Arimoclomol in Sporadic Inclusion Body Myositis
Primary Purpose
Inclusion Body Myositis
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Arimoclomol
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Inclusion Body Myositis focused on measuring myositis, IBM, inclusion body myositis
Eligibility 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
Sites / Locations
- University of Kansas Medical Center
- University College London, MRC Centre for Neuromuscular Disease
Arms of the Study
Arm 1
Arm 2
Arm Type
Active Comparator
Placebo Comparator
Arm Label
1
2
Arm Description
Arimoclomol
Outcomes
Primary Outcome Measures
Count of Adverse Events Reported
Measure reflects the total number of adverse events reported during course of the study.
Secondary Outcome Measures
Heat Shock Protein 70 (HSP70) Levels in the Tissue
Biopsy taken from participants at baseline and month 4 visits. Measured change in HSP70 levels in the tissue.
Muscle Strength Testing
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
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
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
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
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
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
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
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
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.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT00769860
Brief Title
Arimoclomol in Sporadic Inclusion Body Myositis
Official Title
Safety and Tolerability Trial of Arimoclomol for Sporadic Inclusion Body Myositis
Study Type
Interventional
2. Study Status
Record Verification Date
November 2016
Overall Recruitment Status
Completed
Study Start Date
September 2008 (undefined)
Primary Completion Date
September 2012 (Actual)
Study Completion Date
September 2012 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Richard Barohn, MD
4. Oversight
Data Monitoring Committee
Yes
5. 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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Inclusion Body Myositis
Keywords
myositis, IBM, inclusion body myositis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
24 (Actual)
8. Arms, Groups, and Interventions
Arm Title
1
Arm Type
Active Comparator
Arm Description
Arimoclomol
Arm Title
2
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
Arimoclomol
Intervention Description
Arimoclomol 100 mg TID for 4 months
Intervention Type
Other
Intervention Name(s)
Placebo
Intervention Description
Placebo for 4 months
Primary Outcome Measure Information:
Title
Count of Adverse Events Reported
Description
Measure reflects the total number of adverse events reported during course of the study.
Time Frame
Month 12
Secondary Outcome Measure Information:
Title
Heat Shock Protein 70 (HSP70) Levels in the Tissue
Description
Biopsy taken from participants at baseline and month 4 visits. Measured change in HSP70 levels in the tissue.
Time Frame
Change from Baseline to Month 4
Title
Muscle Strength Testing
Description
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.
Time Frame
Change from Baseline to Month 4
Title
Muscle Strength Testing
Description
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.
Time Frame
Change from Baseline to Month 8
Title
Muscle Strength Testing
Description
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.
Time Frame
Change from Baseline to Month 12
Title
Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score
Description
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.
Time Frame
Change from Baseline to Month 4
Title
Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score
Description
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.
Time Frame
Change from Baseline to Month 8
Title
Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score
Description
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.
Time Frame
Change from Baseline to Month 12
Title
Maximum Isometric Voluntary Contraction Testing (MVICT) Score
Description
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.
Time Frame
Change from Baseline to Month 4
Title
Maximum Isometric Voluntary Contraction Testing (MVICT) Score
Description
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.
Time Frame
Change from Baseline to Month 8
Title
Maximum Isometric Voluntary Contraction Testing (MVICT) Score
Description
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.
Time Frame
Change from Baseline to Month 12
10. Eligibility
Sex
All
Minimum Age & Unit of Time
50 Years
Accepts Healthy Volunteers
No
Eligibility 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
Facility Information:
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
University College London, MRC Centre for Neuromuscular Disease
City
London
Country
United Kingdom
12. IPD Sharing Statement
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