- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04049097
Arimoclomol in Sporadic Inclusion Body Myositis - Open Label Extension Trial
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
Study Overview
Detailed Description
Study Type
Enrollment (Actual)
Phase
- Phase 3
Contacts and Locations
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
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
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.
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).
- 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.
Study Plan
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
Sponsor
Investigators
- Principal Investigator: Mazen M Dimachkie, University of Kansas Medical Center
- Principal Investigator: Michael Hanna, University College, London
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- IBM-OLE
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
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.
Clinical Trials on Inclusion Body Myositis
-
Novartis PharmaceuticalsCompletedSporadic Inclusion Body Myositis (sIBM)United States
-
Austin Neuromuscular CenterRegeneron PharmaceuticalsNot yet recruitingIdiopathic Inflammatory Myopathies | Sporadic Inclusion Body Myositis (sIBM)United States
-
Abcuro, Inc.RecruitingInclusion Body Myositis (IBM)United States, Australia
-
University of Southern DenmarkOdense University HospitalCompleted
-
University of Kansas Medical CenterCompletedInclusion Body Myositis | Sporadic Inclusion Body MyositisUnited States
-
Novartis PharmaceuticalsCompletedSporadic Inclusion Body MyositisUnited States
-
Institut National de la Santé Et de la Recherche...CompletedInclusion Body Myositis (IBM)France
-
La-ser Europe LimitedUnknownSporadic Inclusion Body Myositis
-
Oslo University HospitalThe Dam Foundation; The Norwegian Rheumatism AssociationRecruitingFeasibility Study | Inclusion Body Myositis (IBM)Norway
-
Assistance Publique - Hôpitaux de ParisNot yet recruitingInclusion Body Myositis, Sporadic
Clinical Trials on Arimoclomol
-
CytRxCompletedAmyotrophic Lateral Sclerosis (ALS)United States
-
Richard Barohn, MDCompletedInclusion Body MyositisUnited States, United Kingdom
-
University of MiamiMassachusetts General HospitalCompletedAmyotrophic Lateral SclerosisUnited States
-
CytRxWithdrawnAmyotrophic Lateral SclerosisUnited States, Canada
-
ZevraDenmarkAvailableNiemann-Pick Disease, Type CUnited States
-
KemPharm Denmark A/STerminatedGaucher Disease, Type 1 | Gaucher Disease, Type 3India
-
ZevraDenmarkTerminatedAmyotrophic Lateral SclerosisUnited States, Canada, Netherlands, Belgium, Poland, United Kingdom, France, Germany, Italy, Spain, Sweden
-
ZevraDenmarkActive, not recruitingNiemann-Pick Disease, Type CUnited States, Spain, United Kingdom, Denmark, France, Germany, Italy, Poland, Switzerland
-
ZevraDenmarkCompletedAmyotrophic Lateral SclerosisCanada, United States, Belgium, Netherlands, Poland, United Kingdom, France, Germany, Italy, Spain, Sweden, Switzerland
-
ZevraDenmarkUniversity College, London; University of Kansas Medical CenterCompletedInclusion Body MyositisUnited States, United Kingdom