A Study to Evaluate Rozanolixizumab in Study Participants With Generalized Myasthenia Gravis
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| ClinicalTrials.gov Identifier: NCT04650854 |
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Recruitment Status :
Active, not recruiting
First Posted : December 3, 2020
Last Update Posted : June 7, 2023
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| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Generalized Myasthenia Gravis | Drug: Rozanolixizumab | Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 165 participants |
| Allocation: | Randomized |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | An Open-Label Extension Study to Evaluate Rozanolixizumab in Study Participants With Generalized Myasthenia Gravis |
| Actual Study Start Date : | February 3, 2021 |
| Estimated Primary Completion Date : | January 31, 2024 |
| Estimated Study Completion Date : | January 31, 2024 |
| Arm | Intervention/treatment |
|---|---|
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Experimental: Rozanolixizumab dosage regimen 1
Study participants randomized/assigned to dosage regimen 1 will receive assigned dosage of rozanolixizumab for the initial cycle. The dose regimen may be switched before the start of each subsequent treatment cycle based on investigator discretion.
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Drug: Rozanolixizumab
Rozanolixizumab will be administered by subcutaneous infusion in dosage regimen 1 or 2.
Other Name: UCB7665 |
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Experimental: Rozanolixizumab dosage regimen 2
Study participants randomized/assigned to dosage regimen 2 will receive assigned dosage of rozanolixizumab for the initial cycle. The dose regimen may be switched before the start of each subsequent treatment cycle based on investigator discretion.
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Drug: Rozanolixizumab
Rozanolixizumab will be administered by subcutaneous infusion in dosage regimen 1 or 2.
Other Name: UCB7665 |
- Percentage of participants with treatment-emergent adverse events (TEAEs) [ Time Frame: From Baseline (Day 1) to End of Study (average of 20 months) ]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.
- Percentage of participants with TEAEs leading to withdrawal of investigational medicinal product (IMP) [ Time Frame: From Baseline (Day 1) to End of Study (average of 20 months) ]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. AEs leading to permanent withdrawal of study medication.
- Change from Baseline (Day 1) to Day 43 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score within one treatment cycle [ Time Frame: From Baseline (Day 1) to end of treatment cycle (up to 6 weeks) ]
The Outcome Measure is applicable for the first 3 treatment cycles. The total MG-ADL score is obtained by summing the responses to each individual item (8 items; Grades: 0, 1, 2, 3). The score ranges from 0 to 24, with a higher score indicating more disability.
A positive change indicates worsening and a negative change indicates improvement.
- Change from Baseline (Day 1) to Day 43 in Quantitative Myasthenia Gravis (QMG) score within one treatment cycle [ Time Frame: From Baseline (Day 1) to end of treatment cycle (up to 6 weeks) ]The Outcome Measure is applicable for the first 3 treatment cycles. The total QMG 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.
- Change from Baseline (Day 1) to Day 43 in Myasthenia Gravis-Composite (MG-C) score within one treatment cycle [ Time Frame: From Baseline (Day 1) to end of treatment cycle (up to 6 weeks) ]The Outcome Measure is applicable for the first 3 treatment cycles. The total MG-C score is obtained by summing the responses to each individual item (10 items; Grade:0-9 depending on item). The score ranges from 0 to 50, with lower scores indicating lower disease activity.
- 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 [ Time Frame: From Baseline (Day 1) to end of treatment cycle (up to 6 weeks) ]
The Outcome Measure is applicable for the first 3 treatment cycles. The MG symptoms PRO instrument consists of 42 items across 5 scales: ocular symptoms (items 1-5); bulbar symptoms (items 6-15); respiratory symptoms (items 16-18); physical fatigue (items 19-33) and muscle weakness fatigability (items 34-42).
The study participant will be asked to choose the response option that best describes the severity of ocular, bulbar, and respiratory symptoms over the past 7 days using a 4-point Likert scale ("none" to "severe") and how frequently they experience physical fatigue and muscle weakness fatigability over the past 7 days using a 5-point Likert scale ("none of the time" to "all of the time"), respectively.
- Change from Baseline (Day 1) to Day 43 in MG Symptoms PRO 'Physical Fatigue' score within one treatment cycle [ Time Frame: From Baseline (Day 1) to end of treatment cycle (up to 6 weeks) ]
The Outcome Measure is applicable for the first 3 treatment cycles. The MG symptoms PRO instrument consists of 42 items across 5 scales: ocular symptoms (items 1-5); bulbar symptoms (items 6-15); respiratory symptoms (items 16-18); physical fatigue (items 19-33) and muscle weakness fatigability (items 34-42).
The study participant will be asked to choose the response option that best describes the severity of ocular, bulbar, and respiratory symptoms over the past 7 days using a 4-point Likert scale ("none" to "severe") and how frequently they experience physical fatigue and muscle weakness fatigability over the past 7 days using a 5-point Likert scale ("none of the time" to "all of the time"), respectively.
- Change from Baseline (Day 1) to Day 43 in MG Symptoms PRO 'Bulbar symptoms' score within one treatment cycle [ Time Frame: From Baseline (Day 1) to end of treatment cycle (up to 6 weeks) ]
The Outcome Measure is applicable for the first 3 treatment cycles. The MG symptoms PRO instrument consists of 42 items across 5 scales: ocular symptoms (items 1-5); bulbar symptoms (items 6-15); respiratory symptoms (items 16-18); physical fatigue (items 19-33) and muscle weakness fatigability (items 34-42).
The study participant will be asked to choose the response option that best describes the severity of ocular, bulbar, and respiratory symptoms over the past 7 days using a 4-point Likert scale ("none" to "severe") and how frequently they experience physical fatigue and muscle weakness fatigability over the past 7 days using a 5-point Likert scale ("none of the time" to "all of the time"), respectively.
- MG-ADL responder (≥2.0-point improvement from Baseline [Day 1] to end of Day 43) within one treatment cycle [ Time Frame: From Baseline (Day 1) to end of treatment cycle (up to 6 weeks) ]The Outcome Measure is applicable for the first 3 treatment cycles. A MG-ADL responder is defined as achieving at least 2.0-point improvement in the MG-ADL score from Baseline.
- Time to MG-ADL response (≥2.0-point improvement from Baseline [Day 1]) within one treatment cycle [ Time Frame: From Baseline (Day 1) to end of treatment cycle (up to 6 weeks) ]The Outcome Measure is applicable for the first 3 treatment cycles. Time to achieve MG-ADL response, defined as at least 2.0-point improvement from Baseline.
- Time between consecutive treatment cycles [ Time Frame: From end of the 6-week treatment cycle (Day 43) to the next 6-week treatment cycle (Day 1), assessed up to 2.5 years ]Time between consecutive treatment cycles: Study participants will be assessed for MG worsening prior to repeated cycles. In case of symptom worsening (eg, an increase of 2.0 points on the MG-ADL or 3.0 points on the QMG scale) between assessments, resulting in a need for additional treatment, study participants will undergo another 6-week treatment cycle followed by an Observation Period, based on the Investigator's discretion.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
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Study participant must meet one of the following:
- completed MG0003 [NCT03971422]
- required rescue therapy during the Observation Period in MG0003 or
- completed at least 6 visits in MG0004 [NCT04124965]
- Body weight ≥35 kg at Baseline (Day 1)
- Study participants may be male or female
Exclusion Criteria:
- Study participant has a known hypersensitivity to any components of the study medication or other anti-neonatal Fc receptor (FcRn) medications
- Study participant with a known tuberculosis (TB) infection, at high risk of acquiring TB infection, or latent tuberculosis infection (LTBI), or current/history of nontuberculous mycobacterial infection (NTMBI)
- Study participant met any mandatory withdrawal or mandatory study drug discontinuation criteria in MG0003, or MG0004, or permanently discontinued study drug in either study
- Study participant intends to have a live vaccination during the course of the study or within 8 weeks following the final dose of rozanolixizumab
- Study participant with severe (defined as Grade 3 on the Myasthenia Gravis-Activities of Daily Living (MG-ADL) scale) weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04650854
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| Study Director: | UCB Cares | 001 844 599 22733 (UCB) |
| Responsible Party: | UCB Biopharma SRL |
| ClinicalTrials.gov Identifier: | NCT04650854 |
| Other Study ID Numbers: |
MG0007 2020-003230-20 ( EudraCT Number ) |
| First Posted: | December 3, 2020 Key Record Dates |
| Last Update Posted: | June 7, 2023 |
| Last Verified: | June 2023 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | Yes |
| Plan Description: | 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. |
| Supporting Materials: |
Study Protocol Statistical Analysis Plan (SAP) Clinical Study Report (CSR) |
| Time Frame: | Data from this study 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. |
| Access Criteria: | 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. |
| URL: | http://www.Vivli.org |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
| Product Manufactured in and Exported from the U.S.: | Yes |
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UCB7665 generalized myasthenia gravis rozanolixizumab gMG |
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Myasthenia Gravis Muscle Weakness Muscular Diseases Musculoskeletal Diseases Neuromuscular Manifestations Neurologic Manifestations Nervous System Diseases Pathologic Processes Paraneoplastic Syndromes, Nervous System Nervous System Neoplasms Neoplasms by Site Neoplasms |
Paraneoplastic Syndromes Autoimmune Diseases of the Nervous System Neurodegenerative Diseases Neuromuscular Junction Diseases Neuromuscular Diseases Autoimmune Diseases Immune System Diseases Rozanolixizumab Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs |