# Phase II Trial of Arimoclomol in IBM

> **NIH FDA R01** · UNIVERSITY OF KANSAS MEDICAL CENTER · 2020 · $397,836

## Abstract

Abstract
Sporadic inclusion body myositis (IBM) is the most common idiopathic inflammatory myopathy
presenting after age 50 years. It presents with chronic insidious proximal leg and distal arm
asymmetric muscle weakness. Muscle histopathology reveals endomysial inflammatory exudates
surrounding and invading non-necrotic muscle fibers often times accompanied by rimmed vacuoles
and inclusions. Unlike polymyositis and dermatomyositis, patients with IBM do not improve with
therapy; at present there is no effective treatment for IBM. The histopathological features and lack of
response to immunotherapies has led many experts in the field to believe that IBM is primarily a
degenerative disorder of muscle with secondary inflammation.
We completed a randomized controlled pilot study in 24 IBM subjects, 18 of whom received
arimoclomol 100 mg PO TID for four months and 8 were on placebo. Arimoclomol increases heat
shock proteins and may prevent protein misfolding. We found arimoclomol to be safe. The IBM
functional rating scale (IBMFRS) decline at 1 year was less in the arimoclomol group compared to
placebo with the p value approaching significance at 8 months.
We are therefore proposing a twenty months randomized, placebo-controlled Phase II study of
arimoclomol in 150 IBM subjects. The primary aim is to assess the efficacy and safety of arimoclomol
(200 mg TID). The primary efficacy endpoint is the IBMFRS. Secondary efficacy outcomes will
include different measures of strength and function: manual muscle testing (MMT), maximum
voluntary isometric contraction (MVICT) of quadriceps, grip and pinch test, modified timed up and go
(mTUG), 6 minute walk test with 2 minute distance captured; a general physical function measure:
Health Assessment Questionnaire (HAQ- DI); a Health-Related Quality of Life (HRQoL) measure
using SF36. Safety laboratory and adverse events will be collected. Our long-term goal is to find an
effective treatment for people with IBM.

## Key facts

- **NIH application ID:** 9970443
- **Project number:** 5R01FD004809-04
- **Recipient organization:** UNIVERSITY OF KANSAS MEDICAL CENTER
- **Principal Investigator:** Mazen Dimachkie
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** FDA
- **Fiscal year:** 2020
- **Award amount:** $397,836
- **Award type:** 5
- **Project period:** 2015-06-10 → 2022-05-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9970443

## Citation

> US National Institutes of Health, RePORTER application 9970443, Phase II Trial of Arimoclomol in IBM (5R01FD004809-04). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/9970443. Licensed CC0.

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