Arimoclomol in Sporadic Inclusion Body Myositis

Richard Barohn, MD24 enrolled

Overview

Inclusion body myositis (IBM) is the most common progressive and debilitating muscle disease beginning in persons over 50 years of age. This study will assess the safety and tolerability of Arimoclomol in IBM as compared to placebo over 4 months of treatment.

IBM is a chronic disorder in which muscles become inflamed (swollen) and cause muscle weakening. The cause is unknown. There is new evidence to suggest that the pathology in IBM results from cellular changes induced by a variety of stressful events and diseases. In response to these stressful events the body's normal response is to increase the levels of Heat Shock Proteins (HSP) to help counteract and stop these cellular changes. In people with IBM this increase does not appear sufficient enough to reverse these toxic cellular changes. Arimoclomol causes the body to make more of this HSP protein. By increasing HSP levels in IBM patients we hope to reverse the toxic cellular changes that might be responsible for the pathology of IBM.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

Conditions

Inclusion Body Myositis

Interventions

ArimoclomolDRUG

Arimoclomol 100 mg TID for 4 months

PlaceboOTHER

Placebo for 4 months

Eligibility

Sex: ALLMin age: 50 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Meet the diagnostic criteria for definite or probable IBM (Griggs 1995) * Muscle function adequate for quantitative muscle testing * Age \> 50 years * Women must be postmenopausal or status post hysterectomy * For any patient currently taking medication for IBM, they must remain on current dosage for the extent of the study and last dosage change must be \> 30 days previous to enrollment Exclusion Criteria: * Presence of any one of the following medical conditions: diabetes mellitus or patients taking anti-diabetic medications, chronic infection, chronic renal insufficiency, cancer other than skin cancer less than 5 years previously, multiple sclerosis or prior episode or central nervous system demyelination, or other chronic serious medical illnesses * Presence of any of the following on routine blood screening: WBC \< 3000, platelets \< 100,000, hematocrit \< 30%, BUN \> 30 mg%, creatine \> 1.5 mg%, symptomatic liver disease with serum albumin \< 3 g/dl, PT or PTT \> upper range of control values * Women who are pregnant or lactating * History of non-compliance with other therapies * Coexistence of other neuromuscular disease * Drug or alcohol abuse within the last 3 months * Inability to give informed consent * Known bleeding disorder * Use of potentially renal toxic drugs * Prior difficulties with local anesthetic

Locations (2)

University of Kansas Medical Center

Kansas City, Kansas, United States

University College London, MRC Centre for Neuromuscular Disease

London, United Kingdom

Outcomes

Primary Outcomes

Count of Adverse Events Reported

Measure reflects the total number of adverse events reported during course of the study.

Time frame: Month 12

Secondary Outcomes

Heat Shock Protein 70 (HSP70) Levels in the Tissue

Biopsy taken from participants at baseline and month 4 visits. Measured change in HSP70 levels in the tissue.

Time frame: Change from Baseline to Month 4

Muscle Strength Testing

Change in MMT score for the period. The MMT score range is 0-5. A score of 0 is the lowest and represents no visible or palpable contraction. A score of 5 is the highest and represents full range of motion against gravity, maximal resistance.

Time frame: Change from Baseline to Month 4

Muscle Strength Testing

Time frame: Change from Baseline to Month 8

Muscle Strength Testing

Time frame: Change from Baseline to Month 12

Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score

Measured change for the period. The questionnaire has 10 questions. The maximum score is 40. The higher the score the better the functional status of the patient.

Time frame: Change from Baseline to Month 4

Data from ClinicalTrials.gov

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