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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 1 | Active Comparator | Arimoclomol |
|
| 2 | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Arimoclomol | Drug | Arimoclomol 100 mg TID for 4 months |
| |
| Placebo |
| Measure | Description | Time Frame |
|---|---|---|
| Count of Adverse Events Reported | Measure reflects the total number of adverse events reported during course of the study. | Month 12 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Change from Baseline to Month 4 |
| Muscle Strength Testing |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States | ||
| University College London, MRC Centre for Neuromuscular Disease |
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| ID | Title | Description |
|---|---|---|
| FG000 | Arimoclomol | Arimoclomol 100 mg TID for 4 months |
| FG001 | Placebo | Matched placebo pills, 100 mg TID for 4 months |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Arimoclomol | Arimoclomol 100 mg TID for 4 months |
| BG001 | Placebo | Matched placebo pills, 100 mg TID for 4 months |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Count of Adverse Events Reported | Measure reflects the total number of adverse events reported during course of the study. | Posted | Number | adverse events reported | Month 12 |
|
|
Adverse event data was collected for the duration of the subject's participation, 12 months.
Participants were assessed at every study visit for adverse experiences. Safety laboratories were collected and monitored for all study participants.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Arimoclomol | Arimoclomol 100 mg TID for 4 months |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Stroke | Nervous system disorders | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Palpitations | Cardiac disorders | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Richard Barohn | University of Kansas Medical Center | (913) 588-6094 | RBAROHN@kumc.edu |
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| ID | Term |
|---|---|
| D018979 | Myositis, Inclusion Body |
| D009220 | Myositis |
| ID | Term |
|---|---|
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
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| ID | Term |
|---|---|
| C486387 | arimoclomol |
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| Other |
Placebo for 4 months |
|
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 | 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 | 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 | 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 | 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 | 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 | 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 | 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 | 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 |
| London |
| United Kingdom |
| BG002 |
| Total |
Total of all reporting groups |
| years |
|
| Gender | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score | The questionnaire has 10 questions. The maximum score is 40. The higher the score the better the functional status of the patient. | Mean | Standard Deviation | units on a scale |
|
| Manual Muscle Testing (MMT) Score | 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, maximul resistance. | Mean | Standard Deviation | units on a scale |
|
| 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. | Mean | Standard Deviation | newtons |
|
|
|
| Secondary | 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. | Posted | Mean | Standard Deviation | ng/100ng myosin | Change from Baseline to Month 4 |
|
|
|
| Secondary | 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. | Posted | Mean | Standard Deviation | units on a scale | Change from Baseline to Month 4 |
|
|
|
| Secondary | 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. | Posted | Mean | Standard Deviation | units on a scale | Change from Baseline to Month 8 |
|
|
|
| Secondary | 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. | Posted | Mean | Standard Deviation | units on a scale | Change from Baseline to Month 12 |
|
|
|
| Secondary | 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. | Posted | Mean | Standard Deviation | units on a scale | Change from Baseline to Month 4 |
|
|
|
| Secondary | 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. | Posted | Mean | Standard Deviation | units on a scale | Change from Baseline to Month 8 |
|
|
|
| Secondary | 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. | One of the subjects that dropped from the study in the Arimoclomol arm, returned for the 12 month visit. | Posted | Mean | Standard Deviation | units on a scale | Change from Baseline to Month 12 |
|
|
|
| Secondary | 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. | Posted | Mean | Standard Deviation | newtons | Change from Baseline to Month 4 |
|
|
|
| Secondary | 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. | Posted | Mean | Standard Deviation | newtons | Change from Baseline to Month 8 |
|
|
|
| Secondary | 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. | Posted | Mean | Standard Deviation | newtons | Change from Baseline to Month 12 |
|
|
|
| 0 |
| 16 |
| 16 |
| 16 |
| EG001 | Placebo | Matched placebo pills, 100 mg TID for 4 months | 1 | 8 | 8 | 8 |
| Dizziness/tinnitus | Ear and labyrinth disorders | Systematic Assessment |
|
| Conjunctivitis | Eye disorders | Systematic Assessment |
|
| Eye pain | Eye disorders | Systematic Assessment |
|
| Dry eyes | Eye disorders | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | Systematic Assessment |
|
| Throat irritation | Gastrointestinal disorders | Systematic Assessment |
|
| Loose stools | Gastrointestinal disorders | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | Systematic Assessment |
|
| Dry mouth | Gastrointestinal disorders | Systematic Assessment |
|
| Painful parotids | Gastrointestinal disorders | Systematic Assessment |
|
| Bowel movement problems | Gastrointestinal disorders | Systematic Assessment |
|
| Epigastralgia | Gastrointestinal disorders | Systematic Assessment |
|
| Gas pain | Gastrointestinal disorders | Systematic Assessment |
|
| Pyrosis | Gastrointestinal disorders | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | Systematic Assessment |
|
| Geographic tongue | Gastrointestinal disorders | Systematic Assessment |
|
| Weight loss | General disorders | Systematic Assessment |
|
| Dizziness | General disorders | Systematic Assessment |
|
| Loss of consciousness | General disorders | Systematic Assessment |
|
| Fatigue | General disorders | Systematic Assessment |
|
| Sinus infection | Infections and infestations | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | Systematic Assessment |
|
| Lower respiratory tract infection | Infections and infestations | Systematic Assessment |
|
| Tooth infection | Infections and infestations | Systematic Assessment |
|
| Erysipelas | Infections and infestations | Systematic Assessment |
|
| Cellulitis | Infections and infestations | Systematic Assessment |
|
| Leg ulcer infection | Infections and infestations | Systematic Assessment |
|
| Fall/contusion | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Post-biopsy pain | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Post-biopsy fatigue | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Pruritus in biopsy scar | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Finger cut | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Hyponatremia | Investigations | Systematic Assessment |
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| High thyroxine levels | Investigations | Systematic Assessment |
|
| Spinal stenosis | Investigations | Systematic Assessment |
|
| Herniated disk | Investigations | Systematic Assessment |
|
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Cramps | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Rheumatoid arthritis flare | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Heat and soreness of proximal lower limbs | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Headache | Nervous system disorders | Systematic Assessment |
|
| Worsening of restless leg syndrome | Nervous system disorders | Systematic Assessment |
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| Paresthesia | Nervous system disorders | Systematic Assessment |
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| Stroke | Nervous system disorders | Systematic Assessment |
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| Hematuria | Renal and urinary disorders | Systematic Assessment |
|
| Decreased libido | Reproductive system and breast disorders | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Rosacea | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Insect bite with erythema | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Cold sores | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Tooth extraction | Surgical and medical procedures | Systematic Assessment |
|
| Sinus surgery | Surgical and medical procedures | Systematic Assessment |
|
| Solar lentigines removal | Surgical and medical procedures | Systematic Assessment |
|
| Hypertension | Vascular disorders | Systematic Assessment |
|
| Edema | Vascular disorders | Systematic Assessment |
|
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