Trial · NCT04124965
A Study to Investigate the Long-term Safety, Tolerability, and Efficacy of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis
NCT04124965, Phase 3, Completed, UCB Biopharma SRL; results first posted 2023-08-21
3 sourcestrials-2026-11-12-0.4.0
Download JSON
Cite this record
ConductScience Library. A Study to Investigate the Long-term Safety, Tolerability, and Efficacy of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis. Trials, record NCT04124965. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04124965
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Investigate the Long-term Safety, Tolerability, and Efficacy of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis
- NCT number
- NCT04124965
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 71 participants, actual
- Lead sponsor
- UCB Biopharma SRL
- Conditions
- Generalized Myasthenia Gravis
- Interventions
- Rozanolixizumab
- First posted
- 2019-10-14
- Last updated
- 2026-08-20
- Other registries' IDs
- eudract:2019-000969-21
- Results first posted
- 2023-08-21
Source ClinicalTrials.gov
Registered outcomes6
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 until End of Study (up to Week 60) | A TEAE is defined as an AE starting on or after the time of first administration of investigational medicinal product (IMP) or any unresolved event already present before the first administration of IMP that worsened in intensity following exposure to IMP, up to 8 weeks after the last dose of IMP in study participants who discontinued the study or IMP. |
| Primary | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Permanent Withdrawal of Study Medication | From Baseline until End of Study (up to Week 60) | A TEAE is defined as an AE starting on or after the time of first administration of IMP or any unresolved event already present before the first administration of IMP that worsened in intensity following exposure to IMP, up to 8 weeks after the last dose of IMP in study participants who discontinued the study or IMP. |
| Secondary | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Baseline, Weeks 5, 7, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 52 and 60 | The Myasthenia Gravis Activities of Daily Living (MG-ADL) is an 8-item patient-reported outcome (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 positive change indicates worsening and a negative change indicates improvement. |
| Secondary | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Baseline, Weeks 5, 7, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 52 and 60 | MG-C scale is a validated assessment and scale tests 10 items with individual items being weighted differently. The items included ptosis/upward gaze (range: 0 \[\>45 second\] - 3 \[Immediate\]), double vision on lateral gaze (range: 0 \[\>45 second\] - 4 \[Immediate\]), eye closure (range: 0 \[Normal\] - 2 \[severe weakness\]), talking (range: 0 \[Normal\] - 6 \[difficult to understand speech\]), chewing (range: 0 \[Normal\] - 6 \[gastric tube\]), swallowing (range: 0 \[Normal\] - 6 \[gastric tube\]), breathing (range: 0 \[Normal\] - 9 \[ventilator dependence\]), neck flexion (range: 0 \[Normal\] - 4 \[severe weakness\]), shoulder abduction (range: 0 \[Normal\] - 5 \[severe weakness\]) and hip flexion (range: 0 \[Normal\] - 5 \[severe weakness\]), lower scores= lower disease activity. Total MG-C score was obtained by summing responses to each individual item and score ranges from 0 to 50, with lower scores indicating lower disease activity. A positive change indicates worsening and a negative change indicates improvement. |
| Secondary | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Baseline, Weeks 5, 7, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 52 and 60 | 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 indicates worsening and a negative change indicates improvement. |
| Secondary | Percentage of Participants Using Rescue Medication (Intravenous Infusion of Immunoglobulin G (IVIg) or Plasma Exchange (PEX)) | From Baseline until End of Study (up to Week 60) | Rescue therapy consisted of IVIg or PEX. Study participants who experienced disease worsening (eg, an increase of 2 points on the MG-ADL or 3 points on the QMG scale between 2 consecutive visits) may be considered for rescue therapy at the discretion of the Investigator. |
Source ClinicalTrials.gov
Outcomes6
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 until End of Study (up to Week 60) | The Safety Set (SS) consisted of all randomized study participants who received at least 1 dose of IMP in this study. This endpoint was planned to be analyzed using the SS by most recent dose received i.e. the most recent dose received at or before the AE onset. Participants who switched doses were counted in both rozanolixizumab (RLZ) doses. | percentage of participants | NUMBER |
| PRIMARY | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Permanent Withdrawal of Study Medication | From Baseline until End of Study (up to Week 60) | The Safety Set consisted of all randomized study participants who received at least 1 dose of IMP in this study. This endpoint was planned to be analyzed using the SS by most recent dose received i.e. the most recent dose received at or before the AE onset. Participants who switched doses were counted in both rozanolixizumab doses. | percentage of participants | NUMBER |
| SECONDARY | Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Baseline, Weeks 5, 7, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 52 and 60 | The Safety Set consisted of all randomized study participants who received at least 1 dose of IMP in this study. Here, number analyzed signifies those participants who were evaluable at specified time points. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Baseline, Weeks 5, 7, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 52 and 60 | The Safety Set consisted of all randomized study participants who received at least 1 dose of IMP in this study. Here, number analyzed signifies those participants who were evaluable at specified time points. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Baseline, Weeks 5, 7, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 52 and 60 | The Safety Set consisted of all randomized study participants who received at least 1 dose of IMP in this study. Here, number analyzed signifies those participants who were evaluable at specified time points. | units on a scale | MEAN |
| SECONDARY | Percentage of Participants Using Rescue Medication (Intravenous Infusion of Immunoglobulin G (IVIg) or Plasma Exchange (PEX)) | From Baseline until End of Study (up to Week 60) | The Safety Set consisted of all randomized study participants who received at least 1 dose of IMP in this study. | percentage of participants | NUMBER |
Source ClinicalTrials.gov
Results by group96
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 | 76.0 | percentage of participants | ||
| Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) | Rozanolixizumab ~10 mg/kg | 78.6 | percentage of participants | ||
| Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Permanent Withdrawal of Study Medication | Rozanolixizumab ~7 mg/kg | 6.0 | percentage of participants | ||
| Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Permanent Withdrawal of Study Medication | Rozanolixizumab ~10 mg/kg | 0 | percentage of participants | ||
| Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Rozanolixizumab ~7 mg/kg | Week 5 | -2.7 | Standard Deviation 3.3 | units on a scale |
| Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Rozanolixizumab ~10 mg/kg | Week 5 | -3.2 | Standard Deviation 3.8 | units on a scale |
| Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Rozanolixizumab ~7 mg/kg | Week 7 | -2.7 | Standard Deviation 3.8 | units on a scale |
| Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Rozanolixizumab ~10 mg/kg | Week 7 | -3.7 | Standard Deviation 3.4 | units on a scale |
| Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Rozanolixizumab ~7 mg/kg | Week 9 | -2.8 | Standard Deviation 3.4 | units on a scale |
| Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods | Rozanolixizumab ~10 mg/kg | Week 9 | -3.4 | Standard Deviation 3.7 | units on a scale |
1 to 10 of 96Source ClinicalTrials.gov
See all 96 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Rozanolixizumab ~7 mg/kg | 35 | |
| Overall Study | STARTED | Rozanolixizumab ~10 mg/kg | 36 | |
| Overall Study | Safety Set | Rozanolixizumab ~7 mg/kg | 35 | |
| Overall Study | Safety Set | Rozanolixizumab ~10 mg/kg | 35 | |
| Overall Study | COMPLETED | Rozanolixizumab ~7 mg/kg | 5 | |
| Overall Study | COMPLETED | Rozanolixizumab ~10 mg/kg | 3 | |
| Overall Study | NOT COMPLETED | Rozanolixizumab ~7 mg/kg | 30 | |
| Overall Study | NOT COMPLETED | Rozanolixizumab ~10 mg/kg | 33 |
Source ClinicalTrials.gov
Adverse events38
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 | Rozanolixizumab ~7 mg/kg | 6 | 50 | 11 |
| other | Diarrhoea | Gastrointestinal disorders | Rozanolixizumab ~10 mg/kg | 7 | 42 | 10 |
| other | Abdominal pain | Gastrointestinal disorders | Rozanolixizumab ~7 mg/kg | 2 | 50 | 2 |
| other | Abdominal pain | Gastrointestinal disorders | Rozanolixizumab ~10 mg/kg | 2 | 42 | 2 |
| other | Nausea | Gastrointestinal disorders | Rozanolixizumab ~7 mg/kg | 4 | 50 | 4 |
| other | Nausea | Gastrointestinal disorders | Rozanolixizumab ~10 mg/kg | 5 | 42 | 8 |
| other | Vomiting | Gastrointestinal disorders | Rozanolixizumab ~7 mg/kg | 0 | 50 | 0 |
| other | Vomiting | Gastrointestinal disorders | Rozanolixizumab ~10 mg/kg | 4 | 42 | 4 |
| other | Pyrexia | General disorders | Rozanolixizumab ~7 mg/kg | 4 | 50 | 5 |
| other | Pyrexia | General disorders | Rozanolixizumab ~10 mg/kg | 3 | 42 | 4 |
1 to 10 of 38Source ClinicalTrials.gov
See all 38 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Rozanolixizumab ~7 mg/kg | 0 | 50 |
| Total, all-cause mortality | Rozanolixizumab ~10 mg/kg | 0 | 42 |
| Total, other adverse events | Rozanolixizumab ~7 mg/kg | 27 | 50 |
| Total, other adverse events | Rozanolixizumab ~10 mg/kg | 25 | 42 |
| Total, serious adverse events | Rozanolixizumab ~7 mg/kg | 7 | 50 |
| Total, serious adverse events | Rozanolixizumab ~10 mg/kg | 2 | 42 |
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Rozanolixizumab ~7 mg/kg vs Rozanolixizumab ~10 mg/kg: deaths from any cause | Trial comparisons |
| Rozanolixizumab ~7 mg/kg vs Rozanolixizumab ~10 mg/kg: 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.
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/NCT04124965
GET https://conductscience.org/api/trials/v1/NCT04124965/results?page=2