Study of Arimoclomol in Inclusion Body Myositis (IBM)
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT02753530 |
Recruitment Status :
Completed
First Posted : April 28, 2016
Last Update Posted : March 24, 2021
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Condition or disease | Intervention/treatment | Phase |
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Inclusion Body Myositis | Drug: Arimoclomol Other: Placebo | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 152 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Double (Participant, Investigator) |
Primary Purpose: | Treatment |
Official Title: | Phase II Study of Arimoclomol for the Treatment of Sporadic Inclusion Body Myositis (IBM) |
Actual Study Start Date : | August 16, 2017 |
Actual Primary Completion Date : | January 11, 2021 |
Actual Study Completion Date : | January 11, 2021 |

Arm | Intervention/treatment |
---|---|
Experimental: Arimoclomol
Participants will be asked to take 400mg arimoclomol three times a day.
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Drug: Arimoclomol
2 200mg pills taken 3 times per day during breakfast, early afternoon and at bedtime |
Placebo Comparator: Placebo
Participants will be asked to take 400mg placebo three times a day.
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Other: Placebo
2 matched placebo pills to be taken 3 times per day during breakfast, early afternoon and at bedtime |
- Decline in Inclusion body myositis functional rating scale (IBMFRS) [ Time Frame: Change from Baseline to Month 20 ]Measured by rate of decline in the IBMFRS between experimental and placebo groups. The IBMFRS is a 10 item questionnaire. Scores for each item range from 0 to 4. There is a total maximum score of 40 and minimum score of 0. The higher the score the better functional status of the person.
- Manual Muscle Testing (MMT) [ Time Frame: Change from Baseline to Month 20 ]
- Maximum voluntary isometric contraction (MVICT) of quadriceps [ Time Frame: Change from Baseline to Month 20 ]
- Grip and pinch test [ Time Frame: Change from Baseline to Month 20 ]
- modified timed up and go (mTUG) [ Time Frame: Change from Baseline to Month 20 ]
- 6 minute walk test with 2 minute distance captured [ Time Frame: Change from Baseline to Month 20 ]
- Health Assessment Questionnaire (HAQ-DI) [ Time Frame: Change from Baseline to Month 20 ]
- SF-36 [ Time Frame: Change from Baseline to Month 20 ]Measured using the Short form health survey with 36 items (SF-36).
- Count of adverse events [ Time Frame: Change from Baseline to Month 20 ]
- Decline in Inclusion body myositis functional rating scale (IBMFRS) [ Time Frame: Change from Baseline to Month 12 ]Measured by rate of decline in the IBMFRS between experimental and placebo groups. The IBMFRS is a 10 item questionnaire. Scores for each item range from 0 to 4. There is a total maximum score of 40 and minimum score of 0. The higher the score the better functional status of the person.

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Ages Eligible for Study: | 45 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Meet any of the European Neuromuscular Centre Inclusion Body Myositis research diagnostic criteria 2011 categories for IBM
- Demonstrate being able to arise from a chair without support from another person or device
- Able to ambulate at least 20 ft/6 meters with or without assistive device. Once arisen from the chair, participant may use any walking device, i.e. walker/frame, can, crutches, or braces. They cannot be supported by another person and cannot use furniture or wall for support.
- Body weight of >= 40 kg
- Pre-menopausal women must have a negative pregnancy tst prior to dosing iwht study medication.
- If a participant in the bimagrumab study, the participant must be off of the study medication for at least 6 months.
- Able to give informed consent
Exclusion Criteria:
- History of any of the following excludes subject participation in the study: chronic infection particularly HIV or Hepatitis B or C; cancer other than basal cell cancer less than five years prior; or other chronic serious medical illnesses.
- Presence of any of the following on routine blood screening: WEB <3000; platelets < 100,000; hematocrit , 30%; BUN > 30 mg%; creatinine > 1.5 mg%; symptomatic liver disease with serum albumin < 3 g/dL.
- History of most recent creatine kinase >15x the upper limit of normal without any other explanation besides IBM.
- History of non-compliance with other therapies
- Use of testosterone except for physiologic replacement doses in case of androgen deficiency. Participants must have documented proof of the androgen deficiency.
- Coexistence of other disease that would be likely to affect outcome measures.
- Drug or alcohol abuse within past three months
- Participation in a recent drug study in the last 30 days prior to the screening visit or use of a biologic agent less than 6 months prior to the screening visit.
- Women who are lactating or unwilling to use adequate method of birth control who are not surgically sterile. Adequate birth control includes use of intrauterine device, abstinence, or oral contraceptives or a double barrier method, e.g condom plas diaphragm will be necessary for both male and female participants.
- Participants taking >7.5 mg prednisolone or equivalent or participants on IVIg or other immunosuppressants within the last 3 months.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02753530
United States, Arizona | |
Phoenix Neurological Associates | |
Phoenix, Arizona, United States, 85018 | |
United States, California | |
University of California, Irvine | |
Irvine, California, United States, 92697 | |
United States, Colorado | |
University of Colorado School of Medicine | |
Aurora, Colorado, United States, 80045 | |
United States, Kansas | |
University of Kansas Medical Center | |
Kansas City, Kansas, United States, 66160 | |
United States, Maryland | |
Johns Hopkins University | |
Baltimore, Maryland, United States, 21218 | |
United States, Massachusetts | |
Brigham and Women's Hospital | |
Boston, Massachusetts, United States, 02115 | |
United States, New York | |
University of Rochester | |
Rochester, New York, United States, 14642 | |
United States, Ohio | |
The Ohio State University | |
Columbus, Ohio, United States, 43221 | |
United States, Texas | |
Nerve and Muscle Center of Texas | |
Houston, Texas, United States, 77030 | |
United States, Utah | |
University of Utah | |
Salt Lake City, Utah, United States, 84112 | |
United States, Virginia | |
University of Virginia | |
Charlottesville, Virginia, United States, 22908 | |
United Kingdom | |
University College of London | |
London, United Kingdom, WC1N 3BG |
Principal Investigator: | Mazen Dimachkie, MD | University of Kansas Medical Center | |
Principal Investigator: | Michael Hanna, MD | University College, London |
Responsible Party: | Orphazyme |
ClinicalTrials.gov Identifier: | NCT02753530 |
Other Study ID Numbers: |
STUDY00002461 R01FD004809 ( U.S. FDA Grant/Contract ) |
First Posted: | April 28, 2016 Key Record Dates |
Last Update Posted: | March 24, 2021 |
Last Verified: | March 2021 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Myositis Myositis, Inclusion Body Muscular Diseases |
Musculoskeletal Diseases Neuromuscular Diseases Nervous System Diseases |