Arimoclomol in Sporadic Inclusion Body Myositis - Full Text View

Sponsor:	University of Kansas
Collaborator:	Food and Drug Administration (FDA)
Information provided by:	University of Kansas
ClinicalTrials.gov Identifier:	NCT00769860

Purpose

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.

Condition	Intervention	Phase
Inclusion Body Myositis	Drug: Arimoclomol Other: Placebo	Phase II Phase III

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Investigator) Primary Purpose: Treatment
Official Title:	Safety and Tolerability Trial of Arimoclomol for Sporadic Inclusion Body Myositis

Resource links provided by NLM:

Genetics Home Reference related topics: idiopathic inflammatory myopathy

MedlinePlus related topics: Myositis

Drug Information available for: Arimoclomol

U.S. FDA Resources

Further study details as provided by University of Kansas:

Primary Outcome Measures:

Adverse event reporting [ Time Frame: Every 2 weeks for 4 months ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

Muscle Strength Testing [ Time Frame: Monthly for 4 months ] [ Designated as safety issue: No ]
IBM functional rating scale [ Time Frame: Monthly for 4 months ] [ Designated as safety issue: No ]
Muscle biopsy [ Time Frame: pre and post treatment ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	12
Study Start Date:	September 2008
Estimated Study Completion Date:	September 2010
Estimated Primary Completion Date:	September 2010 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Active Comparator: 1 Arimoclomol	Drug: Arimoclomol Arimoclomol 100 mg TID for 4 months
Placebo Comparator: 2	Other: Placebo Placebo for 4 months

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

Ages Eligible for Study:	50 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00769860

Locations

United States, Kansas
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
United Kingdom
University College London, MRC Centre for Neuromuscular Disease
London, United Kingdom

Sponsors and Collaborators

University of Kansas

Food and Drug Administration (FDA)

More Information

No publications provided

Responsible Party:	Richard Barohn MD, University of Kansas Medical Center
ClinicalTrials.gov Identifier:	NCT00769860 History of Changes
Other Study ID Numbers:	10656
Study First Received:	October 8, 2008
Last Updated:	April 23, 2010
Health Authority:	United States: Food and Drug Administration

Keywords provided by University of Kansas:

myositis
IBM
inclusion body myositis

Additional relevant MeSH terms:

Myositis
Myositis, Inclusion Body
Muscular Diseases

Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases

ClinicalTrials.gov processed this record on February 09, 2012