Study of Arimoclomol in Inclusion Body Myositis (IBM)

Phase II Study of Arimoclomol for the Treatment of Sporadic Inclusion Body Myositis (IBM)

Sponsors

Lead Sponsor: Orphazyme

Collaborator: University of Kansas Medical Center
University College, London

Source Orphazyme
Brief Summary

Funding Source - FDA OOPD. The purpose of this study is to evaluate the safety and efficacy of the study drug, Arimoclomol in IBM patients.

Overall Status Completed
Start Date 2017-08-16
Completion Date 2021-01-11
Primary Completion Date 2021-01-11
Phase Phase 2
Study Type Interventional
Primary Outcome
Measure Time Frame
Decline in Inclusion body myositis functional rating scale (IBMFRS) Change from Baseline to Month 20
Secondary Outcome
Measure Time Frame
Manual Muscle Testing (MMT) Change from Baseline to Month 20
Maximum voluntary isometric contraction (MVICT) of quadriceps Change from Baseline to Month 20
Grip and pinch test Change from Baseline to Month 20
modified timed up and go (mTUG) Change from Baseline to Month 20
6 minute walk test with 2 minute distance captured Change from Baseline to Month 20
Health Assessment Questionnaire (HAQ-DI) Change from Baseline to Month 20
SF-36 Change from Baseline to Month 20
Count of adverse events Change from Baseline to Month 20
Decline in Inclusion body myositis functional rating scale (IBMFRS) Change from Baseline to Month 12
Enrollment 152
Condition
Intervention

Intervention Type: Drug

Intervention Name: Arimoclomol

Description: 2 200mg pills taken 3 times per day during breakfast, early afternoon and at bedtime

Arm Group Label: Arimoclomol

Intervention Type: Other

Intervention Name: Placebo

Description: 2 matched placebo pills to be taken 3 times per day during breakfast, early afternoon and at bedtime

Arm Group Label: Placebo

Eligibility

Criteria:

Inclusion Criteria: - Meet any of the European Neuromuscular Centre Inclusion Body Myositis research diagnostic criteria 2011 categories for IBM - Demonstrate being able to arise from a chair without support from another person or device - Able to ambulate at least 20 ft/6 meters with or without assistive device. Once arisen from the chair, participant may use any walking device, i.e. walker/frame, can, crutches, or braces. They cannot be supported by another person and cannot use furniture or wall for support. - Body weight of >= 40 kg - Pre-menopausal women must have a negative pregnancy tst prior to dosing iwht study medication. - If a participant in the bimagrumab study, the participant must be off of the study medication for at least 6 months. - Able to give informed consent Exclusion Criteria: - History of any of the following excludes subject participation in the study: chronic infection particularly HIV or Hepatitis B or C; cancer other than basal cell cancer less than five years prior; or other chronic serious medical illnesses. - Presence of any of the following on routine blood screening: WEB <3000; platelets < 100,000; hematocrit , 30%; BUN > 30 mg%; creatinine > 1.5 mg%; symptomatic liver disease with serum albumin < 3 g/dL. - History of most recent creatine kinase >15x the upper limit of normal without any other explanation besides IBM. - History of non-compliance with other therapies - Use of testosterone except for physiologic replacement doses in case of androgen deficiency. Participants must have documented proof of the androgen deficiency. - Coexistence of other disease that would be likely to affect outcome measures. - Drug or alcohol abuse within past three months - Participation in a recent drug study in the last 30 days prior to the screening visit or use of a biologic agent less than 6 months prior to the screening visit. - Women who are lactating or unwilling to use adequate method of birth control who are not surgically sterile. Adequate birth control includes use of intrauterine device, abstinence, or oral contraceptives or a double barrier method, e.g condom plas diaphragm will be necessary for both male and female participants. - Participants taking >7.5 mg prednisolone or equivalent or participants on IVIg or other immunosuppressants within the last 3 months.

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 California, Irvine | Irvine, California, 92697, 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

2021-03-01

Responsible Party

Type: Sponsor

Has Expanded Access No
Condition Browse
Number Of Arms 2
Arm Group

Label: Arimoclomol

Type: Experimental

Description: Participants will be asked to take 400mg arimoclomol three times a day.

Label: Placebo

Type: Placebo Comparator

Description: Participants will be asked to take 400mg placebo three times a day.

Study Design Info

Allocation: Randomized

Intervention Model: Parallel Assignment

Primary Purpose: Treatment

Masking: Double (Participant, Investigator)

Source: ClinicalTrials.gov