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Arimoclomol in Sporadic Inclusion Body Myositis

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Richard Barohn, MD, University of Kansas Medical Center Research Institute
ClinicalTrials.gov Identifier:
NCT00769860
First received: October 8, 2008
Last updated: March 5, 2013
Last verified: March 2013
History of Changes
  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 2
Phase 3

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:

MedlinePlus related topics: Myositis
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 ]

Enrollment: 24
Study Start Date: September 2008
Study Completion Date: September 2012
Primary Completion Date: September 2012 (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
Richard Barohn, MD
  More Information

No publications provided

Responsible Party: Richard Barohn, MD, Gertrude and Dewey Zeigler Professor of Neurology and Chair, University of Kansas Medical Center Research Institute
ClinicalTrials.gov Identifier: NCT00769860     History of Changes
Other Study ID Numbers: 10656
Study First Received: October 8, 2008
Last Updated: March 5, 2013
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 June 17, 2013