Arimoclomol in Sporadic Inclusion Body Myositis

Inclusion Body Myositis Clinical Trial

Official title:

Safety and Tolerability Trial of Arimoclomol for Sporadic Inclusion Body Myositis

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00769860
• Other study ID #: 10656
• Status: Completed
• Phase: Phase 2/Phase 3
• First received: October 8, 2008
• Last updated: March 5, 2013
• Start date: September 2008
• Est. completion date: September 2012

Study information

• Verified date: March 2013
• Source: University of Kansas Medical Center
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Completed
• Enrollment: 24
• Est. completion date: September 2012
• Est. primary completion date: September 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 50 Years and older

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

Study Design

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Inclusion Body Myositis
• Myositis
• Myositis, Inclusion Body

Intervention

Drug:
• Arimoclomol
Arimoclomol 100 mg TID for 4 months

Other:
• Placebo
Placebo for 4 months

Locations

Country	Name	City	State
United Kingdom	University College London, MRC Centre for Neuromuscular Disease	London
United States	University of Kansas Medical Center	Kansas City	Kansas

Sponsors (1)

Lead Sponsor	Collaborator
Richard Barohn, MD

Countries where clinical trial is conducted

United States,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Adverse event reporting		Every 2 weeks for 4 months	Yes
Secondary	Muscle Strength Testing		Monthly for 4 months	No
Secondary	IBM functional rating scale		Monthly for 4 months	No
Secondary	Muscle biopsy		pre and post treatment	Yes