Trial · NCT02753530
Study of Arimoclomol in Inclusion Body Myositis (IBM)
NCT02753530, Phase 2, Completed, ZevraDenmark; results first posted 2023-05-10
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of Arimoclomol in Inclusion Body Myositis (IBM). Trials, record NCT02753530. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02753530
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Arimoclomol in Inclusion Body Myositis (IBM)
- NCT number
- NCT02753530
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 152 participants, actual
- Lead sponsor
- ZevraDenmark
- Conditions
- Inclusion Body Myositis
- Interventions
- Arimoclomol, Placebo
- First posted
- 2016-04-28
- Last updated
- 2023-05-10
- Results first posted
- 2023-05-10
Source ClinicalTrials.gov
Registered outcomes16
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score | 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 | Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score | 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. |
| Secondary | Grip Strength | Change from Baseline to Month 12 and 20 | Unilateral hand grip strength in both hands measured using the Jamar Dynamometer. Results are for the stronger limb, as identified at baseline. |
| Secondary | Modified Timed up and go (mTUG) | Change from Baseline to Month 12 and Month 20 | 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. |
| Secondary | Manual Muscle Testing (MMT), Total Score | Change from Baseline to Month 12 and Month 20 | 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. |
| Secondary | 6 Minute Walk Test (6MWT); Distance After 6 Minutes (6MWD) | Change from Baseline to Month 12 and Month 20 | The distance patients could walk in 6 minutes. The distance walked in meters was recorded after 6 minutes. |
| Secondary | Short Form-36 (SF-36) Physical Component Score | Change from Baseline to Month 12 and Month 20 | 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. |
| Secondary | Maximum Voluntary Isometric Contraction (MVICT) of Quadriceps | Change from Baseline to Month 12 and Month 20 | 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. |
| Secondary | Health Assessment Questionnaire - Disability Index (HAQ-DI) | Change from Baseline to Month 12 and Month 20 | 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. |
| Secondary | 2 Minute Walk Test (2MWT) | Change from Baseline to Month 12 and Month 20 | The distance patients could walk in 2 minutes (during the 6 Minute Walk Test) recorded in meters. |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 registered outcomesOutcomes16
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score | Change from Baseline to Month 20 | Intention-to-treat; participants with data at Month 20 | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score | Change from Baseline to Month 12 | Intention-to-treat; participants with data at Month 12 | score on a scale | MEAN |
| SECONDARY | Grip Strength | Change from Baseline to Month 12 and 20 | Intention-to-treat; participants with data at Month 12 and Month 20, respectively | kg | MEAN |
| SECONDARY | Modified Timed up and go (mTUG) | Change from Baseline to Month 12 and Month 20 | Intention-to-treat; participants with data at Month 12 and Month 20, respectively | meters per second (m/sec) | MEAN |
| SECONDARY | Manual Muscle Testing (MMT), Total Score | Change from Baseline to Month 12 and Month 20 | Intention-to-treat; participants with data at Month 12 and Month 20, respectively | score on a scale | MEAN |
| SECONDARY | 6 Minute Walk Test (6MWT); Distance After 6 Minutes (6MWD) | Change from Baseline to Month 12 and Month 20 | Intention-to-treat; participants with data at Month 12 and Month 20, respectively | meters | MEAN |
| SECONDARY | Short Form-36 (SF-36) Physical Component Score | Change from Baseline to Month 12 and Month 20 | Intention-to-treat; participants with data at Month 12 and Month 20, respectively | score on a scale (physical component) | MEAN |
| SECONDARY | Maximum Voluntary Isometric Contraction (MVICT) of Quadriceps | Change from Baseline to Month 12 and Month 20 | Intention-to-treat; participants with data at Month 12 and Month 20, respectively | kg | MEAN |
| SECONDARY | Health Assessment Questionnaire - Disability Index (HAQ-DI) | Change from Baseline to Month 12 and Month 20 | Intention-to-treat; participants with data at Month 12 and Month 20, respectively | score on a scale | MEAN |
| SECONDARY | 2 Minute Walk Test (2MWT) | Change from Baseline to Month 12 and Month 20 | Intention-to-treat; participants with data at Month 12 and Month 20, respectively | meters | MEAN |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 outcomesResults by group160
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score | Arimoclomol (20 Months) | -3.26 | 95% Confidence Interval -4.15 to -2.36 | score on a scale | |
| Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score | Placebo (20 Months) | -2.26 | 95% Confidence Interval -3.11 to -1.41 | score on a scale | |
| Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score | Arimoclomol (20 Months) | -1.87 | Standard Deviation 3.494 | score on a scale | |
| Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) Total Score | Placebo (20 Months) | -1.03 | Standard Deviation 3.127 | score on a scale | |
| Grip Strength | Arimoclomol (20 Months) | Change from Baseline to Month 12 | -1.09 | Standard Deviation 5.459 | kg |
| Grip Strength | Placebo (20 Months) | Change from Baseline to Month 12 | -1.84 | Standard Deviation 3.259 | kg |
| Grip Strength | Arimoclomol (20 Months) | Change from Baseline to Month 20 | -3.93 | Standard Deviation 9.201 | kg |
| Grip Strength | Placebo (20 Months) | Change from Baseline to Month 20 | -2.86 | Standard Deviation 4.741 | kg |
| Modified Timed up and go (mTUG) | Arimoclomol (20 Months) | Change from Baseline to Month 12 | -0.075 | Standard Deviation 0.3048 | meters per second (m/sec) |
| Modified Timed up and go (mTUG) | Placebo (20 Months) | Change from Baseline to Month 12 | -0.058 | Standard Deviation 0.2281 | meters per second (m/sec) |
1 to 10 of 160Source ClinicalTrials.gov
See all 160 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Arimoclomol (20 Months) | 74 | |
| Overall Study | STARTED | Placebo (20 Months) | 78 | |
| Overall Study | COMPLETED | Arimoclomol (20 Months) | 62 | |
| Overall Study | COMPLETED | Placebo (20 Months) | 72 | |
| Overall Study | NOT COMPLETED | Arimoclomol (20 Months) | 12 | |
| Overall Study | NOT COMPLETED | Placebo (20 Months) | 6 |
Source ClinicalTrials.gov
Adverse events170
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Contusion | Injury, poisoning and procedural complications | Arimoclomol (20 Months) | 8 | 73 | |
| other | Contusion | Injury, poisoning and procedural complications | Placebo (20 Months) | 13 | 78 | |
| other | Fall | Injury, poisoning and procedural complications | Arimoclomol (20 Months) | 7 | 73 | |
| other | Fall | Injury, poisoning and procedural complications | Placebo (20 Months) | 6 | 78 | |
| other | Laceration | Injury, poisoning and procedural complications | Arimoclomol (20 Months) | 6 | 73 | |
| other | Laceration | Injury, poisoning and procedural complications | Placebo (20 Months) | 8 | 78 | |
| other | Ligament sprain | Injury, poisoning and procedural complications | Arimoclomol (20 Months) | 7 | 73 | |
| other | Ligament sprain | Injury, poisoning and procedural complications | Placebo (20 Months) | 5 | 78 | |
| other | Skin abrasion | Injury, poisoning and procedural complications | Arimoclomol (20 Months) | 5 | 73 | |
| other | Skin abrasion | Injury, poisoning and procedural complications | Placebo (20 Months) | 7 | 78 |
1 to 10 of 170Source ClinicalTrials.gov
See all 170 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Arimoclomol (20 Months) | 1 | 73 |
| Total, all-cause mortality | Placebo (20 Months) | 1 | 78 |
| Total, other adverse events | Arimoclomol (20 Months) | 72 | 73 |
| Total, other adverse events | Placebo (20 Months) | 70 | 78 |
| Total, serious adverse events | Arimoclomol (20 Months) | 11 | 73 |
| Total, serious adverse events | Placebo (20 Months) | 18 | 78 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants1
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years |
|---|---|---|---|---|---|
| R01FD004809 | Phase II Trial of Arimoclomol in IBM | DIMACHKIE, MAZEN; TAWIL, RABI | UNIVERSITY OF KANSAS MEDICAL CENTER | FOOD AND DRUG ADMINISTRATION | 2015 to 2020 |
Source NIH RePORTER
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Arimoclomol (20 Months) vs Placebo (20 Months): deaths from any cause | Trial comparisons |
| Arimoclomol (20 Months) vs Placebo (20 Months): serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02753530
GET https://conductscience.org/api/trials/v1/NCT02753530/results?page=2