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NCT Number: NCT00769860

Arimoclomol in Sporadic Inclusion Body Myositis

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.

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Key information

Conditions

Inclusion Body Myositis Muscular Diseases Musculoskeletal Diseases Myositis Myositis, Inclusion Body Nervous System Diseases Neuromuscular Diseases

Age range

50 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

University College London, MRC Centre for Neuromuscular Disease, London, United Kingdom

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Arimoclomol

Drug

Arimoclomol 100 mg TID for 4 months

Placebo

Other

Placebo for 4 months

Primary outcomes

  1. Count of Adverse Events Reported

    Time frame: Month 12

    Measure reflects the total number of adverse events reported during course of the study.

Secondary outcomes

  1. Heat Shock Protein 70 (HSP70) Levels in the Tissue

    Time frame: Change from Baseline to Month 4

    Biopsy taken from participants at baseline and month 4 visits. Measured change in HSP70 levels in the tissue.

  2. Muscle Strength Testing

    Time frame: Change from Baseline to Month 4

    Change in MMT score for the period. The MMT score range is 0-5. A score of 0 is the lowest and represents no visible or palpable contraction. A score of 5 is the highest and represents full range of motion against gravity, maximal resistance.

  3. Muscle Strength Testing

    Time frame: Change from Baseline to Month 8

    Change in MMT score for the period. The MMT score range is 0-5. A score of 0 is the lowest and represents no visible or palpable contraction. A score of 5 is the highest and represents full range of motion against gravity, maximal resistance.

  4. Muscle Strength Testing

    Time frame: Change from Baseline to Month 12

    Change in MMT score for the period. The MMT score range is 0-5. A score of 0 is the lowest and represents no visible or palpable contraction. A score of 5 is the highest and represents full range of motion against gravity, maximal resistance.

  5. Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score

    Time frame: Change from Baseline to Month 4

    Measured change for the period. The questionnaire has 10 questions. The maximum score is 40. The higher the score the better the functional status of the patient.

  6. Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score

    Time frame: Change from Baseline to Month 8

    Measured change for the period. The questionnaire has 10 questions. The maximum score is 40. The higher the score the better the functional status of the patient.

  7. Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score

    Time frame: Change from Baseline to Month 12

    Measured change for the period. The questionnaire has 10 questions. The maximum score is 40. The higher the score the better the functional status of the patient.

  8. Maximum Isometric Voluntary Contraction Testing (MVICT) Score

    Time frame: Change from Baseline to Month 4

    Measured MVICT using the Quantitative Muscle Assessment (QMA) system designed by Computer Source, Atlanta, GA. The system uses an adjustable cuff to attach the patient's arm or leg to an inelastic strap that is connected to force transducer with a load of 0.5 to 1,000 Newtons. Maximum force is recorded.

  9. Maximum Isometric Voluntary Contraction Testing (MVICT) Score

    Time frame: Change from Baseline to Month 8

    Measured MVICT using the Quantitative Muscle Assessment (QMA) system designed by Computer Source, Atlanta, GA. The system uses an adjustable cuff to attach the patient's arm or leg to an inelastic strap that is connected to force transducer with a load of 0.5 to 1,000 Newtons. Maximum force is recorded.

  10. Maximum Isometric Voluntary Contraction Testing (MVICT) Score

    Time frame: Change from Baseline to Month 12

    Measured MVICT using the Quantitative Muscle Assessment (QMA) system designed by Computer Source, Atlanta, GA. The system uses an adjustable cuff to attach the patient's arm or leg to an inelastic strap that is connected to force transducer with a load of 0.5 to 1,000 Newtons. Maximum force is recorded.

Sponsors and collaborators

Lead sponsor

Richard Barohn, MD

Other

Registry information

Official study title

Safety and Tolerability Trial of Arimoclomol for Sporadic Inclusion Body Myositis

Important dates

Study start
2008
Primary completion
2012
Study completion
2012
First posted
Oct 9, 2008
Registry last updated
Jan 19, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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