Arimoclomol in Sporadic Inclusion Body Myositis

November 22, 2016 updated by: Richard Barohn, MD

Safety and Tolerability Trial of Arimoclomol for 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.

Study Overview

Status

Completed

Conditions

Intervention / 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

Interventional

Enrollment (Actual)

24

Phase

  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

48 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Placebo Comparator: 2
Other: Placebo
Placebo for 4 months
Active Comparator: 1
Arimoclomol
Drug: Arimoclomol
Arimoclomol 100 mg TID for 4 months

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Count of Adverse Events Reported
Time Frame: Month 12
Measure reflects the total number of adverse events reported during course of the study.
Month 12

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Change from Baseline to Month 4
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.
Change from Baseline to Month 4
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.
Change from Baseline to Month 8
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.
Change from Baseline to Month 12
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.
Change from Baseline to Month 4
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.
Change from Baseline to Month 8
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.
Change from Baseline to Month 12
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.
Change from Baseline to Month 4
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.
Change from Baseline to Month 8
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.
Change from Baseline to Month 12

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Richard Barohn, MD

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

September 1, 2008

Primary Completion (Actual)

September 1, 2012

Study Completion (Actual)

September 1, 2012

Study Registration Dates

First Submitted

October 8, 2008

First Submitted That Met QC Criteria

October 8, 2008

First Posted (Estimate)

October 9, 2008

Study Record Updates

Last Update Posted (Estimate)

January 19, 2017

Last Update Submitted That Met QC Criteria

November 22, 2016

Last Verified

November 1, 2016

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 10656

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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