Trial · NCT04650854
A Study to Evaluate Rozanolixizumab in Study Participants With Generalized Myasthenia Gravis
NCT04650854, Phase 3, Completed, UCB Biopharma SRL; results first posted 2025-04-18
3 sourcestrials-2026-11-12-0.4.0
Download JSON
Cite this record
ConductScience Library. A Study to Evaluate Rozanolixizumab in Study Participants With Generalized Myasthenia Gravis. Trials, record NCT04650854. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04650854
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Evaluate Rozanolixizumab in Study Participants With Generalized Myasthenia Gravis
- NCT number
- NCT04650854
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 165 participants, actual
- Lead sponsor
- UCB Biopharma SRL
- Conditions
- Generalized Myasthenia Gravis
- Interventions
- Rozanolixizumab
- First posted
- 2020-12-03
- Last updated
- 2026-08-20
- Other registries' IDs
- eudract:2020-003230-20
- Results first posted
- 2025-04-18
Source ClinicalTrials.gov
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) | From Baseline (Day 1) to End of Study (up to 34 months) | An adverse event (AE) was any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product, which did not necessarily had a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. TEAE was defined as any event that was not present prior to first dose of investigational medicinal product (IMP), or any unresolved event already present that worsened in intensity following treatment, up to 8 weeks after end of Treatment Period or after last dose of IMP in participants who discontinued study or IMP. |
| Primary | Percentage of Participants With TEAEs Leading to Withdrawal of Investigational Medicinal Product (IMP) | From Baseline (Day 1) to End of Study (up to 34 months) | An adverse event (AE) was any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. AEs leading to permanent withdrawal of study medication. |
| Secondary | Change From Baseline (Day 1) to Day 43 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score Within One Treatment Cycle (Cycle 1, 2, and 3) | From Baseline (Day 1) to Day 43 of each cycle (Cycles 1, 2, and 3) | The MG-ADL is an 8-item PRO instrument developed on basis of QMG. The MG-ADL targeted symptoms and disability across ocular, bulbar, respiratory, and axial symptoms. The total MG-ADL score was obtained by summing responses to each individual item (8 items; Grades: 0, 1, 2, 3), where 0= no symptoms or impaired performance and 3= most severe symptoms or impaired performance. The total score ranges from 0 to 24, with higher score indicating more disability. A positive change in score indicates worsening and negative change indicates improvement. For analyses done by study cycle, Baseline values were last available values prior to or on the same date of first administration of IMP at each cycle (Baseline \[Day 1\]) value for that cycle. |
| Secondary | Change From Baseline (Day 1) to Day 43 in Quantitative Myasthenia Gravis (QMG) Score Within One Treatment Cycle (Cycle 1, 2, and 3) | From Baseline (Day 1) to Day 43 of each cycle (Cycles 1, 2, and 3) | The QMG is a validated assessment and the scale tested 13 items, including ocular and facial involvement, swallowing, speech, limb strength, and forced vital capacity. The total QMG score was obtained by summing the responses to each individual item (13 items; Responses: None=0, Mild=1, Moderate=2, Severe=3) and the score ranges from 0 to 39, with lower scores indicating lower disease activity. A positive change in the score indicates worsening and a negative change indicates improvement. For the analyses done by study cycle, Baseline values were the last available values prior to or on the same date (and same time if time was collected for the individual assessment) of first administration of IMP at each cycle (ie, Baseline \[Day 1\]) value for that cycle. |
| Secondary | Change From Baseline (Day 1) to Day 43 in Myasthenia Gravis-Composite (MG-C) Score Within One Treatment Cycle (Cycle 1, 2, and 3) | From Baseline (Day 1) to Day 43 of each cycle (Cycles 1, 2, and 3) | MG-C scale consists of 10 items which included ptosis (score range=0 to 3), double vision on lateral gaze left/right/both (score range=0 to 4), eye closure (score range=0 to 2), talking (score range=0 to 6), chewing (score range=0 to 6), swallowing (score range=0 to 6), breathing (score range=0 to 9), neck flexion or extension (score range=0 to 4), shoulder abduction (score range=0 to 5) and hip flexion (score range= 0 to 5), lower scores= lower disease activity. Total MG-C score obtained by summing responses to each individual item and score ranges from 0 - 50, (lower scores indicating lower disease activity). Positive change = worsening, and negative change = improvement. For analyses done by study cycle, Baseline values were last available values prior to or on the same date of first administration of IMP at each cycle (Baseline \[Day 1\]) value for that cycle. |
| Secondary | Change From Baseline (Day 1) to Day 43 in Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Muscle Weakness Fatigability' Score Within One Treatment Cycle (Cycle 1, 2, and 3) | From Baseline (Day 1) to Day 43 of each cycle (Cycles 1, 2, and 3) | The MG symptoms PRO instrument consisted of 42 items across 5 scales: ocular symptoms (1-5); bulbar muscle weakness (6-15); respiratory muscle weakness (16-18); physical fatigue (19-33) and muscle weakness fatigability (34-42). Study participants had to choose response option that best described severity of ocular, bulbar, and respiratory symptoms over past 7 days using a 4-point Likert scale ("none" to "severe") and frequency of experiencing physical fatigue and muscle weakness fatigability over past 7 days using a 5-point Likert scale (1="none of the time" - 5= "all of the time"), for each item. Sum of each item score is linearly transformed to have all domain scores ranging from 0 to 100, higher scores indicates more severe symptoms. For analyses done by study cycle, Baseline values were last available values prior to or on the same date of first administration of IMP at each cycle (Baseline \[Day 1\]) value for that cycle. |
| Secondary | Change From Baseline (Day 1) to Day 43 in MG Symptoms PRO 'Physical Fatigue' Score Within One Treatment Cycle (Cycle 1, 2, and 3) | From Baseline (Day 1) to Day 43 of each cycle (Cycles 1, 2, and 3) | The MG symptoms PRO instrument consisted of 42 items across 5 scales: ocular muscle weakness (1-5); bulbar muscle weakness (6-15); respiratory muscle weakness (16-18); physical fatigue (19-33) and muscle weakness fatigability (34-42). Study participants had to choose response option based on frequency of experiencing physical fatigue (19-33) over past 7 days using a 5-point Likert scale (1="none of time" - 5="all of time") for each item. Sum of each item score is linearly transformed to have all domain scores ranging from 0 to 100, higher scores indicated severe symptoms. |
| Secondary | Change From Baseline (Day 1) to Day 43 in MG Symptoms PRO 'Bulbar Muscle Weakness' Score Within One Treatment Cycle (Cycle 1, 2, and 3) | From Baseline (Day 1) to Day 43 of each cycle (Cycles 1, 2, and 3) | The MG symptoms PRO instrument consisted of 42 items across 5 scales: ocular muscle weakness (1-5); bulbar muscle weakness (6-15); respiratory muscle weakness (16-18); physical fatigue (19-33) and muscle weakness fatigability (34-42). Bulbar symptoms are now recognised as bulbar muscle weakness. Study participants were asked to choose response option that best described severity of bulbar muscle weakness (6-15) symptoms over past 7 days using a 4-point Likert scale (1="none" to 4="severe") for each item. Sum of each item score is linearly transformed to have all domain scores ranging from 0 to 100, higher scores indicated severe symptoms. |
| Secondary | MG-ADL Responder Rate (>=2.0-point Improvement From Baseline [Day 1]) Within One Treatment Cycle (Cycle 1, 2, and 3 [Day 43]) | Day 43 of Cycle 1, 2, and 3 | The MG-ADL is an 8-item PRO instrument developed on the basis of the Quantitative Myasthenia Gravis (QMG). The MG-ADL targeted symptoms and disability across ocular, bulbar, respiratory, and axial symptoms. The total MG-ADL score was obtained by summing the responses to each individual item (8 items; Grades: 0, 1, 2, 3), where 0 represents no symptoms or impaired performance and 3 represents the most severe symptoms or impaired performance. The total score ranges from 0 to 24, with a higher score indicating more disability. A MG-ADL responder was defined as achieving at least 2.0-point improvement (decrease) in the MG-ADL score from Baseline. |
| Secondary | Time to MG-ADL Response (>=2.0-point Improvement From Baseline [Day 1]) Within One Treatment Cycle (Cycle 1, 2, and 3) | From Baseline (Day 1) to Day 43 of each cycle (Cycles 1, 2, and 3) | Time to achieve MG-ADL response, defined as at least 2.0-point improvement from Baseline. Time to first MG-ADL response (in days) by study cycle was defined as date of first MG-ADL Response within study cycle - date of MG-ADL Baseline within study cycle + 1. |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 registered outcomesOutcomes11
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) | From Baseline (Day 1) to End of Study (up to 34 months) | Safety Set (SS): All study participants in the FAS who received at least one dose of IMP. Participants who switched doses were counted in both Rozanolixizumab (RLZ) doses. | percentage of participants | NUMBER |
| PRIMARY | Percentage of Participants With TEAEs Leading to Withdrawal of Investigational Medicinal Product (IMP) | From Baseline (Day 1) to End of Study (up to 34 months) | Safety Set (SS): All study participants in the FAS who received at least one dose of IMP. Participants who switched doses were counted in both RLZ doses. | percentage of participants | NUMBER |
| SECONDARY | Change From Baseline (Day 1) to Day 43 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score Within One Treatment Cycle (Cycle 1, 2, and 3) | From Baseline (Day 1) to Day 43 of each cycle (Cycles 1, 2, and 3) | Safety Set (SS): All study participants in the FAS who received at least one dose of IMP. Number of Participants Analyzed included those participants who were evaluable for the assessment. Number Analyzed included those participants who were evaluable at specified timepoint. | score on a scale | MEAN |
| SECONDARY | Change From Baseline (Day 1) to Day 43 in Quantitative Myasthenia Gravis (QMG) Score Within One Treatment Cycle (Cycle 1, 2, and 3) | From Baseline (Day 1) to Day 43 of each cycle (Cycles 1, 2, and 3) | Safety Set (SS): All study participants in the FAS who received at least one dose of IMP. Number of Participants Analyzed included those participants who were evaluable for the assessment. Number Analyzed included those participants who were evaluable at specified timepoint. | score on a scale | MEAN |
| SECONDARY | Change From Baseline (Day 1) to Day 43 in Myasthenia Gravis-Composite (MG-C) Score Within One Treatment Cycle (Cycle 1, 2, and 3) | From Baseline (Day 1) to Day 43 of each cycle (Cycles 1, 2, and 3) | Safety Set (SS): All study participants in FAS who received at least 1 dose of IMP. Number of Participants Analyzed included participants who were evaluable for the assessment. Number Analyzed included participants who were evaluable at specified timepoint. | score on a scale | MEAN |
| SECONDARY | Change From Baseline (Day 1) to Day 43 in Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Muscle Weakness Fatigability' Score Within One Treatment Cycle (Cycle 1, 2, and 3) | From Baseline (Day 1) to Day 43 of each cycle (Cycles 1, 2, and 3) | Safety Set (SS): All study participants in FAS who received at least 1 dose of IMP. Number of Participants Analyzed: participants who were evaluable for the assessment. Number Analyzed: participants who were evaluable at specified timepoint. | score on a scale | MEAN |
| SECONDARY | Change From Baseline (Day 1) to Day 43 in MG Symptoms PRO 'Physical Fatigue' Score Within One Treatment Cycle (Cycle 1, 2, and 3) | From Baseline (Day 1) to Day 43 of each cycle (Cycles 1, 2, and 3) | Safety Set (SS): All study participants in FAS who received at least 1 dose of IMP. Number of Participants Analyzed: participants who were evaluable for the assessment. Number Analyzed: participants who were evaluable at specified timepoint. | score on a scale | MEAN |
| SECONDARY | Change From Baseline (Day 1) to Day 43 in MG Symptoms PRO 'Bulbar Muscle Weakness' Score Within One Treatment Cycle (Cycle 1, 2, and 3) | From Baseline (Day 1) to Day 43 of each cycle (Cycles 1, 2, and 3) | Safety Set (SS): All study participants in the FAS who received at least one dose of IMP. Number of Participants Analyzed: participants who were evaluable for the assessment. Number Analyzed included those participants who were evaluable at specified timepoint. | score on a scale | MEAN |
| SECONDARY | MG-ADL Responder Rate (>=2.0-point Improvement From Baseline [Day 1]) Within One Treatment Cycle (Cycle 1, 2, and 3 [Day 43]) | Day 43 of Cycle 1, 2, and 3 | Safety Set (SS): All study participants in the FAS who received at least one dose of IMP. Number of Participants Analyzed included those participants who were evaluable for the assessment. Number Analyzed: participants who were evaluable at specified timepoint. | percentage of participants | NUMBER |
| SECONDARY | Time to MG-ADL Response (>=2.0-point Improvement From Baseline [Day 1]) Within One Treatment Cycle (Cycle 1, 2, and 3) | From Baseline (Day 1) to Day 43 of each cycle (Cycles 1, 2, and 3) | Safety Set (SS): All study participants in the FAS who received at least one dose of IMP. Number Analyzed: participants who were evaluable at specified timepoint. | days | MEDIAN |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 outcomesResults by group84
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) | Rozanolixizumab ~7 mg/kg | 78.4 | percentage of participants | ||
| Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) | Rozanolixizumab ~10 mg/kg | 94.1 | percentage of participants | ||
| Percentage of Participants With TEAEs Leading to Withdrawal of Investigational Medicinal Product (IMP) | Rozanolixizumab ~7 mg/kg | 9.8 | percentage of participants | ||
| Percentage of Participants With TEAEs Leading to Withdrawal of Investigational Medicinal Product (IMP) | Rozanolixizumab ~10 mg/kg | 16.7 | percentage of participants | ||
| Change From Baseline (Day 1) to Day 43 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score Within One Treatment Cycle (Cycle 1, 2, and 3) | Rozanolixizumab ~7 mg/kg | Cycle 1: Treatment (Day 43) | -3.7 | Standard Deviation 3.5 | score on a scale |
| Change From Baseline (Day 1) to Day 43 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score Within One Treatment Cycle (Cycle 1, 2, and 3) | Rozanolixizumab ~10 mg/kg | Cycle 1: Treatment (Day 43) | -3.1 | Standard Deviation 2.9 | score on a scale |
| Change From Baseline (Day 1) to Day 43 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score Within One Treatment Cycle (Cycle 1, 2, and 3) | Rozanolixizumab ~7 mg/kg | Cycle 2: Treatment (Day 43) | -2.9 | Standard Deviation 3.1 | score on a scale |
| Change From Baseline (Day 1) to Day 43 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score Within One Treatment Cycle (Cycle 1, 2, and 3) | Rozanolixizumab ~10 mg/kg | Cycle 2: Treatment (Day 43) | -3.8 | Standard Deviation 3.9 | score on a scale |
| Change From Baseline (Day 1) to Day 43 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score Within One Treatment Cycle (Cycle 1, 2, and 3) | Rozanolixizumab ~7 mg/kg | Cycle 3: Treatment (Day 43) | -3.3 | Standard Deviation 2.7 | score on a scale |
| Change From Baseline (Day 1) to Day 43 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score Within One Treatment Cycle (Cycle 1, 2, and 3) | Rozanolixizumab ~10 mg/kg | Cycle 3: Treatment (Day 43) | -3.2 | Standard Deviation 3.3 | score on a scale |
1 to 10 of 84Source ClinicalTrials.gov
See all 84 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Enrolled, But Not Treated | 8 | |
| Overall Study | STARTED | Rozanolixizumab ~7 mg/kg | 80 | |
| Overall Study | STARTED | Rozanolixizumab ~10 mg/kg | 77 | |
| Overall Study | COMPLETED | Enrolled, But Not Treated | 3 | |
| Overall Study | COMPLETED | Rozanolixizumab ~7 mg/kg | 15 | |
| Overall Study | COMPLETED | Rozanolixizumab ~10 mg/kg | 16 | |
| Overall Study | NOT COMPLETED | Enrolled, But Not Treated | 5 | |
| Overall Study | NOT COMPLETED | Rozanolixizumab ~7 mg/kg | 65 | |
| Overall Study | NOT COMPLETED | Rozanolixizumab ~10 mg/kg | 61 |
Source ClinicalTrials.gov
Adverse events246
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 | Diarrhoea | Gastrointestinal disorders | Enrolled, But Not Treated | 0 | 8 | 0 |
| other | Diarrhoea | Gastrointestinal disorders | Rozanolixizumab ~7 mg/kg | 20 | 102 | 49 |
| other | Diarrhoea | Gastrointestinal disorders | Rozanolixizumab ~10 mg/kg | 28 | 102 | 55 |
| other | Abdominal pain | Gastrointestinal disorders | Enrolled, But Not Treated | 0 | 8 | 0 |
| other | Abdominal pain | Gastrointestinal disorders | Rozanolixizumab ~7 mg/kg | 10 | 102 | 11 |
| other | Abdominal pain | Gastrointestinal disorders | Rozanolixizumab ~10 mg/kg | 7 | 102 | 11 |
| other | Abdominal pain upper | Gastrointestinal disorders | Enrolled, But Not Treated | 0 | 8 | 0 |
| other | Abdominal pain upper | Gastrointestinal disorders | Rozanolixizumab ~7 mg/kg | 2 | 102 | 2 |
| other | Abdominal pain upper | Gastrointestinal disorders | Rozanolixizumab ~10 mg/kg | 6 | 102 | 8 |
| other | Nausea | Gastrointestinal disorders | Enrolled, But Not Treated | 0 | 8 | 0 |
1 to 10 of 246Source ClinicalTrials.gov
See all 246 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Enrolled, But Not Treated | 1 | 8 |
| Total, all-cause mortality | Rozanolixizumab ~7 mg/kg | 1 | 102 |
| Total, all-cause mortality | Rozanolixizumab ~10 mg/kg | 3 | 102 |
| Total, other adverse events | Enrolled, But Not Treated | 5 | 8 |
| Total, other adverse events | Rozanolixizumab ~7 mg/kg | 67 | 102 |
| Total, other adverse events | Rozanolixizumab ~10 mg/kg | 78 | 102 |
| Total, serious adverse events | Enrolled, But Not Treated | 3 | 8 |
| Total, serious adverse events | Rozanolixizumab ~7 mg/kg | 16 | 102 |
| Total, serious adverse events | Rozanolixizumab ~10 mg/kg | 31 | 102 |
Source ClinicalTrials.gov
Papers3
The papers the trial lists on ClinicalTrials.gov.
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.
Source
- Release
- trials-2026-11-12-0.4.0
- Release manifest SHA-256
- 0f4a8d8c48aee2a7573ae0fa94ecbeab70c1adbe449bbc95c424bc7d3e97b9d4
- Registry capture
- 2026-11-12
- Results copy, AACT
- 2026-03-23
- NIH clinical studies file
- 2026-11-13
Source ClinicalTrials.gov
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNone 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/NCT04650854
GET https://conductscience.org/api/trials/v1/NCT04650854/results?page=2