Arimoclomol in Sporadic Inclusion Body Myositis

Richard Barohn, MD

Status and phase

Completed

Phase 3

Phase 2

Conditions

Inclusion Body Myositis

Treatments

Drug: Arimoclomol

Other: Placebo

Study type

Interventional

Funder types

Other

Identifiers

NCT00769860

10656

Details and patient eligibility

About

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.

Full 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.

Enrollment

24 patients

Sex

All

Ages

50+ years old

Volunteers

No Healthy Volunteers

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