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Arimoclomol in Sporadic Inclusion Body Myositis - Open Label Extension Trial

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ClinicalTrials.gov Identifier: NCT04049097
Recruitment Status : Completed
First Posted : August 7, 2019
Last Update Posted : November 18, 2021
University of Kansas Medical Center
University College, London
Information provided by (Responsible Party):
KemPharm Denmark A/S

Brief Summary:
A multicenter, nonrandomized, open-label, uncontrolled clinical extension trial designed to compare the efficacy and safety of early versus delayed start of arimoclomol in the treatment of sIBM

Condition or disease Intervention/treatment Phase
Inclusion Body Myositis Drug: Arimoclomol Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 121 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label, Non-randomized Trial to Investigate the Efficacy and Safety of Early Versus Delayed Start of Arimoclomol in Patients With Sporadic Inclusion Body Myositis Who Have Completed the IBM4809 Trial
Actual Study Start Date : May 20, 2019
Actual Primary Completion Date : November 15, 2021
Actual Study Completion Date : November 15, 2021

Arm Intervention/treatment
Experimental: Arimoclomol
1200 mg/day arimoclomol citrate (400 mg t.i.d.)
Drug: Arimoclomol
1200 mg/day arimoclomol citrate (400 mg t.i.d.)

Primary Outcome Measures :
  1. Change from baseline to Month 20 in the Inclusion Body Myositis Functional Rating Scale (IBMFRS) total score [ Time Frame: Change from Baseline to Month 20 ]
    The IBMFRS includes 10 measures (swallowing, handwriting, cutting food and handling utensils, fine motor tasks, dressing, hygiene, turning in bed and adjusting covers, changing position from sitting to standing, walking, and climbing stairs), each graded on a Likert scale from 0 (being unable to perform) to 4 (normal). The sum of the 10 items gives a value between 0 and 40.

Secondary Outcome Measures :
  1. 6-Minute Walk Test [ Time Frame: Change from Baseline to Month 20 ]
  2. Modified Timed Up and Go (mTUG) [ Time Frame: Change from Baseline to Month 20 ]
  3. Maximal Voluntary Isometric Contraction Testing (MVICT) [ Time Frame: Change from Baseline to Month 20 ]
    Muscle Strength Testing

  4. Grip Strength Testing (Jamar) [ Time Frame: Change from Baseline to Month 20 ]
  5. 36-Item Short Form Health Survey (SF-36) [ Time Frame: Change from Baseline to Month 20 ]

    Measure of functional health and well-being from the patient's point of view by the 36-Item short form health survey scale (SF-36).

    For each of the eight domains that the SF-36 measures an aggregate percentage score is produced. The percentage scores range from 0% (lowest or worst possible level of functioning) to 100% (highest or best possible level of functioning).

  6. Number of Falls and Near Falls [ Time Frame: Change from Baseline to Month 20 ]

Other Outcome Measures:
  1. Number of Treatment Emergent Adverse Events (TEAEs) in the open label extension [ Time Frame: Baseline (start of OLE, week 0) to end of OLE (week 20) ]
    Number of Treatment Emergent Adverse Events (TEAEs) in the open label extension

  2. Magnetic Resonance Imaging (MRI) whole fat fraction of the thigh [ Time Frame: Change from Baseline to Month 20 ]

Information from the National Library of Medicine

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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:

  1. Patient is able to comprehend and is willing to provide written informed consent and is capable and willing to comply with trial procedures.
  2. Patient has completed the IBM4809 trial on treatment with IMP. -

Exclusion Criteria:

  1. Known or suspected allergy or intolerance to arimoclomol or its constituents.
  2. Exposure to any other investigational treatment within 30 days or <5 half-lives of the baseline visit or taking part or planning to take part in another interventional trial.
  3. Significant protocol deviation in the blinded IBM4809 trial based on the investigator's judgement in discussion with the medical monitor.
  4. Women who are lactating or pregnant, or men or women unwilling to use a highly effective method of birth control if not surgically sterile (defined as bilateral tubal ligation, bilateral oophorectomy, or hysterectomy for women; vasectomy for men) for female participants until 4 weeks after last dose and for male participants up to 3 months after last dose. Premenopausal women must have a negative pregnancy test prior to dosing with trial medication. Acceptable methods of birth control are:

    • Hormonal methods associated with inhibition of ovulation such as oral, implantable, injectable, or transdermal contraceptives for a minimum of 1 full cycle (based on the patient's usual menstrual cycle period) before arimoclomol administration.
    • Total abstinence from sexual intercourse since the last menses before arimoclomol administration. (The reliability of sexual abstinence needs to be evaluated in relation to the duration of the clinical trial and the preferred and usual lifestyle of the patient. Periodic abstinence [calendar, symptothermal, post-ovulation] methods are not acceptable methods of contraception).
    • Intrauterine device (IUD) or intrauterine hormone-releasing system (IUS).
  5. Any concurrent condition that in the investigator's opinion will significantly interfere with assessment of safety or efficacy.
  6. Inability to comply with the protocol-specified procedures/evaluations and scheduled visits as per the investigator.

Information from the National Library of Medicine

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): NCT04049097

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United States, Arizona
Phoenix Neurological Associates
Phoenix, Arizona, United States, 85018
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
Sponsors and Collaborators
KemPharm Denmark A/S
University of Kansas Medical Center
University College, London
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Principal Investigator: Mazen M Dimachkie University of Kansas Medical Center
Principal Investigator: Michael Hanna University College, London
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Responsible Party: KemPharm Denmark A/S
ClinicalTrials.gov Identifier: NCT04049097    
Other Study ID Numbers: IBM-OLE
First Posted: August 7, 2019    Key Record Dates
Last Update Posted: November 18, 2021
Last Verified: November 2021

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Myositis, Inclusion Body
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases