Trial · NCT04049097
Arimoclomol in Sporadic Inclusion Body Myositis - Open Label Extension Trial
NCT04049097, Phase 3, Terminated, ZevraDenmark; results first posted 2023-09-15
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Arimoclomol in Sporadic Inclusion Body Myositis - Open Label Extension Trial. Trials, record NCT04049097. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04049097
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Arimoclomol in Sporadic Inclusion Body Myositis - Open Label Extension Trial
- NCT number
- NCT04049097
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 121 participants, actual
- Lead sponsor
- ZevraDenmark
- Conditions
- Inclusion Body Myositis
- Interventions
- Arimoclomol
- First posted
- 2019-08-07
- Last updated
- 2023-09-15
- Results first posted
- 2023-09-15
Source ClinicalTrials.gov
Registered outcomes7
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 in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks). | The Inclusion Body Myositis Functional Rating Scale (IBMFRS) includes 10 measures (swallowing, handwriting, cutting food and handling utensils, fine motor tasks, dressing, hygiene, turning in bed and adjusting covers, changing position from sitting to standing, walking, and climbing stairs), each graded on a Likert scale from 0 (being unable to perform) to 4 (normal). The sum of the 10 items gives a value between 0 and 40. A higher score represents less functional limitation. After the study was terminated early by the sponsor, the analyses of the efficacy endpoints were simplified from what was planned in the study protocol and data were only summarized descriptively. |
| Secondary | Change in Six Minutes Walking Distance Test; Distance at 6 Minutes (6MWD) | Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks). | Patients were instructed to walk down one side of a track and back along the opposite side as quickly and safely as possible for 6 minutes. Patients were allowed to take breaks as needed during the walking period, but timing continued during breaks. The distance walked in meters was recorded after 6 minutes. After the study was terminated early by the sponsor, the analyses of the efficacy endpoints were simplified from what was planned in the study protocol and data were only summarized descriptively. |
| Secondary | Change in Modified Timed Up and Go (mTUG) | Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks). | The Modified Timed Up and Go (mTUG) measures the patient's ability to get up from a chair (allowing patients to use their arms), walk 3 meters, turn around, walk back to the chair, and sit down. The use of nearby walls or assistance from a caregiver was not allowed. After the study was terminated early by the sponsor, the analyses of the efficacy endpoints were simplified from what was planned in the study protocol and data were only summarized descriptively. |
| Secondary | Change in Quadriceps Muscle Strength | Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks). | Maximal voluntary isometric contraction testing (MVICT) of the patient's quadriceps muscle (extensor strength of the knee) were performed using a hand myometer which is a hand-held device that allows the examiner to push against a muscle while the patient resists. The test was performed on each side. The results for the stronger knee are reported here. After the study was terminated early by the sponsor, the analyses of the efficacy endpoints were simplified from what was planned in the study protocol and data were only summarized descriptively. |
| Secondary | Change in Hand Grip Strength | Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks). | Hand grip strength was assessed using a dynamometer. The test was performed on each hand. The grip strength of the stronger hand is reported here. After the study was terminated early by the sponsor, the analyses of the efficacy endpoints were simplified from what was planned in the study protocol and data were only summarized descriptively. |
| Secondary | Change in the 36-Item Short Form Health Survey (SF-36) | Change from Baseline in IBM-OLE to Early Termination Visit | The 36-Item Short Form Health Survey (SF-36) is a 36-item, patient-reported survey of health status. After the study was terminated early by the sponsor, the analyses of the efficacy endpoints were simplified from what was planned in the study protocol. No derived scores were calculated for SF-36 and no summary tabulation was done. |
| Secondary | Number of Falls and Near Falls | Baseline to Early Termination Visit | Patients recorded the number of falls and near falls in a falls diary. After the study was terminated early by the sponsor, the analyses of the efficacy endpoints were simplified from what was planned in the study protocol. No tabulation of falls and near falls was performed. |
Source ClinicalTrials.gov
Outcomes7
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 in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks). | Patients in the modified intention-to-treat population with data for IBMFRS at the early termination visit | score on a scale | MEAN |
| SECONDARY | Change in Six Minutes Walking Distance Test; Distance at 6 Minutes (6MWD) | Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks). | Patients in the modified intention-to-treat population with data for the Six Minutes Walking distance test at the early termination visit | meters | MEAN |
| SECONDARY | Change in Modified Timed Up and Go (mTUG) | Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks). | Patients in the modified intention-to-treat population with data for the Modified Timed Up and Go (mTUG) at the early termination visit | meters/second | MEAN |
| SECONDARY | Change in Quadriceps Muscle Strength | Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks). | Patients in the modified intention-to-treat population with data for the Quadriceps Muscle Strength at the early termination visit | kg | MEAN |
| SECONDARY | Change in Hand Grip Strength | Change from Baseline in IBM-OLE to Early Termination Visit (variable, an average of approximately 28 weeks). | Patients in the modified intention-to-treat population with data for the Hand Grip Strength test at the early termination visit | kg | MEAN |
| SECONDARY | Change in the 36-Item Short Form Health Survey (SF-36) | Change from Baseline in IBM-OLE to Early Termination Visit | Since the double-blind lead-in trial (NCT02753530) did not meet any of its efficacy endpoints, the open-label extension trial was terminated prematurely, and the statistical analysis plan (SAP) was amended prior to database lock to align with the focus on safety recommended by FDA (Guidance for Industry: Submission of Abbreviated Reports and Synopses in Support of Marketing Applications). The amended SAP pre-specified that the SF-36 data would not be analyzed since it has no relevance to safety. | ||
| SECONDARY | Number of Falls and Near Falls | Baseline to Early Termination Visit | Since the double-blind lead-in trial (NCT02753530) did not meet any of its efficacy endpoints, the open-label extension trial was terminated prematurely, and the statistical analysis plan (SAP) was amended prior to database lock to align with the focus on safety recommended by FDA (Guidance for Industry: Submission of Abbreviated Reports and Synopses in Support of Marketing Applications). The amended SAP pre-specified that the falls data would not be analyzed since it has no relevance to safety. |
Source ClinicalTrials.gov
Results by group5
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 (Open-label) | -1.7 | Standard Deviation 2.60 | score on a scale | |
| Change in Six Minutes Walking Distance Test; Distance at 6 Minutes (6MWD) | Arimoclomol (Open-label) | -17.2 | Standard Deviation 61.06 | meters | |
| Change in Modified Timed Up and Go (mTUG) | Arimoclomol (Open-label) | -0.027 | Standard Deviation 0.0847 | meters/second | |
| Change in Quadriceps Muscle Strength | Arimoclomol (Open-label) | -0.48 | Standard Deviation 3.719 | kg | |
| Change in Hand Grip Strength | Arimoclomol (Open-label) | -0.23 | Standard Deviation 4.616 | kg |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Arimoclomol (Open-label) | 121 | |
| Overall Study | COMPLETED | Arimoclomol (Open-label) | 0 | |
| Overall Study | NOT COMPLETED | Arimoclomol (Open-label) | 121 |
Source ClinicalTrials.gov
Adverse events27
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 (Open-label) | 7 | 121 | 9 |
| other | Transaminases increased | Investigations | Arimoclomol (Open-label) | 10 | 121 | 10 |
| other | Aspartate aminotransferase increased | Investigations | Arimoclomol (Open-label) | 13 | 121 | 13 |
| other | Blood lactate dehydrogenase increased | Investigations | Arimoclomol (Open-label) | 7 | 121 | 7 |
| other | Gamma-glutamyltransferase increased | Investigations | Arimoclomol (Open-label) | 10 | 121 | 11 |
| other | Alanine aminotransferase increased | Investigations | Arimoclomol (Open-label) | 14 | 121 | 14 |
| other | Diarrhoea | Gastrointestinal disorders | Arimoclomol (Open-label) | 13 | 121 | 15 |
| other | Constipation | Gastrointestinal disorders | Arimoclomol (Open-label) | 15 | 121 | 15 |
| other | Abdominal discomfort | Gastrointestinal disorders | Arimoclomol (Open-label) | 7 | 121 | 7 |
| other | Insomnia | Psychiatric disorders | Arimoclomol (Open-label) | 9 | 121 | 9 |
1 to 10 of 27Source ClinicalTrials.gov
See all 27 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Arimoclomol (Open-label) | 2 | 121 |
| Total, other adverse events | Arimoclomol (Open-label) | 97 | 121 |
| Total, serious adverse events | Arimoclomol (Open-label) | 13 | 121 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
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.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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/NCT04049097
GET https://conductscience.org/api/trials/v1/NCT04049097/events?page=2