Clinical trial NCT00769860: Arimoclomol in Sporadic Inclusion Body Myositis
Official title: Safety and Tolerability Trial of Arimoclomol for Sporadic Inclusion Body Myositis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Inclusion Body Myositis
Keywords: myositis
IBM
inclusion body myositis
Lead sponsor: University of Kansas Medical Center
Sponsor class: OTHER
First posted: 2008-10-09
Enrollment: 24
Sex: ALL
Minimum age (years): 50
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Count of Adverse Events Reported
timeFrame: Month 12
Secondary outcomes:
measure: Heat Shock Protein 70 (HSP70) Levels in the Tissue
timeFrame: Change from Baseline to Month 4
measure: Muscle Strength Testing
timeFrame: Change from Baseline to Month 4
measure: Muscle Strength Testing
timeFrame: Change from Baseline to Month 8
measure: Muscle Strength Testing
timeFrame: Change from Baseline to Month 12
measure: Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score
timeFrame: Change from Baseline to Month 4
measure: Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score
timeFrame: Change from Baseline to Month 8
measure: Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score
timeFrame: Change from Baseline to Month 12
measure: Maximum Isometric Voluntary Contraction Testing (MVICT) Score
timeFrame: Change from Baseline to Month 4
measure: Maximum Isometric Voluntary Contraction Testing (MVICT) Score
timeFrame: Change from Baseline to Month 8
measure: Maximum Isometric Voluntary Contraction Testing (MVICT) Score
timeFrame: Change from Baseline to Month 12
Other outcomes:
Locations:
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: London
country: United Kingdom
facility: University College London, MRC Centre for Neuromuscular Disease
Study officials:
References: