Study of Arimoclomol in Inclusion Body Myositis (IBM)

April 18, 2023 updated by: ZevraDenmark

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

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

Study Type

Interventional

Enrollment (Actual)

152

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United Kingdom
      • London, United Kingdom, WC1N 3BG
        • University College of London
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85018
        • Phoenix Neurological Associates
    • California
      • Irvine, California, United States, 92697
        • University of California, Irvine
    • Colorado
      • Aurora, Colorado, United States, 80045
        • University of Colorado School of Medicine
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • University of Kansas Medical Center
    • Maryland
      • Baltimore, Maryland, United States, 21218
        • Johns Hopkins University
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Brigham and Women's Hospital
    • New York
      • Rochester, New York, United States, 14642
        • University of Rochester
    • Ohio
      • Columbus, Ohio, United States, 43221
        • The Ohio State University
    • Texas
      • Houston, Texas, United States, 77030
        • Nerve and Muscle Center of Texas
    • Utah
      • Salt Lake City, Utah, United States, 84112
        • University of Utah
    • Virginia
      • Charlottesville, Virginia, United States, 22908
        • University of Virginia

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

43 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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.
  • Age at onset of weakness >45 years.
  • Body weight of >= 40 kg.
  • 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: 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
  • 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Arimoclomol
248 mg arimoclomol base (equivalent to 400 mg arimoclomol citrate) 3 times daily
Drug: Arimoclomol
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
Placebo Comparator: Placebo
248 mg matching placebo 3 times daily
Other: Placebo
2 matched placebo capsules taken 3 times daily during breakfast, early afternoon, and at bedtime

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score
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.
Change from Baseline to Month 20

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score
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.
Change from Baseline to Month 12
Grip Strength
Time Frame: Change from Baseline to Month 12 and 20
Unilateral hand grip strength in both hands measured using the Jamar Dynamometer. Results are for the stronger limb, as identified at baseline.
Change from Baseline to Month 12 and 20
Modified Timed up and go (mTUG)
Time Frame: Change from Baseline to Month 12 and Month 20
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.
Change from Baseline to Month 12 and Month 20
Manual Muscle Testing (MMT), Total Score
Time Frame: Change from Baseline to Month 12 and Month 20
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.
Change from Baseline to Month 12 and Month 20
6 Minute Walk Test (6MWT); Distance After 6 Minutes (6MWD)
Time Frame: Change from Baseline to Month 12 and Month 20
The distance patients could walk in 6 minutes. The distance walked in meters was recorded after 6 minutes.
Change from Baseline to Month 12 and Month 20
Short Form-36 (SF-36) Physical Component Score
Time Frame: Change from Baseline to Month 12 and Month 20
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.
Change from Baseline to Month 12 and Month 20
Maximum Voluntary Isometric Contraction (MVICT) of Quadriceps
Time Frame: Change from Baseline to Month 12 and Month 20
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.
Change from Baseline to Month 12 and Month 20
Health Assessment Questionnaire - Disability Index (HAQ-DI)
Time Frame: Change from Baseline to Month 12 and Month 20
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.
Change from Baseline to Month 12 and Month 20
2 Minute Walk Test (2MWT)
Time Frame: Change from Baseline to Month 12 and Month 20
The distance patients could walk in 2 minutes (during the 6 Minute Walk Test) recorded in meters.
Change from Baseline to Month 12 and Month 20
Short Form-36 (SF-36) Mental Component Score
Time Frame: Change from Baseline to Month 12 and Month 20
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.
Change from Baseline to Month 12 and Month 20
Patient Global Impression of Severity (PGIS)
Time Frame: Change from Baseline to Month 12 and Month 20
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".
Change from Baseline to Month 12 and Month 20
Patient Global Impression of Change (PGIC)
Time Frame: Change from Baseline to Month 12 and Month 20
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".
Change from Baseline to Month 12 and Month 20
Clinician Global Impression of Severity (CGIS)
Time Frame: Change from Baseline to Month 12 and Month 20
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"
Change from Baseline to Month 12 and Month 20
Clinician Global Impression of Change (CGIC)
Time Frame: Change from Baseline to Month 12 and Month 20
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".
Change from Baseline to Month 12 and Month 20
Falls and Near Falls
Time Frame: Accumulated number from Baseline to Month 20
Falls and near falls registered by the participants in a diary
Accumulated number from Baseline to Month 20

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

ZevraDenmark

Collaborators

University College, London
University of Kansas Medical Center

Investigators

  • Principal Investigator: Michael Hanna, MD, University College, London
  • Principal Investigator: Mazen Dimachkie, MD, University of Kansas Medical Center

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 16, 2017

Primary Completion (Actual)

January 11, 2021

Study Completion (Actual)

January 11, 2021

Study Registration Dates

First Submitted

April 25, 2016

First Submitted That Met QC Criteria

April 27, 2016

First Posted (Estimate)

April 28, 2016

Study Record Updates

Last Update Posted (Actual)

May 10, 2023

Last Update Submitted That Met QC Criteria

April 18, 2023

Last Verified

April 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • STUDY00002461
  • R01FD004809 (U.S. FDA Grant/Contract)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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