Trial · NCT04999020
Ravulizumab Versus Placebo in Adult Participants With Dermatomyositis
NCT04999020, Phase 2, Phase 3, Terminated, Alexion Pharmaceuticals, Inc.; results first posted 2024-12-06
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Ravulizumab Versus Placebo in Adult Participants With Dermatomyositis. Trials, record NCT04999020. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04999020
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Ravulizumab Versus Placebo in Adult Participants With Dermatomyositis
- NCT number
- NCT04999020
- Status
- Terminated
- Phases
- Phase 2, Phase 3
- Enrollment
- 38 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Dermatomyositis
- Interventions
- Ravulizumab, Placebo
- First posted
- 2021-08-10
- Last updated
- 2025-07-09
- Other registries' IDs
- eudract:2021-001200-15
- Results first posted
- 2024-12-06
- Limitations and caveats
- Part A did not meet its primary endpoint and the study was terminated.
Source ClinicalTrials.gov
Registered outcomes16
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With International Myositis Assessment and Clinical Studies Total Improvement Score (IMACS-TIS) (TIS40) Response at Week 26 of the Randomized Controlled Period | Week 26 | Data are presented for the number of participants with a TIS40 response, defined as an IMACS-TIS score ≥ 40 at Week 26. IMACS-TIS is a clinical instrument that encompasses 6 core set measure (CSMs) (physician, patient, extra-muscular global activity, muscle strength, Health Assessment Questionnaire \[HAQ\], and muscle enzyme levels). A Total Improvement Score (TIS: 0-100), was determined by summing scores in each CSM, and was based on the improvement and relative weight of each CSM. A higher score indicated greater improvement. TIS40 was considered a moderate improvement score. |
| Secondary | TIS at Week 26 | Week 26 | TIS scores ranged from 0-100 with higher scores indicating a greater improvement. Scores were determined by summing scores in each of the 6 CSMs of the IMAC (physician, patient, extra-muscular global activity, muscle strength, HAQ, and muscle enzyme levels). Clinically meaningful thresholds for improvement were defined as ≥ 20 point improvement response on IMACS-TIS (TIS20; mild), ≥ 40 point improvement response on IMACS TIS (TIS40; moderate) and ≥ 60 point improvement response on IMACS-TIS (TIS60; severe). Scores were based on the improvement and relative weight of each CSM. Data are presented for TIS (least squares mean) at Week 26. |
| Secondary | Change From Baseline In Cutaneous Dermatomyositis Disease Area And Severity Index (CDASI) Activity Score at Week 26 | Baseline, Week 26 | The CDASI is an instrument that separately measures activity and damage in the skin of dermatomyositis (DM) participants. It contains 3 activity measures (erythema, scale, and erosion/ulceration) and 2 damage measures (poikiloderma and calcinosis). CDASI score is calculated by rating the severity of skin disease in 15 anatomical locations on the body based on the activity and damage components. CDASI was completed by the Clinician or Clinician-Investigator while examining the participant. Total CDASI scores ranged from 0-100, with higher scores indicating a greater disease severity. Change from baseline in CDASI Total Activity Score at Week 26 was analyzed using a mixed model repeated measures (MMRM). The MMRM model included the observed Total Activity Score values at post baseline visits (Week 26) as the dependent variable. |
| Secondary | Number of Participants With Response Related to Muscle Enzymes: Normalization of Most Abnormal Baseline Enzyme at Week 26 | Baseline, Week 26 | Laboratory tests were conducted to measure serum activities of muscle associated enzymes including creatine kinase (CK), alanine aminotransferase (ALT), aspartate aminotransferase (AST), lactate dehydrogenase (LDH), and aldolase. Data are presented for the number of participants who had an abnormal muscle enzyme at baseline that had been normalized at Week 26. |
| Secondary | Change From Baseline In IMACS CSMs: Extra-Muscular Disease Activity Based on Myositis Disease Activity Assessment Tool (MDAAT) at Week 26 | Baseline, Week 26 | The MDAAT assesses disease activity of extra-muscular organ systems and muscles in participants with DM. The validated MDAAT tool measures the degree of disease activity of extra-muscular organ systems and muscle on a 0-10 centimeter (cm) visual analog scale (VAS). Extra-muscular activity ranged between 0 and 10, where, 0 cm = absent and 10 cm = maximum disease activity. |
| Secondary | Change From Baseline In IMACS CSMs: Physician Global Activity Assessment at Week 26 | Baseline, Week 26 | The physician global activity assessment provides an overall rating of disease activity related to myositis. Disease activity is judged by the physician based on all information available at the time of evaluation, including the participant's appearance, medical history, physical examination, laboratory testing, and prescribed medical therapy. The global disease activity score is recorded on a 10-cm VAS, where 0 cm= no evidence of disease activity and 10 cm= extremely severe disease activity. |
| Secondary | Change From Baseline In IMACS CSMs: Patient Global Activity Assessment at Week 26 | Baseline, Week 26 | The patient global activity assessment provides an overall rating of disease activity related to myositis from the participant's perspective. Participants were asked to consider all of the active inflammation in their own muscles, skin, joints, intestines, heart, lungs, or other parts of the body that can improve with treatment. The patient global disease activity score was recorded on a 10-cm VAS that contained a smiley face at the 0-cm anchor and a sad face at the 10 cm anchor to help participants understand the scale. Scores ranged from 0 (no evidence of disease activity) to 10 (extremely active or severe disease activity). |
| Secondary | Change From Baseline In IMACS CSMs: Manual Muscle Testing Subset 8 Muscles (MMT-8) at Week 26 | Baseline, Week 26 | The purpose of the MMT-8 was to measure muscle strength as part of the physical examination. It included a subset of 8 muscle groups: neck flexors, deltoids, biceps, wrist, extensors, gluteus maximus and medius, quadriceps, and ankle dorsiflexors. Total MMT8 scores ranged from 0 (lowest strength) to 150 (highest strength). |
| Secondary | Change From Baseline In IMACS CSMs: Health Assessment Questionnaire (HAQ) at Week 26 | Baseline, Week 26 | The HAQ is a brief self-report questionnaire that assesses physical function pertaining to activities of daily living in a variety of domains. The HAQ includes 20 questions relating to 8 domains of function: dressing and grooming, arising, eating, walking, hygiene, reach, grip and usual activities. For each of the categories, participants reported the amount of difficulty they had in performing 2 or 3 specific subcategory items. The standard disability score is calculated from the 8 categories by dividing the sum of the individual categories by the number of categories answered, yielding a score from 0 (without any difficulty) to 3 (unable to do), with higher values indicating higher disability. |
| Secondary | Number of Participants With CDASI Response (>=7-point Improvement) at Week 26 | Week 26 | The CDASI is an instrument that separately measures activity and damage in the skin of dermatomyositis (DM) participants. It contains 3 activity measures (erythema, scale, and erosion/ulceration) and 2 damage measures (poikiloderma and calcinosis). CDASI score is calculated by rating the severity of skin disease in 15 anatomical locations on the body based on the activity and damage components. CDASI was completed by the Clinician or Clinician-Investigator while examining the participant. Total CDASI scores ranged from 0-100, with higher scores indicating a greater disease severity. Data are presented for the number of participants with a CDASI response. Response was defined as a \>=7 point improvement in participants who did not have an intercurrent event at or prior to the relevant timepoint. |
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 | Number of Participants With International Myositis Assessment and Clinical Studies Total Improvement Score (IMACS-TIS) (TIS40) Response at Week 26 of the Randomized Controlled Period | Week 26 | Randomized Set, which included all randomized participants grouped by randomized treatment group. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | TIS at Week 26 | Week 26 | Randomized Set, which included all randomized participants grouped by randomized treatment group. Overall number of participants analyzed = participants with evaluable data for the outcome measure. | scores on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline In Cutaneous Dermatomyositis Disease Area And Severity Index (CDASI) Activity Score at Week 26 | Baseline, Week 26 | Randomized Set, which included all randomized participants grouped by randomized treatment group. Overall number of participants analyzed = participants with evaluable data for the outcome measure. | scores on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Number of Participants With Response Related to Muscle Enzymes: Normalization of Most Abnormal Baseline Enzyme at Week 26 | Baseline, Week 26 | Randomized Set, which included all randomized participants grouped by randomized treatment group. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline In IMACS CSMs: Extra-Muscular Disease Activity Based on Myositis Disease Activity Assessment Tool (MDAAT) at Week 26 | Baseline, Week 26 | Randomized Set, which included all randomized participants grouped by randomized treatment group. Overall number of participants analyzed = participants with evaluable data for the outcome measure. | scores on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline In IMACS CSMs: Physician Global Activity Assessment at Week 26 | Baseline, Week 26 | Randomized Set, which included all randomized participants grouped by randomized treatment group. Overall number of participants analyzed = participants with evaluable data for the outcome measure. | scores on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline In IMACS CSMs: Patient Global Activity Assessment at Week 26 | Baseline, Week 26 | Randomized Set, which included all randomized participants grouped by randomized treatment group. Overall number of participants analyzed = participants with evaluable data for the outcome measure. | scores on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline In IMACS CSMs: Manual Muscle Testing Subset 8 Muscles (MMT-8) at Week 26 | Baseline, Week 26 | Randomized Set, which included all randomized participants grouped by randomized treatment group. Overall number of participants analyzed = participants with evaluable data for the outcome measure. | scores on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline In IMACS CSMs: Health Assessment Questionnaire (HAQ) at Week 26 | Baseline, Week 26 | Randomized Set, which included all randomized participants grouped by randomized treatment group. Overall number of participants analyzed = participants with evaluable data for the outcome measure. | scores on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Number of Participants With CDASI Response (>=7-point Improvement) at Week 26 | Week 26 | Randomized Set, which included all randomized participants grouped by randomized treatment group. | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 outcomesResults by group36
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With International Myositis Assessment and Clinical Studies Total Improvement Score (IMACS-TIS) (TIS40) Response at Week 26 of the Randomized Controlled Period | RCP: Ravulizumab | 9 | Participants | ||
| Number of Participants With International Myositis Assessment and Clinical Studies Total Improvement Score (IMACS-TIS) (TIS40) Response at Week 26 of the Randomized Controlled Period | RCP: Placebo | 6 | Participants | ||
| TIS at Week 26 | RCP: Ravulizumab | 31.16 | Standard Error 4.185 | scores on a scale | |
| TIS at Week 26 | RCP: Placebo | 43.28 | Standard Error 6.650 | scores on a scale | |
| Change From Baseline In Cutaneous Dermatomyositis Disease Area And Severity Index (CDASI) Activity Score at Week 26 | RCP: Ravulizumab | -3.80 | Standard Error 1.249 | scores on a scale | |
| Change From Baseline In Cutaneous Dermatomyositis Disease Area And Severity Index (CDASI) Activity Score at Week 26 | RCP: Placebo | -7.47 | Standard Error 2.021 | scores on a scale | |
| Number of Participants With Response Related to Muscle Enzymes: Normalization of Most Abnormal Baseline Enzyme at Week 26 | RCP: Ravulizumab | 4 | Participants | ||
| Number of Participants With Response Related to Muscle Enzymes: Normalization of Most Abnormal Baseline Enzyme at Week 26 | RCP: Placebo | 1 | Participants | ||
| Change From Baseline In IMACS CSMs: Extra-Muscular Disease Activity Based on Myositis Disease Activity Assessment Tool (MDAAT) at Week 26 | RCP: Ravulizumab | -0.92 | Standard Error 0.383 | scores on a scale | |
| Change From Baseline In IMACS CSMs: Extra-Muscular Disease Activity Based on Myositis Disease Activity Assessment Tool (MDAAT) at Week 26 | RCP: Placebo | -2.13 | Standard Error 0.614 | scores on a scale |
1 to 10 of 36Source ClinicalTrials.gov
See all 36 results by groupParticipant flow32
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Randomized Controlled Period (RCP) | STARTED | RCP: Ravulizumab | 26 | |
| Randomized Controlled Period (RCP) | STARTED | RCP: Placebo | 12 | |
| Randomized Controlled Period (RCP) | STARTED | OLE: Ravulizumab to Ravulizumab | 0 | |
| Randomized Controlled Period (RCP) | STARTED | OLE: Placebo to Ravulizumab | 0 | |
| Randomized Controlled Period (RCP) | Received at Least 1 Dose of Treatment | RCP: Ravulizumab | 26 | |
| Randomized Controlled Period (RCP) | Received at Least 1 Dose of Treatment | RCP: Placebo | 12 | |
| Randomized Controlled Period (RCP) | Received at Least 1 Dose of Treatment | OLE: Ravulizumab to Ravulizumab | 0 | |
| Randomized Controlled Period (RCP) | Received at Least 1 Dose of Treatment | OLE: Placebo to Ravulizumab | 0 | |
| Randomized Controlled Period (RCP) | COMPLETED | RCP: Ravulizumab | 22 | |
| Randomized Controlled Period (RCP) | COMPLETED | RCP: Placebo | 9 |
1 to 10 of 32Source ClinicalTrials.gov
See all 32 participant flowAdverse events256
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 | Anaemia | Blood and lymphatic system disorders | RCP: Ravulizumab | 0 | 25 | |
| other | Anaemia | Blood and lymphatic system disorders | RCP: Placebo | 2 | 13 | |
| other | Anaemia | Blood and lymphatic system disorders | OLE: Ravulizumab to Ravulizumab | 0 | 22 | |
| other | Anaemia | Blood and lymphatic system disorders | OLE: Placebo to Ravulizumab | 0 | 9 | |
| other | Vertigo | Ear and labyrinth disorders | RCP: Ravulizumab | 0 | 25 | |
| other | Vertigo | Ear and labyrinth disorders | RCP: Placebo | 1 | 13 | |
| other | Vertigo | Ear and labyrinth disorders | OLE: Ravulizumab to Ravulizumab | 0 | 22 | |
| other | Vertigo | Ear and labyrinth disorders | OLE: Placebo to Ravulizumab | 0 | 9 | |
| other | Cushingoid | Endocrine disorders | RCP: Ravulizumab | 0 | 25 | |
| other | Cushingoid | Endocrine disorders | RCP: Placebo | 1 | 13 |
1 to 10 of 256Source ClinicalTrials.gov
See all 256 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | RCP: Ravulizumab | 0 | 25 |
| Total, all-cause mortality | RCP: Placebo | 0 | 13 |
| Total, all-cause mortality | OLE: Ravulizumab to Ravulizumab | 0 | 22 |
| Total, all-cause mortality | OLE: Placebo to Ravulizumab | 0 | 9 |
| Total, other adverse events | RCP: Ravulizumab | 9 | 25 |
| Total, other adverse events | RCP: Placebo | 10 | 13 |
| Total, other adverse events | OLE: Ravulizumab to Ravulizumab | 11 | 22 |
| Total, other adverse events | OLE: Placebo to Ravulizumab | 7 | 9 |
| Total, serious adverse events | RCP: Ravulizumab | 2 | 25 |
| Total, serious adverse events | RCP: Placebo | 0 | 13 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers0
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 comparisons8
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| RCP: Ravulizumab vs RCP: Placebo: Number of Participants With CDASI Response (>=7-point Improvement) at Week 26 | Trial comparisons |
| RCP: Ravulizumab vs RCP: Placebo: Number of Participants With Clinical Worsening (CW) During the RCP At 2 Consecutive Visits | Trial comparisons |
| RCP: Ravulizumab vs RCP: Placebo: Number of Participants With ≥ 20-Point Improvement Response on IMACS-TIS (TIS20) Response at Week 26 | Trial comparisons |
| RCP: Ravulizumab vs RCP: Placebo: Number of Participants With Response Related to Muscle Enzymes: Normalization of Most Abnormal Baseline Enzyme at Week 26 | Trial comparisons |
| RCP: Ravulizumab vs RCP: Placebo: Number of Participants With ≥ 60-Point Improvement Response on IMACS-TIS (TIS60) Response at Week 26 | Trial comparisons |
RCP: Ravulizumab vs RCP: Placebo: Number of Participants With International Myositis Assessment and Clinical Studies Total Improvement Score (IMACS-TIS) (TIS40) Response at Week 26 of the Randomized Controlled Period | Trial comparisons |
| RCP: Ravulizumab vs RCP: Placebo: Number of Participants Who Received Acute Rescue Therapy With Standard DM Treatment | Trial comparisons |
| RCP: Ravulizumab vs RCP: Placebo: Number of Participants With Cutaneous Dermatomyositis Activity Physician's Global Assessment (CDA-IGA) Response at Week 26 | 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/NCT04999020
GET https://conductscience.org/api/trials/v1/NCT04999020/results?page=2