Arimoclomol in Sporadic Inclusion Body Myositis

Phase 2

Completed

Brief Summary: 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.

Detailed Description: 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.

Recruitment & Eligibility

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

Arm && Interventions

Group	Intervention	Description
1	Arimoclomol	Arimoclomol
2	Placebo	-

Primary Outcome Measures

Name	Time	Method
Count of Adverse Events Reported	Month 12	Measure reflects the total number of adverse events reported during course of the study.

Secondary Outcome Measures

Name	Time	Method
Heat Shock Protein 70 (HSP70) Levels in the Tissue	Change from Baseline to Month 4	Biopsy taken from participants at baseline and month 4 visits. Measured change in HSP70 levels in the tissue.
Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score	Change from Baseline to Month 12	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.
Muscle Strength Testing	Change from Baseline to Month 12	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.
Maximum Isometric Voluntary Contraction Testing (MVICT) Score	Change from Baseline to Month 12	Measured MVICT using the Quantitative Muscle Assessment (QMA) system designed by Computer Source, Atlanta, GA. The system uses an adjustable cuff to attach the patient's arm or leg to an inelastic strap that is connected to force transducer with a load of 0.5 to 1,000 Newtons. Maximum force is recorded.

Locations (2): University College London, MRC Centre for Neuromuscular Disease
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London, United Kingdom
University of Kansas Medical Center
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Kansas City, Kansas, United States