Inclusion Body MyositisPhase 3NCT04049097
Arimoclomol in Sporadic Inclusion Body Myositis - Open Label Extension Trial
A Phase 3 study testing Arimoclomol in people with Inclusion Body Myositis, run by ZevraDenmark at 12 sites in 2 countries.
Trial 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 Inclusion Body Myositis (IBM) 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?
- No, everyone receives an active treatment
- How many people
- 121 participants
- Where
- 12 sitesin 2 countries
- Study dates
- 20 May 2019to 15 Nov 2021
- Phase
- Phase 3
- 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
- Patient is able to comprehend and is willing to provide written informed consent and is capable and willing to comply with trial procedures.
- Patient has completed the IBM4809 trial on treatment with Investigational Medicinal Product (IMP).
Who cannot join
- Known or suspected allergy or intolerance to arimoclomol or its constituents.
- 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.
- Significant protocol deviation in the blinded IBM4809 trial based on the investigator's judgement in discussion with the medical monitor.
- 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).
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- Intrauterine device (IUD) or intrauterine hormone-releasing system (IUS).
- Any concurrent condition that in the investigator's opinion will significantly interfere with assessment of safety or efficacy.
- Inability to comply with the protocol-specified procedures/evaluations and scheduled visits as per the investigator.
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, 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
About this study
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
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)
Read the 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.
- Conditions
- Inclusion Body Myositis
- Being tested
- Arimoclomol
- 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
| Study group | Role | What they receive |
|---|---|---|
| Arimoclomol | Experimental | 248 mg arimoclomol base (equivalent to 400 mg arimoclomol citrate) 3 times daily Receives: Drug: Arimoclomol |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score
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.
Measured: Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).
Secondary outcomes (6)
Change in Six Minutes Walking Distance Test; Distance at 6 Minutes (6MWD)
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.
Measured: 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)
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.
Measured: Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).
Change in Quadriceps Muscle Strength
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.
Measured: Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).
Change in Hand Grip Strength
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.
Measured: 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)
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.
Measured: Change from Baseline in IBM-OLE to Early Termination Visit
Number of Falls and Near Falls
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.
Measured: Baseline to Early Termination Visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Aug 2019
- Study start20 May 2019
- Primary completion15 Nov 2021
- Study completion15 Nov 2021
Why it stopped: As a consequence of the results of IBM4809 which did not meet any of its efficacy endpoints. The planned duration of open-label treatment was 40 months. After termination, the actual mean duration of treatment was approx. 28 weeks.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Inclusion Body MyositisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 121 participants (actual)
Sponsor and investigators
- Lead sponsor
- ZevraDenmark
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Mazen M Dimachkie, Principal investigator, University of Kansas Medical CenterMichael Hanna, Principal investigator, University College, London
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04049097
- Sponsor's protocol ID
- IBM-OLE
- Status last verified
- Aug 2023
- First submitted
- 1 Apr 2019
- Last updated
- 15 Sep 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04049097
- Acronym / sponsor ID
- IBM-OLE
- Conditions
- Inclusion Body Myositis
- Keywords
- IBM
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: As a consequence of the results of IBM4809 which did not meet any of its efficacy endpoints. The planned duration of open-label treatment was 40 months. After termination, the actual mean duration of treatment was approx. 28 weeks.
- Enrolment
- 121 participants (actual)
- Lead sponsor
- ZevraDenmarkIndustry (pharma, biotech, devices)
- Collaborators
- University of Kansas Medical Center, University College, London
- Study start
- 20 May 2019 (actual)
- Primary completion
- 15 Nov 2021 (actual)
- Study completion
- 15 Nov 2021 (actual)
- First posted
- 7 Aug 2019 (actual)
- Results
- Posted 15 Sep 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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)
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Arimoclomol | Experimental | Drug: Arimoclomol248 mg arimoclomol base (equivalent to 400 mg arimoclomol citrate) 3 times daily |
Who may qualify
- Ages
- 45 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Patient is able to comprehend and is willing to provide written informed consent and is capable and willing to comply with trial procedures.
- Patient has completed the IBM4809 trial on treatment with Investigational Medicinal Product (IMP).
Key exclusion criteria (9 listed)
- Known or suspected allergy or intolerance to arimoclomol or its constituents.
- 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.
- Significant protocol deviation in the blinded IBM4809 trial based on the investigator's judgement in discussion with the medical monitor.
- 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:
- Any concurrent condition that in the investigator's opinion will significantly interfere with assessment of safety or efficacy.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Arimoclomol2 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 | Drug | Arimoclomol |
Primary outcome
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.
6 secondary outcomes
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).
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).
Change in Quadriceps Muscle Strength
Time frame: 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).
Change in the 36-Item Short Form Health Survey (SF-36)
Time frame: Change from Baseline in IBM-OLE to Early Termination Visit
Number of Falls and Near Falls
Time frame: Baseline to Early Termination Visit
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Sep 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mazen M Dimachkie principal investigator
University of Kansas Medical Center
Michael Hanna principal investigator
University College, London
12 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 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 2 inclusion and 9 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: stopped early, as of the sponsor's last update on 15 Sep 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. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 15 Sep 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 in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 15 Sep 2023.