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Arimoclomol in Sporadic Inclusion Body Myositis - Open Label Extension Trial

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. Identifier: NCT04049097
Recruitment Status : Enrolling by invitation
First Posted : August 7, 2019
Last Update Posted : August 7, 2019
Information provided by (Responsible Party):

Tracking Information
First Submitted Date  ICMJE April 1, 2019
First Posted Date  ICMJE August 7, 2019
Last Update Posted Date August 7, 2019
Actual Study Start Date  ICMJE May 20, 2019
Estimated Primary Completion Date May 22, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 6, 2019)
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.
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: August 6, 2019)
  • 6-Minute Walk Test [ Time Frame: Change from Baseline to Month 20 ]
  • Modified Timed Up and Go (mTUG) [ Time Frame: Change from Baseline to Month 20 ]
  • Maximal Voluntary Isometric Contraction Testing (MVICT) [ Time Frame: Change from Baseline to Month 20 ]
    Muscle Strength Testing
  • Grip Strength Testing (Jamar) [ Time Frame: Change from Baseline to Month 20 ]
  • 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).
  • Number of Falls and Near Falls [ Time Frame: Change from Baseline to Month 20 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures
 (submitted: August 6, 2019)
  • 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
  • Magnetic Resonance Imaging (MRI) whole fat fraction of the thigh [ Time Frame: Change from Baseline to Month 20 ]
Original Other Pre-specified Outcome Measures Same as current
Descriptive Information
Brief Title  ICMJE Arimoclomol in Sporadic Inclusion Body Myositis - Open Label Extension Trial
Official Title  ICMJE 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
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
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Inclusion Body Myositis
Intervention  ICMJE Drug: Arimoclomol
1200 mg/day arimoclomol citrate (400 mg t.i.d.)
Study Arms  ICMJE Experimental: Arimoclomol
1200 mg/day arimoclomol citrate (400 mg t.i.d.)
Intervention: Drug: Arimoclomol
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Enrolling by invitation
Estimated Enrollment  ICMJE
 (submitted: August 6, 2019)
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE May 22, 2022
Estimated Primary Completion Date May 22, 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 45 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United Kingdom,   United States
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT04049097
Other Study ID Numbers  ICMJE IBM-OLE
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Orphazyme
Study Sponsor  ICMJE Orphazyme
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Orphazyme
Verification Date August 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP