Arimoclomol in Sporadic Inclusion Body Myositis - Open Label Extension Trial (NCT04049097) | Clinical Trial Compass
TerminatedPhase 3
Arimoclomol in Sporadic Inclusion Body Myositis - Open Label Extension Trial
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.
United States, United Kingdom121 participantsStarted 2019-05-20
Plain-language 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)
Who can participate
Age range
45 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. 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).
Exclusion criteria
. 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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score
Timeframe: Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks).
. 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.
. Inability to comply with the protocol-specified procedures/evaluations and scheduled visits as per the investigator.