Arimoclomol in Sporadic Inclusion Body Myositis - Open Label Extension Trial

August 22, 2023 updated by: ZevraDenmark

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

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 Inclusion Body Myositis (IBM)

Study Overview

Status

Terminated

Intervention / Treatment

Detailed Description

This was planned to be a 40-month open-label extension trial of the 20-month randomized, double-blind, placebo-controlled IBM4809 trial. The open-label trial was terminated early by the sponsor as a consequence of the results of IBM4809 which did not meet any of its efficacy endpoints. Therefore, the actual average duration of open-label treatment was approximately 28 weeks.

Study Type

Interventional

Enrollment (Actual)

121

Phase

  • Phase 3

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

      • London, United Kingdom, WC1N 3BG
        • University College of London
    • Arizona
      • Phoenix, Arizona, United States, 85018
        • Phoenix Neurological Associates
    • California
      • Irvine, California, United States, 92697
        • University of California
    • 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:

  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 Investigational Medicinal Product (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.

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: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Arimoclomol
248 mg arimoclomol base (equivalent to 400 mg arimoclomol citrate) 3 times daily
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

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 in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).

The Inclusion Body Myositis Functional Rating Scale (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. A higher score represents less functional limitation.

After the study was terminated early by the sponsor, the analyses of the efficacy endpoints were simplified from what was planned in the study protocol and data were only summarized descriptively.

Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in Six Minutes Walking Distance Test; Distance at 6 Minutes (6MWD)
Time Frame: Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).

Patients were instructed to walk down one side of a track and back along the opposite side as quickly and safely as possible for 6 minutes. Patients were allowed to take breaks as needed during the walking period, but timing continued during breaks. The distance walked in meters was recorded after 6 minutes.

After the study was terminated early by the sponsor, the analyses of the efficacy endpoints were simplified from what was planned in the study protocol and data were only summarized descriptively.

Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).
Change in Modified Timed Up and Go (mTUG)
Time Frame: Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).

The Modified Timed Up and Go (mTUG) measures the patient's ability to get up from a chair (allowing patients to use their arms), walk 3 meters, turn around, walk back to the chair, and sit down. The use of nearby walls or assistance from a caregiver was not allowed.

After the study was terminated early by the sponsor, the analyses of the efficacy endpoints were simplified from what was planned in the study protocol and data were only summarized descriptively.

Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).
Change in Quadriceps Muscle Strength
Time Frame: Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).

Maximal voluntary isometric contraction testing (MVICT) of the patient's quadriceps muscle (extensor strength of the knee) were performed using a hand myometer which is a hand-held device that allows the examiner to push against a muscle while the patient resists. The test was performed on each side. The results for the stronger knee are reported here.

After the study was terminated early by the sponsor, the analyses of the efficacy endpoints were simplified from what was planned in the study protocol and data were only summarized descriptively.

Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).
Change in Hand Grip Strength
Time Frame: Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).

Hand grip strength was assessed using a dynamometer. The test was performed on each hand. The grip strength of the stronger hand is reported here.

After the study was terminated early by the sponsor, the analyses of the efficacy endpoints were simplified from what was planned in the study protocol and data were only summarized descriptively.

Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).
Change in the 36-Item Short Form Health Survey (SF-36)
Time Frame: Change from Baseline in IBM-OLE to Early Termination Visit

The 36-Item Short Form Health Survey (SF-36) is a 36-item, patient-reported survey of health status.

After the study was terminated early by the sponsor, the analyses of the efficacy endpoints were simplified from what was planned in the study protocol. No derived scores were calculated for SF-36 and no summary tabulation was done.

Change from Baseline in IBM-OLE to Early Termination Visit
Number of Falls and Near Falls
Time Frame: Baseline to Early Termination Visit
Patients recorded the number of falls and near falls in a falls diary. After the study was terminated early by the sponsor, the analyses of the efficacy endpoints were simplified from what was planned in the study protocol. No tabulation of falls and near falls was performed.
Baseline to Early Termination Visit

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Baseline (start of OLE, week 0) to end of OLE (week 20)
Magnetic Resonance Imaging (MRI) whole fat fraction of the thigh
Time Frame: Change from Baseline to Month 20
Change from Baseline to Month 20

Collaborators and Investigators

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

Sponsor

Investigators

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

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)

May 20, 2019

Primary Completion (Actual)

November 15, 2021

Study Completion (Actual)

November 15, 2021

Study Registration Dates

First Submitted

April 1, 2019

First Submitted That Met QC Criteria

August 6, 2019

First Posted (Actual)

August 7, 2019

Study Record Updates

Last Update Posted (Actual)

September 15, 2023

Last Update Submitted That Met QC Criteria

August 22, 2023

Last Verified

August 1, 2023

More Information

Terms related to this study

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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