drugset / Trial / NCT02753530
Study of Arimoclomol in Inclusion Body Myositis (IBM)
Phase 2
Completed
152 enrolled
ZevraDenmark
University College, London · collabUniversity of Kansas Medical Center · collab
RandomizedParallel-groupDouble-blindTreatment
Summary
Funding Source - FDA Office of Orphan Products Development (OOPD). The purpose of this study is to evaluate the safety and efficacy of the study drug, arimoclomol in IBM patients.
Timeline
- Start
- 2017-08-16
- Primary completion
- 2021-01-11
- Completion
- 2021-01-11
Outcome
Missed primary endpoint
registry analysis (superiority test); Arimoclomol (20 Months) vs Placebo (20 Months); p = 0.1146; Least Square (LS) Mean Difference -0.99 (95% CI -2.23 to 0.24) NCT02753530 ↗
paper At month 20, mean IBMFRS change from baseline was not statistically significantly different between arimoclomol and placebo PMID 37739573 ↗
Publications
- 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.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Arimoclomol | Small molecule | 248 mg | Oral |