Celerion

• The study enrolled 9 participants across France, Israel, Italy, and the Republic of Korea.
• A May 2022 amendment changed the design from a single-arm two-stage study to a single-arm study with two interim analyses.
• Eligibility criteria were updated in amendments dated May 2022 and March 2023.
• A March 2023 amendment updated definitions for time to first deterioration in cough, pain, dyspnea, and physical functioning.
• Paper diary cards were added for sites in China in a March 2023 amendment.

These findings describe the trial, not the service provider’s performance or responsibility.

• Recruitment began on 1 March 2022, and 98 participants were randomized at 57 sites in 19 countries.
• A May 2022 amendment changed the primary endpoint from complete remission on minimal OCS to CRoff at week 36 and added CRmin and IGA-BP-off-OCS secondary endpoints.
• Infection-related eligibility criteria were tightened across amendments, including exclusion of active or chronic infections and later clinically significant active infection unresolved before baseline.
• A March 2023 amendment added exclusions for latent infection, severe renal impairment, specified safety or procedure-affecting conditions, concurrent interventional-study enrollment, inadequately treated HIV, and recent live or live-attenuated vaccination.
• Sponsor confirmation of participant eligibility was removed in the March 2023 amendment.
• The March 2023 amendment required at least 30 minutes of on-site safety monitoring after investigational-product administration, and this requirement was removed in February 2024.
• The Day 10 PK sampling requirement in Part A was clarified to require samples from at least 28 participants rather than all participants.
• All adverse events in the MedDRA Infections and Infestations system organ class were designated adverse events of special interest in the May 2022 amendment.

These findings describe the trial, not the service provider’s performance or responsibility.

• The study was conducted in 11 countries: Bulgaria, Croatia, Germany, Greece, Hungary, Japan, Poland, Romania, Slovakia, Spain, and the United States of America.

These findings describe the trial, not the service provider’s performance or responsibility.

• The study was conducted in China, Japan, Serbia, Slovakia, South Africa, and the United States of America.

These findings describe the trial, not the service provider’s performance or responsibility.

• The study enrolled 69 participants with AChR-Ab-seropositive generalized myasthenia gravis at 31 sites in 11 countries after screening 98 participants.
• The fixed cyclic-regimen arm enrolled only 17 participants because of the 3:1 randomization, which was identified as a study limitation.
• A protocol amendment required the first three efgartigimod IV infusions of the first treatment period to be administered at the site.
• A protocol amendment added a safety follow-up visit that could be conducted remotely or by telephone.

These findings describe the trial, not the service provider’s performance or responsibility.