Arimoclomol in Sporadic Inclusion Body Myositis - Open Label Extension Trial
Study Details
Study Description
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 |
|---|---|---|
| Phase 3 |
Study Design
Arms and Interventions
| 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.)
|
Outcome Measures
Primary Outcome Measures
- Change from baseline to Month 20 in the Inclusion Body Myositis Functional Rating Scale (IBMFRS) total score [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
- 6-Minute Walk Test [Change from Baseline to Month 20]
- Modified Timed Up and Go (mTUG) [Change from Baseline to Month 20]
- Maximal Voluntary Isometric Contraction Testing (MVICT) [Change from Baseline to Month 20]
Muscle Strength Testing
- Grip Strength Testing (Jamar) [Change from Baseline to Month 20]
- 36-Item Short Form Health Survey (SF-36) [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 [Change from Baseline to Month 20]
Other Outcome Measures
- Number of Treatment Emergent Adverse Events (TEAEs) in the open label extension [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 [Change from Baseline to Month 20]
Eligibility Criteria
Criteria
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 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.
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Significant protocol deviation in the blinded IBM4809 trial based on the investigator's judgement in discussion with the medical monitor.
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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.
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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).
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Any concurrent condition that in the investigator's opinion will significantly interfere with assessment of safety or efficacy.
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Inability to comply with the protocol-specified procedures/evaluations and scheduled visits as per the investigator.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Phoenix Neurological Associates | Phoenix | Arizona | United States | 85018 |
| 2 | University of Colorado School of Medicine | Aurora | Colorado | United States | 80045 |
| 3 | University of Kansas Medical Center | Kansas City | Kansas | United States | 66160 |
| 4 | Johns Hopkins University | Baltimore | Maryland | United States | 21218 |
| 5 | Brigham and Women's Hospital | Boston | Massachusetts | United States | 02115 |
| 6 | University of Rochester | Rochester | New York | United States | 14642 |
| 7 | The Ohio State University | Columbus | Ohio | United States | 43221 |
| 8 | Nerve and Muscle Center of Texas | Houston | Texas | United States | 77030 |
| 9 | University of Utah | Salt Lake City | Utah | United States | 84112 |
| 10 | University of Virginia | Charlottesville | Virginia | United States | 22908 |
| 11 | University College of London | London | United Kingdom | WC1N 3BG |
Sponsors and Collaborators
- KemPharm Denmark A/S
- University of Kansas Medical Center
- University College, London
Investigators
- Principal Investigator: Mazen M Dimachkie, University of Kansas Medical Center
- Principal Investigator: Michael Hanna, University College, London
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- IBM-OLE