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Trial record 8 of 11 for:    Arimoclomol

Arimoclomol in Sporadic Inclusion Body Myositis

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ClinicalTrials.gov Identifier: NCT00769860
Recruitment Status : Completed
First Posted : October 9, 2008
Results First Posted : January 19, 2017
Last Update Posted : January 19, 2017
Sponsor:
Information provided by (Responsible Party):
Richard Barohn, MD, University of Kansas Medical Center

Tracking Information
First Submitted Date  ICMJE October 8, 2008
First Posted Date  ICMJE October 9, 2008
Results First Submitted Date  ICMJE November 22, 2016
Results First Posted Date  ICMJE January 19, 2017
Last Update Posted Date January 19, 2017
Study Start Date  ICMJE September 2008
Actual Primary Completion Date September 2012   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 22, 2016)
Count of Adverse Events Reported [ Time Frame: Month 12 ]
Measure reflects the total number of adverse events reported during course of the study.
Original Primary Outcome Measures  ICMJE
 (submitted: October 8, 2008)
Adverse event reporting [ Time Frame: Every 2 weeks for 4 months ]
Change History Complete list of historical versions of study NCT00769860 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: November 22, 2016)
  • 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.
  • 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.
  • 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.
  • 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.
  • 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.
  • 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.
  • 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.
  • 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.
  • 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.
  • 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.
Original Secondary Outcome Measures  ICMJE
 (submitted: October 8, 2008)
  • Muscle Strength Testing [ Time Frame: Monthly for 4 months ]
  • IBM functional rating scale [ Time Frame: Monthly for 4 months ]
  • Muscle biopsy [ Time Frame: pre and post treatment ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Arimoclomol in Sporadic Inclusion Body Myositis
Official Title  ICMJE Safety and Tolerability Trial of Arimoclomol for Sporadic Inclusion Body Myositis
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Inclusion Body Myositis
Intervention  ICMJE
  • Drug: Arimoclomol
    Arimoclomol 100 mg TID for 4 months
  • Other: Placebo
    Placebo for 4 months
Study Arms  ICMJE
  • Active Comparator: 1
    Arimoclomol
    Intervention: Drug: Arimoclomol
  • Placebo Comparator: 2
    Intervention: Other: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: March 5, 2013)
24
Original Estimated Enrollment  ICMJE
 (submitted: October 8, 2008)
12
Actual Study Completion Date  ICMJE September 2012
Actual Primary Completion Date September 2012   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 50 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00769860
Other Study ID Numbers  ICMJE 10656
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Richard Barohn, MD, University of Kansas Medical Center
Study Sponsor  ICMJE Richard Barohn, MD
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account University of Kansas Medical Center
Verification Date November 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP