Arimoclomol in Sporadic Inclusion Body Myositis

NCT ID: NCT00769860

Last Updated: 2017-01-19

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 24 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2008-09-30
• Study Completion Date: 2012-09-30

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.

Conditions

Inclusion Body Myositis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

1

Arimoclomol

Group Type: ACTIVE_COMPARATOR

Arimoclomol

Intervention Type: DRUG

Arimoclomol 100 mg TID for 4 months

2

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: OTHER

Placebo for 4 months

Interventions

Arimoclomol

Arimoclomol 100 mg TID for 4 months

Intervention Type: DRUG

Placebo

Placebo for 4 months

Intervention Type: OTHER

Eligibility Criteria

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

• Minimum Eligible Age: 50 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Richard Barohn, MD

OTHER

Sponsor Role: lead

Responsible Party

Richard Barohn, MD

Gertrude and Dewey Zeigler Professor of Neurology and Chair

Responsibility Role: SPONSOR_INVESTIGATOR

Locations

University of Kansas Medical Center

Kansas City, Kansas, United States

University College London, MRC Centre for Neuromuscular Disease

London, , United Kingdom

Countries

United States; United Kingdom

Other Identifiers

10656

Identifier Type: -

Identifier Source: org_study_id