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Trial record 13 of 215 for:    Inflammatory Myopathies

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

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Condition Inclusion Body Myositis
Interventions Drug: Arimoclomol
Other: Placebo
Enrollment 24
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Arimoclomol Placebo
Hide Arm/Group Description Arimoclomol 100 mg TID for 4 months Matched placebo pills, 100 mg TID for 4 months
Period Title: Overall Study
Started 16 8
Month 4 16 8
Month 8 14 8
Completed 14 8
Not Completed 2 0
Reason Not Completed
Withdrawal by Subject             2             0
Arm/Group Title Arimoclomol Placebo Total
Hide Arm/Group Description Arimoclomol 100 mg TID for 4 months Matched placebo pills, 100 mg TID for 4 months Total of all reporting groups
Overall Number of Baseline Participants 16 8 24
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 16 participants 8 participants 24 participants
65.85  (7.86) 68.83  (6.70) 66.84  (7.49)
Gender  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 16 participants 8 participants 24 participants
Female
4
  25.0%
3
  37.5%
7
  29.2%
Male
12
  75.0%
5
  62.5%
17
  70.8%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 16 participants 8 participants 24 participants
United States 8 4 12
United Kingdom 8 4 12
Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 16 participants 8 participants 24 participants
27.5  (7.0) 24.6  (5.1) 26.6  (6.4)
[1]
Measure Description: The questionnaire has 10 questions. The maximum score is 40. The higher the score the better the functional status of the patient.
Manual Muscle Testing (MMT) Score   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 16 participants 8 participants 24 participants
4.2  (0.5) 3.6  (1.5) 4.2  (0.4)
[1]
Measure Description: 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.
Maximum Isometric Voluntary Contraction Testing (MVICT) Score   [1] 
Mean (Standard Deviation)
Unit of measure:  Newtons
Number Analyzed 16 participants 8 participants 24 participants
130.4  (70.4) 94.4  (39.8) 119.4  (63.4)
[1]
Measure 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.
1.Primary Outcome
Title Count of Adverse Events Reported
Hide Description Measure reflects the total number of adverse events reported during course of the study.
Time Frame Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Arimoclomol Placebo
Hide Arm/Group Description:
Arimoclomol 100 mg TID for 4 months
Matched placebo pills, 100 mg TID for 4 months
Overall Number of Participants Analyzed 16 8
Measure Type: Number
Unit of Measure: adverse events reported
109 52
2.Secondary Outcome
Title Heat Shock Protein 70 (HSP70) Levels in the Tissue
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Arimoclomol Placebo
Hide Arm/Group Description:
Arimoclomol 100 mg TID for 4 months
Matched placebo pills, 100 mg TID for 4 months
Overall Number of Participants Analyzed 15 8
Mean (Standard Deviation)
Unit of Measure: ng/100ng myosin
-110.72  (757.40) -34.70  (336.35)
3.Secondary Outcome
Title Muscle Strength Testing
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Arimoclomol Placebo
Hide Arm/Group Description:
Arimoclomol 100 mg TID for 4 months
Matched placebo pills, 100 mg TID for 4 months
Overall Number of Participants Analyzed 15 8
Mean (Standard Deviation)
Unit of Measure: units on a scale
-0.04  (0.19) -0.12  (0.20)
4.Secondary Outcome
Title Muscle Strength Testing
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Arimoclomol Placebo
Hide Arm/Group Description:
Arimoclomol 100 mg TID for 4 months
Matched placebo pills, 100 mg TID for 4 months
Overall Number of Participants Analyzed 13 8
Mean (Standard Deviation)
Unit of Measure: units on a scale
-0.12  (0.22) -0.26  (0.27)
5.Secondary Outcome
Title Muscle Strength Testing
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Arimoclomol Placebo
Hide Arm/Group Description:
Arimoclomol 100 mg TID for 4 months
Matched placebo pills, 100 mg TID for 4 months
Overall Number of Participants Analyzed 14 7
Mean (Standard Deviation)
Unit of Measure: units on a scale
-0.21  (0.21) -0.35  (0.20)
6.Secondary Outcome
Title Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Arimoclomol Placebo
Hide Arm/Group Description:
Arimoclomol 100 mg TID for 4 months
Matched placebo pills, 100 mg TID for 4 months
Overall Number of Participants Analyzed 16 8
Mean (Standard Deviation)
Unit of Measure: units on a scale
-0.34  (1.38) -0.88  (1.16)
7.Secondary Outcome
Title Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Arimoclomol Placebo
Hide Arm/Group Description:
Arimoclomol 100 mg TID for 4 months
Matched placebo pills, 100 mg TID for 4 months
Overall Number of Participants Analyzed 14 8
Mean (Standard Deviation)
Unit of Measure: units on a scale
-0.68  (1.58) -2.50  (3.31)
8.Secondary Outcome
Title Inclusion Body Myositis-Functional Rating Scale (IBMFRS) Score
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
One of the subjects that dropped from the study in the Arimoclomol arm, returned for the 12 month visit.
Arm/Group Title Arimoclomol Placebo
Hide Arm/Group Description:
Arimoclomol 100 mg TID for 4 months
Matched placebo pills, 100 mg TID for 4 months
Overall Number of Participants Analyzed 15 8
Mean (Standard Deviation)
Unit of Measure: units on a scale
-2.03  (2.68) -3.50  (3.35)
9.Secondary Outcome
Title Maximum Isometric Voluntary Contraction Testing (MVICT) Score
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Arimoclomol Placebo
Hide Arm/Group Description:
Arimoclomol 100 mg TID for 4 months
Matched placebo pills, 100 mg TID for 4 months
Overall Number of Participants Analyzed 14 8
Mean (Standard Deviation)
Unit of Measure: newtons
0.46  (12.11) -0.30  (14.49)
10.Secondary Outcome
Title Maximum Isometric Voluntary Contraction Testing (MVICT) Score
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Arimoclomol Placebo
Hide Arm/Group Description:
Arimoclomol 100 mg TID for 4 months
Matched placebo pills, 100 mg TID for 4 months
Overall Number of Participants Analyzed 13 8
Mean (Standard Deviation)
Unit of Measure: newtons
7.20  (19.65) -1.71  (17.80)
11.Secondary Outcome
Title Maximum Isometric Voluntary Contraction Testing (MVICT) Score
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Arimoclomol Placebo
Hide Arm/Group Description:
Arimoclomol 100 mg TID for 4 months
Matched placebo pills, 100 mg TID for 4 months
Overall Number of Participants Analyzed 14 8
Mean (Standard Deviation)
Unit of Measure: newtons
-1.21  (20.76) 0.52  (17.98)
Time Frame Adverse event data was collected for the duration of the subject's participation, 12 months.
Adverse Event Reporting Description Participants were assessed at every study visit for adverse experiences. Safety laboratories were collected and monitored for all study participants.
 
Arm/Group Title Arimoclomol Placebo
Hide Arm/Group Description Arimoclomol 100 mg TID for 4 months Matched placebo pills, 100 mg TID for 4 months
All-Cause Mortality
Arimoclomol Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Arimoclomol Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/16 (0.00%)      1/8 (12.50%)    
Nervous system disorders     
Stroke   0/16 (0.00%)  0 1/8 (12.50%)  1
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Arimoclomol Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   16/16 (100.00%)      8/8 (100.00%)    
Cardiac disorders     
Palpitations   1/16 (6.25%)  1 0/8 (0.00%)  0
Ear and labyrinth disorders     
Dizziness/tinnitus   2/16 (12.50%)  2 0/8 (0.00%)  0
Eye disorders     
Conjunctivitis   1/16 (6.25%)  1 0/8 (0.00%)  0
Eye pain   1/16 (6.25%)  1 0/8 (0.00%)  0
Dry eyes   0/16 (0.00%)  0 1/8 (12.50%)  1
Gastrointestinal disorders     
Constipation   4/16 (25.00%)  4 4/8 (50.00%)  4
Throat irritation   3/16 (18.75%)  4 0/8 (0.00%)  0
Loose stools   2/16 (12.50%)  2 4/8 (50.00%)  4
Nausea   2/16 (12.50%)  2 0/8 (0.00%)  0
Dry mouth   2/16 (12.50%)  2 0/8 (0.00%)  0
Painful parotids   0/16 (0.00%)  0 2/8 (25.00%)  2
Bowel movement problems   1/16 (6.25%)  1 0/8 (0.00%)  0
Epigastralgia   1/16 (6.25%)  1 0/8 (0.00%)  0
Gas pain   1/16 (6.25%)  1 0/8 (0.00%)  0
Pyrosis   1/16 (6.25%)  1 0/8 (0.00%)  0
Vomiting   1/16 (6.25%)  1 0/8 (0.00%)  0
Geographic tongue   1/16 (6.25%)  1 0/8 (0.00%)  0
General disorders     
Weight loss   1/16 (6.25%)  1 0/8 (0.00%)  0
Dizziness   1/16 (6.25%)  1 0/8 (0.00%)  0
Loss of consciousness   1/16 (6.25%)  1 0/8 (0.00%)  0
Fatigue   0/16 (0.00%)  0 1/8 (12.50%)  1
Infections and infestations     
Sinus infection   2/16 (12.50%)  2 0/8 (0.00%)  0
Upper respiratory tract infection   5/16 (31.25%)  7 3/8 (37.50%)  3
Lower respiratory tract infection   2/16 (12.50%)  2 0/8 (0.00%)  0
Tooth infection   1/16 (6.25%)  1 1/8 (12.50%)  4
Erysipelas   1/16 (6.25%)  1 0/8 (0.00%)  0
Cellulitis   0/16 (0.00%)  0 1/8 (12.50%)  1
Leg ulcer infection   0/16 (0.00%)  0 1/8 (12.50%)  1
Injury, poisoning and procedural complications     
Fall/contusion   8/16 (50.00%)  23 5/8 (62.50%)  9
Post-biopsy pain   3/16 (18.75%)  3 1/8 (12.50%)  1
Post-biopsy fatigue   1/16 (6.25%)  1 0/8 (0.00%)  0
Pruritus in biopsy scar   0/16 (0.00%)  0 1/8 (12.50%)  1
Finger cut   0/16 (0.00%)  0 1/8 (12.50%)  1
Investigations     
Hyponatremia   2/16 (12.50%)  2 0/8 (0.00%)  0
High thyroxine levels   1/16 (6.25%)  1 0/8 (0.00%)  0
Spinal stenosis   0/16 (0.00%)  0 1/8 (12.50%)  1
Herniated disk   0/16 (0.00%)  0 1/8 (12.50%)  1
Musculoskeletal and connective tissue disorders     
Musculoskeletal pain   6/16 (37.50%)  10 2/8 (25.00%)  2
Cramps   1/16 (6.25%)  1 0/8 (0.00%)  0
Rheumatoid arthritis flare   1/16 (6.25%)  1 0/8 (0.00%)  0
Heat and soreness of proximal lower limbs   1/16 (6.25%)  1 0/8 (0.00%)  0
Nervous system disorders     
Headache   3/16 (18.75%)  7 2/8 (25.00%)  3
Worsening of restless leg syndrome   1/16 (6.25%)  1 0/8 (0.00%)  0
Paresthesia   1/16 (6.25%)  1 1/8 (12.50%)  1
Stroke   0/16 (0.00%)  0 1/8 (12.50%)  1
Renal and urinary disorders     
Hematuria   1/16 (6.25%)  1 0/8 (0.00%)  0
Reproductive system and breast disorders     
Decreased libido   0/16 (0.00%)  0 1/8 (12.50%)  1
Respiratory, thoracic and mediastinal disorders     
Cough   1/16 (6.25%)  2 1/8 (12.50%)  1
Skin and subcutaneous tissue disorders     
Rash   2/16 (12.50%)  2 1/8 (12.50%)  1
Rosacea   1/16 (6.25%)  1 0/8 (0.00%)  0
Insect bite with erythema   1/16 (6.25%)  1 0/8 (0.00%)  0
Cold sores   1/16 (6.25%)  1 0/8 (0.00%)  0
Surgical and medical procedures     
Tooth extraction   1/16 (6.25%)  1 1/8 (12.50%)  1
Sinus surgery   1/16 (6.25%)  1 0/8 (0.00%)  0
Solar lentigines removal   1/16 (6.25%)  1 0/8 (0.00%)  0
Vascular disorders     
Hypertension   2/16 (12.50%)  3 2/8 (25.00%)  3
Edema   2/16 (12.50%)  2 3/8 (37.50%)  3
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Dr. Richard Barohn
Organization: University of Kansas Medical Center
Phone: (913) 588-6094
Responsible Party: Richard Barohn, MD, University of Kansas Medical Center
ClinicalTrials.gov Identifier: NCT00769860     History of Changes
Other Study ID Numbers: 10656
First Submitted: October 8, 2008
First Posted: October 9, 2008
Results First Submitted: November 22, 2016
Results First Posted: January 19, 2017
Last Update Posted: January 19, 2017