A Study to Evaluate Rozanolixizumab in Study Participants With Generalized Myasthenia Gravis

• ClinicalTrials.gov Identifier: NCT04650854
• Recruitment Status: Completed
• First Posted: December 3, 2020
• Last Update Posted: February 26, 2024
• Sponsor: UCB Biopharma SRL
• Information provided by (Responsible Party): UCB Pharma ( UCB Biopharma SRL )

Tracking Information

• First Submitted Date: November 5, 2020
• First Posted Date: December 3, 2020
• Last Update Posted Date: February 26, 2024
• Actual Study Start Date: February 3, 2021
• Actual Primary Completion Date: January 25, 2024 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: November 24, 2020)

• 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.

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: November 24, 2020)

• 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.

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Study to Evaluate Rozanolixizumab in Study Participants With Generalized Myasthenia Gravis
• Official Title: An Open-Label Extension Study to Evaluate Rozanolixizumab in Study Participants With Generalized Myasthenia Gravis
• Brief Summary: The purpose of this study is to assess the safety, tolerability and efficacy of additional 6-week treatment cycles with rozanolixizumab in study participants with generalized myasthenia gravis (gMG).
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Randomized; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Generalized Myasthenia Gravis

Intervention

Drug: Rozanolixizumab

Rozanolixizumab will be administered by subcutaneous infusion in dosage regimen 1 or 2.

Other Name: UCB7665

Study Arms

• 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.
  Intervention: Drug: Rozanolixizumab
• 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.
  Intervention: Drug: Rozanolixizumab

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: February 10, 2022): 165
• Original Estimated Enrollment (submitted: November 24, 2020): 230
• Actual Study Completion Date: January 25, 2024
• Actual Primary Completion Date: January 25, 2024 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Study participant must meet one of the following:

  1. completed MG0003 [NCT03971422]
  2. required rescue therapy during the Observation Period in MG0003 or
  3. 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Canada, Czechia, Denmark, France, Georgia, Germany, Italy, Japan, Poland, Russian Federation, Serbia, Spain, Taiwan, United States
• Removed Location Countries: Hungary

Administrative Information

• NCT Number: NCT04650854
• Other Study ID Numbers: MG0007; 2020-003230-20 ( EudraCT Number )
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• 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

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
• Supporting Materials: Statistical Analysis Plan (SAP)
• Supporting Materials: 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

• Current Responsible Party: UCB Pharma ( UCB Biopharma SRL )
• Original Responsible Party: Same as current
• Current Study Sponsor: UCB Biopharma SRL
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Director: UCB Cares; 001 844 599 22733 (UCB)

• PRS Account: UCB Pharma
• Verification Date: February 2024