Arimoclomol in Sporadic Inclusion Body Myositis

• 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: Richard Barohn, MD
• Information provided by (Responsible Party): Richard Barohn, MD, University of Kansas Medical Center

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Inclusion Body Myositis	Drug: Arimoclomol; Other: Placebo	Phase 2; Phase 3

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 Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 24 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Double (Participant, Investigator)
• Primary Purpose: Treatment
• Official Title: Safety and Tolerability Trial of Arimoclomol for Sporadic Inclusion Body Myositis
• Study Start Date: September 2008
• Actual Primary Completion Date: September 2012
• Actual Study Completion Date: September 2012

Arms and Interventions

Arm	Intervention/treatment
Active Comparator: 1; Arimoclomol	Drug: Arimoclomol; Arimoclomol 100 mg TID for 4 months
Placebo Comparator: 2	Other: Placebo; Placebo for 4 months

Outcome Measures

Primary Outcome Measures :

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 Outcome Measures :

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.

Eligibility Criteria

• Ages Eligible for Study: 50 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• 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

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

• Responsible Party: Richard Barohn, MD, Gertrude and Dewey Zeigler Professor of Neurology and Chair, University of Kansas Medical Center
• ClinicalTrials.gov Identifier: NCT00769860
• Other Study ID Numbers: 10656
• First Posted: October 9, 2008
• Results First Posted: January 19, 2017
• Last Update Posted: January 19, 2017
• Last Verified: November 2016

Keywords provided by Richard Barohn, MD, University of Kansas Medical Center:

myositis; IBM; inclusion body myositis

Additional relevant MeSH terms:

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