN) \>30 mg/dL; creatinine \>1.5 x upper limit of normal; symptomatic liver disease with serum albumin \<3 g/dL.
* History of most recent creatine kinase \>15x the upper limit of normal without any other explanation besides IBM.
* History of non-compliance with other therapies.
* Use of testosterone except for physiologic replacement doses in case of androgen deficiency. Participants must have documented proof of the androgen deficiency.
* Coexistence of other disease that would be likely to affect outcome measures
* Drug or alcohol abuse within past three months.
* Participation in a recent drug study in the last 30 days prior to the screening visit or use of a biologic agent less than 6 months prior to the screening visit.
* Women who are lactating or unwilling to use adequate method of birth control who are not surgically sterile. Adequate birth control includes use of intrauterine device, abstinence, or oral contraceptives or a double barrier method, e.g condom plus diaphragm will be necessary for both male and female participants.
* Participants taking \>7.5 mg prednisolone or equivalent or participants on Intravenous Immunoglobulin Therapy (IVIg) or other immunosuppressants within the last 3 months.
* Clinically significant renal or hepatic disease, as indicated by clinical laboratory assessment.
45 Years
ALL
No
Sponsors
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University of Kansas Medical Center
OTHER
University College, London
OTHER
ZevraDenmark
INDUSTRY
Responsible Party
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Principal Investigators
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Mazen Dimachkie, MD
Role: PRINCIPAL_INVESTIGATOR
University of Kansas Medical Center
Michael Hanna, MD
Role: PRINCIPAL_INVESTIGATOR
University College, London
Locations
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Phoenix Neurological Associates
Phoenix, Arizona, United States
University of California, Irvine
Irvine, California, United States
University of Colorado School of Medicine
Aurora, Colorado, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Johns Hopkins University
Baltimore, Maryland, United States
Brigham and Women's Hospital
Boston, Massachusetts, United States
University of Rochester
Rochester, New York, United States
The Ohio State University
Columbus, Ohio, United States
Nerve and Muscle Center of Texas
Houston, Texas, United States
University of Utah
Salt Lake City, Utah, United States
University of Virginia
Charlottesville, Virginia, United States
University College of London
London, , United Kingdom
Countries
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References
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Salam S, Symonds T, Doll H, Rousell S, Randall J, Lloyd-Price L, Hudgens S, Guldberg C, Herbelin L, Barohn RJ, Hanna MG, Dimachkie MM, Machado PM; Members of the Arimoclomol in IBM Investigator Team of the Neuromuscular Study Group. Measurement properties of the Inclusion Body Myositis Functional Rating Scale. J Neurol Neurosurg Psychiatry. 2025 Jan 16;96(2):122-131. doi: 10.1136/jnnp-2024-333617.
Machado PM, McDermott MP, Blaettler T, Sundgreen C, Amato AA, Ciafaloni E, Freimer M, Gibson SB, Jones SM, Levine TD, Lloyd TE, Mozaffar T, Shaibani AI, Wicklund M, Rosholm A, Carstensen TD, Bonefeld K, Jorgensen AN, Phonekeo K, Heim AJ, Herbelin L, Barohn RJ, Hanna MG, Dimachkie MM; Arimoclomol in IBM Investigator Team of the Neuromuscular Study Group. Safety and efficacy of arimoclomol for inclusion body myositis: a multicentre, randomised, double-blind, placebo-controlled trial. Lancet Neurol. 2023 Oct;22(10):900-911. doi: 10.1016/S1474-4422(23)00275-2.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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STUDY00002461
Identifier Type: -
Identifier Source: org_study_id
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