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Study of Arimoclomol in Inclusion Body Myositis (IBM)

Primary Purpose

Inclusion Body Myositis

Status

Completed

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

Arimoclomol

Placebo

About this trial

This is an interventional treatment trial for Inclusion Body Myositis focused on measuring IBM

Eligibility Criteria

45 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Arimoclomol

Placebo

Arm Description

248 mg arimoclomol base (equivalent to 400 mg arimoclomol citrate) 3 times daily

248 mg matching placebo 3 times daily

Outcomes

Primary Outcome Measures

Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score

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

Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score

Grip Strength

Unilateral hand grip strength in both hands measured using the Jamar Dynamometer. Results are for the stronger limb, as identified at baseline.

Modified Timed up and go (mTUG)

The patient's combined ability to rise from a chair and walk 3 meters, turn around and walk back to the chair and sit down. The test was performed twice and the fastest time was used. The results were expressed as velocity in meters/second.

Manual Muscle Testing (MMT), Total Score

The Manual Muscle Testing (MMT) scores the strength of 24 muscles (axial, proximal, and distal muscles, tested bilaterally) on a scale from 0 to 10 points. The total score is calculated as an average across the 24 muscles and ranges from 0 to 10. The total score will increase if a patient is getting stronger and decrease if a patient is getting weaker.

6 Minute Walk Test (6MWT); Distance After 6 Minutes (6MWD)

The distance patients could walk in 6 minutes. The distance walked in meters was recorded after 6 minutes.

Short Form-36 (SF-36) Physical Component Score

Measured using the Short Form health survey with 36 items (SF-36). The questionnaire measures 8 health concepts which yields 2 summary measures: physical and mental health. The physical component score includes 4 scales of physical functioning (10 items), role limitations due to physical health (4 items), bodily pain (2 items), and general health (5 items). To score the SF-36, scales are standardized with a scoring algorithm to obtain a score ranging from 0 to 100, and the summary measure is calculated as the average score. Higher scores indicate better health status.

Maximum Voluntary Isometric Contraction (MVICT) of Quadriceps

Unilateral strength of the knee extensor muscles on both limbs using the MicroFET hand-held dynamometer. Results are for the stronger limb, as identified at baseline.

Health Assessment Questionnaire - Disability Index (HAQ-DI)

The disability index of the HAQ measures self-reported functional status (disability) including the patient's use of aids or devices and/or help from other persons. The scale is composed of 20 items in 8 domains (dressing and grooming, arising, eating, walking, hygiene, reach, grip, and common daily activities). Each domain has at least 2 subcategory items, scored on a scale from 0 to 3, and for each of the 8 domains the domain score was the highest score of the involved subcategory scores. The total score reported is an average over 8 domains and scores from at least 6 domains had to be available for the total score to be calculated. The average total score ranges from 0 to 3, and a higher score corresponds to a worsening in functional status.

2 Minute Walk Test (2MWT)

The distance patients could walk in 2 minutes (during the 6 Minute Walk Test) recorded in meters.

Short Form-36 (SF-36) Mental Component Score

Measured using the Short Form health survey with 36 items (SF-36). The questionnaire measures 8 health concepts which yields 2 summary measures: physical and mental health. The mental component score is composed of energy/fatigue (4 items), social functioning (2 items), role limitations due to emotional problems (3 items), and emotional well-being (5 items). To score the SF-36, scales are standardized with a scoring algorithm to obtain a score ranging from 0 to 100, and the summary measure is calculated as the average score. Higher scores indicate better health status.

Patient Global Impression of Severity (PGIS)

Patient-reported assessment of the impact of IBM on the ability to complete activities of daily living (e.g. dressing, walking, bathing) at the time of the assessment. The response options for the impact of IBM were "none" (i.e. no impact), "very mild", "mild", "moderate", "severe", and "very severe".

Patient Global Impression of Change (PGIC)

Patient-reported assessment of the change from start of study treatment in the impact of IBM on the ability to complete activities of daily living (e.g. dressing, walking, bathing). The response options for change in impact were "very much worse", "much worse", "a little worse", "no change", "a little improved", "much improved", and "very much improved".

Clinician Global Impression of Severity (CGIS)

Clinician-reported assessment of the severity of the patient's IBM symptoms at the time of the assessment. The response options were "none", "very mild", "mild", "moderate", "severe", and "very severe"

Clinician Global Impression of Change (CGIC)

Clinician-reported assessment of the change from start of study treatment in the patient's IBM symptoms. The response options for change in IBM were "very much worse", "much worse", "a little worse", "no change", "a little improved", "much improved", and "very much improved".

Falls and Near Falls

Falls and near falls registered by the participants in a diary

Full Information

NCT ID

NCT02753530

First Posted

April 25, 2016

Last Updated

April 18, 2023

Sponsor

ZevraDenmark

Collaborators

University of Kansas Medical Center, University College, London

1. Study Identification

Unique Protocol Identification Number

NCT02753530

Brief Title

Study of Arimoclomol in Inclusion Body Myositis (IBM)

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

April 2023

Overall Recruitment Status

Completed

Study Start Date

August 16, 2017 (Actual)

Primary Completion Date

January 11, 2021 (Actual)

Study Completion Date

January 11, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

ZevraDenmark

Collaborators

University of Kansas Medical Center, University College, London

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

Funding Source - FDA Office of Orphan Products Development (OOPD). The purpose of this study is to evaluate the safety and efficacy of the study drug, arimoclomol in IBM patients.

Detailed Description

A Phase 2/3, randomized, double-blind, placebo-controlled, international, 20-month intervention study in patients with IBM.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Inclusion Body Myositis

Keywords

IBM

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantInvestigator

Allocation

Randomized

Enrollment

152 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Arimoclomol

Arm Type

Experimental

Arm Description

248 mg arimoclomol base (equivalent to 400 mg arimoclomol citrate) 3 times daily

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

248 mg matching placebo 3 times daily

Intervention Type

Drug

Intervention Name(s)

Arimoclomol

Intervention Description

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

Intervention Type

Other

Intervention Name(s)

Placebo

Intervention Description

2 matched placebo capsules taken 3 times daily during breakfast, early afternoon, and at bedtime

Primary Outcome Measure Information:

Title

Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score

Description

Time Frame

Change from Baseline to Month 20

Secondary Outcome Measure Information:

Title

Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score

Description

Time Frame

Change from Baseline to Month 12

Title

Grip Strength

Description

Unilateral hand grip strength in both hands measured using the Jamar Dynamometer. Results are for the stronger limb, as identified at baseline.

Time Frame

Change from Baseline to Month 12 and 20

Title

Modified Timed up and go (mTUG)

Description

Time Frame