Study of Arimoclomol in Inclusion Body Myositis (IBM)

• ClinicalTrials.gov Identifier: NCT02753530
• Recruitment Status: Completed
• First Posted: April 28, 2016
• Last Update Posted: March 24, 2021
• Sponsor: Orphazyme
• Collaborators: University of Kansas Medical Center; University College, London
• Information provided by (Responsible Party): Orphazyme

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Inclusion Body Myositis	Drug: Arimoclomol; Other: Placebo	Phase 2

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 152 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Double (Participant, Investigator)
• Primary Purpose: Treatment
• Official Title: Phase II Study of Arimoclomol for the Treatment of Sporadic Inclusion Body Myositis (IBM)
• Actual Study Start Date: August 16, 2017
• Actual Primary Completion Date: January 11, 2021
• Actual Study Completion Date: January 11, 2021

Arms and Interventions

Arm	Intervention/treatment
Experimental: Arimoclomol; Participants will be asked to take 400mg arimoclomol three times a day.	Drug: Arimoclomol; 2 200mg pills taken 3 times per day during breakfast, early afternoon and at bedtime
Placebo Comparator: Placebo; Participants will be asked to take 400mg placebo three times a day.	Other: Placebo; 2 matched placebo pills to be taken 3 times per day during breakfast, early afternoon and at bedtime

Outcome Measures

Primary Outcome Measures :

1. Decline in Inclusion body myositis functional rating scale (IBMFRS) [ Time Frame: Change from Baseline to Month 20 ]
   Measured by rate of decline in the IBMFRS between experimental and placebo groups. The IBMFRS is a 10 item questionnaire. Scores for each item range from 0 to 4. There is a total maximum score of 40 and minimum score of 0. The higher the score the better functional status of the person.

Secondary Outcome Measures :

1. Manual Muscle Testing (MMT) [ Time Frame: Change from Baseline to Month 20 ]
2. Maximum voluntary isometric contraction (MVICT) of quadriceps [ Time Frame: Change from Baseline to Month 20 ]
3. Grip and pinch test [ Time Frame: Change from Baseline to Month 20 ]
4. modified timed up and go (mTUG) [ Time Frame: Change from Baseline to Month 20 ]
5. 6 minute walk test with 2 minute distance captured [ Time Frame: Change from Baseline to Month 20 ]
6. Health Assessment Questionnaire (HAQ-DI) [ Time Frame: Change from Baseline to Month 20 ]
7. SF-36 [ Time Frame: Change from Baseline to Month 20 ]
   Measured using the Short form health survey with 36 items (SF-36).
8. Count of adverse events [ Time Frame: Change from Baseline to Month 20 ]
9. Decline in Inclusion body myositis functional rating scale (IBMFRS) [ Time Frame: Change from Baseline to Month 12 ]
   Measured by rate of decline in the IBMFRS between experimental and placebo groups. The IBMFRS is a 10 item questionnaire. Scores for each item range from 0 to 4. There is a total maximum score of 40 and minimum score of 0. The higher the score the better functional status of the person.

Eligibility Criteria

• Ages Eligible for Study: 45 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Locations

United States, Arizona

Phoenix Neurological Associates

Phoenix, Arizona, United States, 85018

United States, California

University of California, Irvine

Irvine, California, United States, 92697

United States, Colorado

University of Colorado School of Medicine

Aurora, Colorado, United States, 80045

United States, Kansas

University of Kansas Medical Center

Kansas City, Kansas, United States, 66160

United States, Maryland

Johns Hopkins University

Baltimore, Maryland, United States, 21218

United States, Massachusetts

Brigham and Women's Hospital

Boston, Massachusetts, United States, 02115

United States, New York

University of Rochester

Rochester, New York, United States, 14642

United States, Ohio

The Ohio State University

Columbus, Ohio, United States, 43221

United States, Texas

Nerve and Muscle Center of Texas

Houston, Texas, United States, 77030

United States, Utah

University of Utah

Salt Lake City, Utah, United States, 84112

United States, Virginia

University of Virginia

Charlottesville, Virginia, United States, 22908

United Kingdom

University College of London

London, United Kingdom, WC1N 3BG

Sponsors and Collaborators

Orphazyme

University of Kansas Medical Center

University College, London

Investigators

• Principal Investigator: Mazen Dimachkie, MD; University of Kansas Medical Center
• Principal Investigator: Michael Hanna, MD; University College, London

More Information

• Responsible Party: Orphazyme
• ClinicalTrials.gov Identifier: NCT02753530
• Other Study ID Numbers: STUDY00002461; R01FD004809 ( U.S. FDA Grant/Contract )
• First Posted: April 28, 2016
• Last Update Posted: March 24, 2021
• Last Verified: March 2021

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Myositis; Myositis, Inclusion Body; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases