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| ID | Type | Description | Link |
|---|---|---|---|
| 2022-502074-16-00 | Registry Identifier | CTIS | |
| U1111-1285-0787 | Other Identifier | Universal Trial Number |
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The purpose of the study is to assess the safety and tolerability of subcutaneous (sc) administration of rozanolixizumab in pediatric participants aged ≥2 to <18 years with generalized Myasthenia Gravis (gMG).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| rozanolixizumab | Experimental | Study participants will receive pre-defined doses of rozanolixizumab for 6 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| rozanolixizumab | Drug | rozanolixizumab solution for injection |
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| Measure | Description | Time Frame |
|---|---|---|
| Occurrence of serious Treatment-Emergent Adverse Events (TEAEs) up to the End of Study (EOS) Visit | Serious TEAEs are any untoward medical incidence in a subject during administered study treatment, whether or not these events are related to study treatment and additionally are emergent untoward medical occurrence that at any dose:
| From Baseline up to the EOS Visit (up to 18 weeks) |
| Occurrence of TEAEs leading to permanent withdrawal of Investigational Medicinal Product (IMP) up to the EOS Visit | An Adverse Event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have 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. | From Baseline up to the EOS Visit (up to 18 weeks) |
| Occurrence of Adverse Event(s) of Special Monitoring (AESM) up to the EOS Visit (up to 18 weeks) | AESMs are: Severe and/or serious headache, suspected aseptic meningitis, severe Gastrointestinal (GI) disorders, and opportunistic infection. | From Baseline up to the EOS Visit (up to 18 weeks) |
| Measure | Description | Time Frame |
|---|---|---|
| Percent change in total Immunoglobulin G (IgG) from Baseline at the end of Week 6 | Plasma concentration analyses of total IgG will be done for all study participants on an ongoing basis throughout the study. | From Baseline to the end of Week 6 |
| Absolute change in total IgG from Baseline at the end of Week 6 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| UCB Cares | 001 844 599 2273 | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Mg0006 40290 | Bologna | Italy | ||||
| Mg0006 40144 |
Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.
Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.
Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed.All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.
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Plasma concentration analyses of total IgG will be done for all study participants on an ongoing basis throughout the study. |
| From Baseline to the end of Week 6 |
| Percent change from Baseline in myasthenia gravis (MG) autoantibody levels at the end of Week 6 | Plasma concentration analyses of MG specific anti-acetylcholine receptor (AChR) or anti-muscle-specific kinase (MuSK) autoantibodies will be done for all study participants on an ongoing basis throughout the study. | From Baseline to the end of Week 6 |
| Absolute change from Baseline in MG-specific autoantibody levels at the end of Week 6 | Plasma concentration analyses of anti-AChR or anti-MuSK autoantibodies will be done for all study participants on an ongoing basis throughout the study. | From Baseline to the end of Week 6 |
| Change from Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score at the end of Week 6 | The MG-ADL score is an 8-item patient-reported outcome (PRO) instrument. The MG-ADL targets symptoms and disability across ocular, bulbar, respiratory, and axial symptoms. The item responses are scored from 0 to 3, and the total score of MG-ADL is the sum of the 8 items and ranges from 0 to 24, with a higher score indicating more disability. | From Baseline to the end of Week 6 |
| Change from Baseline in Quantitative Myasthenia Gravis (QMG) total score at the end of Week 6 | QMG score is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The QMG total score is obtained by summing the responses to each individual item (13 items; Responses: None=0, Mild=1, Moderate=2, Severe=3). The score ranges from 0 to 39, with lower scores indicating lower disease activity. | From Baseline to the end of Week 6 |
| Occurrence of other TEAEs (including headache, nausea, and infusion site reactions) during Treatment Period 1 (TP1) and Observation Period 1 (OP1) | An Adverse Event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have 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. | During TP1 and OP1 (up to 14 weeks) |
| Evaluation of local tolerability at each scheduled assessment during TP1 | Local tolerability will be evaluated at each scheduled assessment for all study participants during TP1. | At each scheduled assessment during TP1 (Baseline, week 2, 3, 4, 5, up to 6 weeks) |
| Plasma concentration of rozanolixizumab at the 6-week treatment cycle | Plasma concentration analyses of rozanolixizumab will be done for all study participants on an ongoing basis throughout the study. | At the 6-week treatment cycle |
| Incidence of antidrug antibodies (ADAs) at the end of Week 6 | Plasma concentration analyses of ADAs will be done for all study participants on an ongoing basis throughout the study. | At the end of Week 6 |
| Milan |
| Italy |
| Mg0006 40733 | Naples | Italy |
| Mg0006 20340 | Fuchu-shi | Japan |
| Mg0006 20339 | Ōbu | Japan |
| Mg0006 20343 | Sagamihara | Japan |
| Mg0006 40155 | Warsaw | Poland |
| Mg0006 40734 | Warsaw | Poland |
| Mg0006 20081 | Taipei | Taiwan |
| Mg0006 20095 | Taipei | Taiwan |
| Mg0006 40841 | Altindağ/ankara | Turkey (Türkiye) |
| Mg0006 40843 | Ankara | Turkey (Türkiye) |
| Mg0006 40836 | Kocaeli | Turkey (Türkiye) |
| ID | Term |
|---|---|
| D009157 | Myasthenia Gravis |
| ID | Term |
|---|---|
| D020361 | Paraneoplastic Syndromes, Nervous System |
| D009423 | Nervous System Neoplasms |
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
| D010257 | Paraneoplastic Syndromes |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
| D019636 | Neurodegenerative Diseases |
| D020511 | Neuromuscular Junction Diseases |
| D009468 | Neuromuscular Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
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| ID | Term |
|---|---|
| C000627812 | rozanolixizumab |
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