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Inclusion Body MyositisPhase 2NCT02753530

Study of Arimoclomol in Inclusion Body Myositis (IBM)

A Phase 2 study testing Arimoclomol in people with Inclusion Body Myositis, run by ZevraDenmark at 12 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Funding Source - FDA Office of Orphan Products Development (OOPD). The purpose of this study is to evaluate the safety and efficacy of the study drug, arimoclomol in IBM patients. Read more about this study

Who is it for?
Ages 45 years and older
Conditions the study is for
Inclusion Body Myositis
What is being tested?
Arimoclomol
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
152 participants
Where
12 sitesin 2 countries
Study dates
16 Aug 2017to 11 Jan 2021
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 45 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.
  • Age at onset of weakness >45 years.
  • Body weight of >= 40 kg.
  • Able to give informed consent.

Who cannot join

  • 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: White blood cells (WBC) <3000; platelets < 100,000; hematocrit <30%; blood urea nitrogen (BUN) >30 mg/dL; creatinine >1.5 x upper limit of normal; 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
Show all 11Show fewer
  • 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 plus diaphragm will be necessary for both male and female participants.
  • Participants taking >7.5 mg prednisolone or equivalent or participants on Intravenous Immunoglobulin Therapy (IVIg) or other immunosuppressants within the last 3 months.
  • Clinically significant renal or hepatic disease, as indicated by clinical laboratory assessment.

Trial locations and contacts

12 sites in 2 countries.

12 sites in 2 countries

United States11 sites

  • University of Colorado School of Medicine

    Aurora, Colorado, 80045

  • Johns Hopkins University

    Baltimore, Maryland, 21218

  • Brigham and Women's Hospital

    Boston, Massachusetts, 02115

  • University of Virginia

    Charlottesville, Virginia, 22908

  • The Ohio State University

    Columbus, Ohio, 43221

  • Nerve and Muscle Center of Texas

    Houston, Texas, 77030

  • University of California, Irvine

    Irvine, California, 92697

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160

  • Phoenix Neurological Associates

    Phoenix, Arizona, 85018

  • University of Rochester

    Rochester, New York, 14642

  • University of Utah

    Salt Lake City, Utah, 84112

Other trials in United States

United Kingdom1 site

  • University College of London

    London, WC1N 3BG

Other trials in United Kingdom

About this study

Official title: Phase II Study of Arimoclomol for the Treatment of Sporadic Inclusion Body Myositis (IBM)

Funding Source - FDA Office of Orphan Products Development (OOPD). The purpose of this study is to evaluate the safety and efficacy of the study drug, arimoclomol in IBM patients.

Read the detailed description

A Phase 2/3, randomized, double-blind, placebo-controlled, international, 20-month intervention study in patients with IBM.

Being tested
Arimoclomol, Placebo
Keywords
IBM
Medical subject headings
Myositis, Inclusion Body

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ArimoclomolDrug

    2 capsules (2 x 124 mg arimoclomol base; equivalent to 2 x 200 mg arimoclomol citrate) taken 3 times daily during breakfast, early afternoon, and at bedtime

  • PlaceboOther

    2 matched placebo capsules taken 3 times daily during breakfast, early afternoon, and at bedtime

Study groups in NCT02753530
Study groupRoleWhat they receive
ArimoclomolExperimental248 mg arimoclomol base (equivalent to 400 mg arimoclomol citrate) 3 times daily
Receives: Drug: Arimoclomol
PlaceboPlacebo comparator248 mg matching placebo 3 times daily
Receives: Other: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score

    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.

    Measured: Change from Baseline to Month 20

Secondary outcomes (15)

  1. Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score

    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.

    Measured: Change from Baseline to Month 12

  2. Grip Strength

    Unilateral hand grip strength in both hands measured using the Jamar Dynamometer. Results are for the stronger limb, as identified at baseline.

    Measured: Change from Baseline to Month 12 and 20

  3. Modified Timed up and go (mTUG)

    The patient's combined ability to rise from a chair and walk 3 meters, turn around and walk back to the chair and sit down. The test was performed twice and the fastest time was used. The results were expressed as velocity in meters/second.

    Measured: Change from Baseline to Month 12 and Month 20

  4. Manual Muscle Testing (MMT), Total Score

    The Manual Muscle Testing (MMT) scores the strength of 24 muscles (axial, proximal, and distal muscles, tested bilaterally) on a scale from 0 to 10 points. The total score is calculated as an average across the 24 muscles and ranges from 0 to 10. The total score will increase if a patient is getting stronger and decrease if a patient is getting weaker.

    Measured: Change from Baseline to Month 12 and Month 20

  5. 6 Minute Walk Test (6MWT); Distance After 6 Minutes (6MWD)

    The distance patients could walk in 6 minutes. The distance walked in meters was recorded after 6 minutes.

    Measured: Change from Baseline to Month 12 and Month 20

  6. Short Form-36 (SF-36) Physical Component Score

    Measured using the Short Form health survey with 36 items (SF-36). The questionnaire measures 8 health concepts which yields 2 summary measures: physical and mental health. The physical component score includes 4 scales of physical functioning (10 items), role limitations due to physical health (4 items), bodily pain (2 items), and general health (5 items). To score the SF-36, scales are standardized with a scoring algorithm to obtain a score ranging from 0 to 100, and the summary measure is calculated as the average score. Higher scores indicate better health status.

    Measured: Change from Baseline to Month 12 and Month 20

  7. Maximum Voluntary Isometric Contraction (MVICT) of Quadriceps

    Unilateral strength of the knee extensor muscles on both limbs using the MicroFET hand-held dynamometer. Results are for the stronger limb, as identified at baseline.

    Measured: Change from Baseline to Month 12 and Month 20

  8. Health Assessment Questionnaire - Disability Index (HAQ-DI)

    The disability index of the HAQ measures self-reported functional status (disability) including the patient's use of aids or devices and/or help from other persons. The scale is composed of 20 items in 8 domains (dressing and grooming, arising, eating, walking, hygiene, reach, grip, and common daily activities). Each domain has at least 2 subcategory items, scored on a scale from 0 to 3, and for each of the 8 domains the domain score was the highest score of the involved subcategory scores. The total score reported is an average over 8 domains and scores from at least 6 domains had to be available for the total score to be calculated. The average total score ranges from 0 to 3, and a higher score corresponds to a worsening in functional status.

    Measured: Change from Baseline to Month 12 and Month 20

  9. 2 Minute Walk Test (2MWT)

    The distance patients could walk in 2 minutes (during the 6 Minute Walk Test) recorded in meters.

    Measured: Change from Baseline to Month 12 and Month 20

  10. Short Form-36 (SF-36) Mental Component Score

    Measured using the Short Form health survey with 36 items (SF-36). The questionnaire measures 8 health concepts which yields 2 summary measures: physical and mental health. The mental component score is composed of energy/fatigue (4 items), social functioning (2 items), role limitations due to emotional problems (3 items), and emotional well-being (5 items). To score the SF-36, scales are standardized with a scoring algorithm to obtain a score ranging from 0 to 100, and the summary measure is calculated as the average score. Higher scores indicate better health status.

    Measured: Change from Baseline to Month 12 and Month 20

  11. Patient Global Impression of Severity (PGIS)

    Patient-reported assessment of the impact of IBM on the ability to complete activities of daily living (e.g. dressing, walking, bathing) at the time of the assessment. The response options for the impact of IBM were "none" (i.e. no impact), "very mild", "mild", "moderate", "severe", and "very severe".

    Measured: Change from Baseline to Month 12 and Month 20

  12. Patient Global Impression of Change (PGIC)

    Patient-reported assessment of the change from start of study treatment in the impact of IBM on the ability to complete activities of daily living (e.g. dressing, walking, bathing). The response options for change in impact were "very much worse", "much worse", "a little worse", "no change", "a little improved", "much improved", and "very much improved".

    Measured: Change from Baseline to Month 12 and Month 20

  13. Clinician Global Impression of Severity (CGIS)

    Clinician-reported assessment of the severity of the patient's IBM symptoms at the time of the assessment. The response options were "none", "very mild", "mild", "moderate", "severe", and "very severe"

    Measured: Change from Baseline to Month 12 and Month 20

  14. Clinician Global Impression of Change (CGIC)

    Clinician-reported assessment of the change from start of study treatment in the patient's IBM symptoms. The response options for change in IBM were "very much worse", "much worse", "a little worse", "no change", "a little improved", "much improved", and "very much improved".

    Measured: Change from Baseline to Month 12 and Month 20

  15. Falls and Near Falls

    Falls and near falls registered by the participants in a diary

    Measured: Accumulated number from Baseline to Month 20

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted28 Apr 2016 (estimated)
  2. Study start16 Aug 2017
  3. Primary completion11 Jan 2021
  4. Study completion11 Jan 2021

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
152 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02753530
Sponsor's protocol ID
STUDY00002461
Other IDs
R01FD004809
Status last verified
Apr 2023
First submitted
25 Apr 2016
Last updated
10 May 2023
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Salam S, Symonds T, Doll H, Rousell S, Randall J, Lloyd-Price L, Hudgens S, Guldberg C, Herbelin L, Barohn RJ, Hanna MG, Dimachkie MM, Machado PM; Members of the Arimoclomol in IBM Investigator Team of the Neuromuscular Study Group. Measurement properties of the Inclusion Body Myositis Functional Rating Scale. J Neurol Neurosurg Psychiatry. 2025 Jan 16;96(2):122-131. doi: 10.1136/jnnp-2024-333617.

    PubMed 38960586

  • Machado PM, McDermott MP, Blaettler T, Sundgreen C, Amato AA, Ciafaloni E, Freimer M, Gibson SB, Jones SM, Levine TD, Lloyd TE, Mozaffar T, Shaibani AI, Wicklund M, Rosholm A, Carstensen TD, Bonefeld K, Jorgensen AN, Phonekeo K, Heim AJ, Herbelin L, Barohn RJ, Hanna MG, Dimachkie MM; Arimoclomol in IBM Investigator Team of the Neuromuscular Study Group. Safety and efficacy of arimoclomol for inclusion body myositis: a multicentre, randomised, double-blind, placebo-controlled trial. Lancet Neurol. 2023 Oct;22(10):900-911. doi: 10.1016/S1474-4422(23)00275-2.

    PubMed 37739573

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Arimoclomol in people with Inclusion Body Myositis, run by ZevraDenmark at 12 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 10 May 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 12 sites in United States, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 10 May 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score (time frame: Change from Baseline to Month 20). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02753530, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 May 2023.

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