A Phase 3, Open-label, Crossover Study to Evaluate Self-administration of Rozanolixizumab by Study Participants With Generalized Myasthenia Gravis (gMG)

• ClinicalTrials.gov Identifier: NCT05681715
• Recruitment Status: Active, not recruiting
• First Posted: January 12, 2023
• Last Update Posted: April 26, 2024
• Sponsor: UCB Biopharma SRL
• Information provided by (Responsible Party): UCB Pharma ( UCB Biopharma SRL )

Tracking Information

• First Submitted Date: January 4, 2023
• First Posted Date: January 12, 2023
• Last Update Posted Date: April 26, 2024
• Actual Study Start Date: April 17, 2023
• Estimated Primary Completion Date: April 24, 2024 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: January 4, 2023)

• Successful self-administration of rozanolixizumab (with correct use of syringe driver and manual push, respectively) during the Self-administration Period at Visit 13 [ Time Frame: Visit 13 (Week 12; last dose of Self-administration Period 1) ]
  Successful self-administration is defined by the participant (i) choosing the correct infusion site, (ii) administering subcutaneous, and (iii) delivering the intended dose.
• Successful self-administration of rozanolixizumab (with correct use of syringe driver and manual push, respectively) during the Self-administration Period at Visit 19 [ Time Frame: Visit 19 (Week 18; last dose of Self-administration Period 2) ]
  Successful self-administration is defined by the participant (i) choosing the correct infusion site, (ii) administering subcutaneous, and (iii) delivering the intended dose.

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: January 4, 2023)

• Occurrence of Treatment-Emergent Adverse Events (TEAEs) after syringe driver or manual push self-administration from Visit 2 up to the End of Study Visit [ Time Frame: From Visit 2 (Week 1) up to the End of Study Visit (Visit 21 [Week 26]) ]
  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.
• Occurrence of local site reactions up to 24 hours after each administration during the Training Period and Self-administration Periods [ Time Frame: Up to 24 hours after each administration during the Training Period (Baseline to Visit 7 [Week 6] and Self-administration Periods (Visit 8 [Week 7] to Visit 19 [Week 18]) ]
  Local site reaction Adverse Events (AEs) will be considered treatment-emergent up to 24 hours after each administration during the Training Period and Self-administration Periods.
• Occurrence of medication errors associated with adverse reactions during the 2 Self-administration Periods of the study [ Time Frame: During the Self-administration Periods (Visit 8 [Week 7] to Visit 19 [Week 18]) ]
  Medication errors are defined as an unintended failure in the drug treatment process that leads to, or has the potential to lead to, harm to the study participant. Medication Errors associated with adverse reactions during the 2 Self-administration Periods will be measured.

• 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 Phase 3, Open-label, Crossover Study to Evaluate Self-administration of Rozanolixizumab by Study Participants With Generalized Myasthenia Gravis (gMG)
• Official Title: An Open-label, Crossover Study to Evaluate Rozanolixizumab Self-administration by Study Participants With Generalized Myasthenia Gravis
• Brief Summary: The purpose of this study is to evaluate the ability of study participants with generalized Myasthenia Gravis (gMG) to successfully self-administer rozanolixizumab after training in the self-administration technique using the syringe driver and manual push methods.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Randomized; Intervention Model: Crossover Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Generalized Myasthenia Gravis

Intervention

Drug: Rozanolixizumab

Rozanolixizumab self-administration via Syringe Driver or Manual Push.

Study Arms

• Experimental: Rozanolixizumab Sequence 1: Syringe Driver - Manual Push
  Study participants will receive predefined weekly doses of rozanolixizumab for 18 weeks.
  Intervention: Drug: Rozanolixizumab
• Experimental: Rozanolixizumab Sequence 2: Manual Push - Syringe Driver
  Study participants will receive predefined weekly doses of rozanolixizumab for 18 weeks.
  Intervention: Drug: Rozanolixizumab

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Active, not recruiting
• Actual Enrollment (submitted: December 7, 2023): 62
• Original Estimated Enrollment (submitted: January 4, 2023): 30
• Estimated Study Completion Date: April 24, 2024
• Estimated Primary Completion Date: April 24, 2024 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Study participant must have a documented diagnosis of generalized Myasthenia Gravis (gMG)
• Study participant is willing to perform and capable of performing home self-administration
• Study participant is considered by the investigator for additional rozanolixizumab treatment with the posology proposed in this study.
• Body weight ≥35 kg
• Study participants may be male or female

Exclusion Criteria:

• Study participant has a known hypersensitivity to other anti-Fc receptor (FcRn) medications, to any components of the study medication, to any of the excipients (including polysorbate 80), or has a known history of hyperprolinemia, since both polysorbate 80 and L-proline are constituents of the rozanolixizumab formulation
• Study participant with a known tuberculosis (TB) infection, at high risk of acquiring TB infection, or latent tuberculosis infection (LTBI), or current or history of nontuberculous mycobacterial infection (NTMBI)
• Study participant has a clinically relevant active infection or a history of serious infection (resulting in hospitalization or requiring IV antibiotic treatment) within 6 weeks before the Baseline Visit
• The study participant previously participated in any rozanolixizumab MG study and met any mandatory withdrawal criteria (unless the reason is directly related to MG0020 participation) or mandatory study drug discontinuation criteria.
• Study participant has received a live vaccination within 4 weeks before starting treatment, or a Bacillus Calmette-Guérin (BCG) vaccine within 1 year before starting treatment; or intends to have a live vaccination during the course of the study or within 8 weeks following the last 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, Georgia, Germany, Italy, Japan, Poland, Serbia, Spain, United Kingdom, United States

Administrative Information

• NCT Number: NCT05681715
• Other Study ID Numbers: MG0020; 2022-003870-21 ( 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.: No

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: https://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 2273

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