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

Sponsors

Lead Sponsor: Orphazyme

Collaborator: University of Kansas Medical Center
University College, London

Source Orphazyme
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

Overall Status Enrolling by invitation
Start Date 2019-05-20
Completion Date 2022-05-22
Primary Completion Date 2022-05-22
Phase Phase 3
Study Type Interventional
Primary Outcome
Measure Time Frame
Change from baseline to Month 20 in the Inclusion Body Myositis Functional Rating Scale (IBMFRS) total score Change from Baseline to Month 20
Secondary Outcome
Measure Time Frame
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
Grip Strength Testing (Jamar) Change from Baseline to Month 20
36-Item Short Form Health Survey (SF-36) Change from Baseline to Month 20
Number of Falls and Near Falls Change from Baseline to Month 20
Enrollment 150
Condition
Intervention

Intervention Type: Drug

Intervention Name: Arimoclomol

Description: 1200 mg/day arimoclomol citrate (400 mg t.i.d.)

Arm Group Label: Arimoclomol

Eligibility

Criteria:

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 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.

Gender:

All

Minimum Age:

45 Years

Maximum Age:

N/A

Healthy Volunteers:

No

Overall Official
Location
Facility:
Phoenix Neurological Associates | Phoenix, Arizona, 85018, United States
University of Colorado School of Medicine | Aurora, Colorado, 80045, United States
University of Kansas Medical Center | Kansas City, Kansas, 66160, United States
Johns Hopkins University | Baltimore, Maryland, 21218, United States
Brigham and Women's Hospital | Boston, Massachusetts, 02115, United States
University of Rochester | Rochester, New York, 14642, United States
The Ohio State University | Columbus, Ohio, 43221, United States
Nerve and Muscle Center of Texas | Houston, Texas, 77030, United States
University of Utah | Salt Lake City, Utah, 84112, United States
University of Virginia | Charlottesville, Virginia, 22908, United States
University College of London | London, WC1N 3BG, United Kingdom
Location Countries

United Kingdom

United States

Verification Date

2020-11-01

Responsible Party

Type: Sponsor

Has Expanded Access No
Condition Browse
Number Of Arms 1
Arm Group

Label: Arimoclomol

Type: Experimental

Description: 1200 mg/day arimoclomol citrate (400 mg t.i.d.)

Study Design Info

Allocation: N/A

Intervention Model: Single Group Assignment

Primary Purpose: Treatment

Masking: None (Open Label)

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