Sutimlimab (BIVV009) for the Adult Participants With Cold Agglutinin Disease (CAD) Who Have Completed Phase 3 Studies (CARDINAL or CADENZA) in Japan

Study Overview
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Brief Summary
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This was a multi-center, single treatment-group, open-label study to provide sutimlimab to the adult participants with cold agglutinin disease (CAD) who had completed the CARDINAL (NCT number: NCT03347396) or CADENZA (NCT number: NCT03347422) studies and benefitted from sutimlimab treatment in Japan. • Study and treatment duration: the period between the participant's completion of the CARDINAL and CADENZA studies and sutimlimab or other appropriate CAD therapy becoming commercially available to participants in Japan.

Detailed Description
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The period between screening/baseline visit (upon the participant's completion* of the CARDINAL and CADENZA studies) and end of treatment with sutimlimab in this study was determined by sutimlimab or other appropriate CAD therapy becoming commercially available to participants in Japan.

Official Title
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An Open-label Study for Sutimlimab in Participants With Cold Agglutinin Disease (CAD) Who Have Completed the CARDINAL Study (BIVV009-03/EFC16215, Part B) or CADENZA Study (BIVV009-04/EFC16216, Part B) in Japan

Conditions
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Cold Agglutinin Disease

Intervention / Treatment
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* Drug: sutimlimab


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: --Participant must be adults. Participants who had been enrolled in and had completed Part B of CARDINAL or CADENZA study. Participants who had ongoing diagnosis of CAD. Participants who continued to require treatment for CAD upon completion of participation in the previous study evidenced by return of CAD-related symptoms of anemia and/or deterioration on markers of hemolysis after the end of study visit following the 9-week safety follow up period. (9-week follow up period). Participants who had acceptable benefit/risk profile. Participant who had acceptable infection risk. Participants who had no available appropriate alternative therapy for CAD. Body weight >= 39 kg. Gave signed informed consent. Exclusion Criteria: --Clinical diagnosis of systemic lupus erythematosus or immune complex-mediated autoimmune disorders. Participants who met recent Rituximab and/or immunosuppressive therapy. Any of the following medical conditions: Active, serious intercurrent illness which precluded enrolment until recovery was complete. Pregnancy or breast-feeding. End of Study visit in CARDINAL or CADENZA took place more than 3 months before Baseline visit in this study. Hypersensitivity reactions to sutimlimab or components thereof, or other allergy that, in the opinion of the Investigator, contraindicated participation in the study.

Ages Eligible for Study
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Minimum Age: 20 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Sutimlimab<br>Participants with body weight greater than or equal to (>=) 39 kilograms (kg) to less than (<) 75 kg and who had completed Part B of CARDINAL or CADENZA study were enrolled in the current study and received sutimlimab (BIVV009) 6.5 grams as intravenous (IV) infusion on Day 0, Day 7, Day 21 and thereafter every 2 weeks (maximum duration: 49 weeks) in the current study. | Drug: sutimlimab<br>* Pharmaceutical form: solution for injection Route of administration: intravenous (IV)<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) | An Adverse Event (AE) was defined as any untoward medical occurrence in a participant who received study intervention and did not necessarily had to have a causal relationship with the treatment. Serious adverse events (SAEs) were defined as any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/ incapacity, was a congenital anomaly/birth defect, suspected transmission of any infectious agent via an authorized medicinal product, was a medically important event. TEAEs were defined as AEs that developed, worsened or became serious during the treatment-emergent period (from first dose of study intervention up to 9 weeks after the last dose of study intervention in the current study). | From first dose of study intervention up to 9 weeks after the last dose of study intervention (maximum duration: 49 weeks) | 
| Number of Participants With Treatment-emergent Adverse Events of Special Interest (AESI) | An AE was defined as any untoward medical occurrence in a participant who received study intervention and did not necessarily had to have a causal relationship with the treatment. AESIs were AE (serious or non-serious) of scientific and medical concern specific to the Sponsor's product or program, for which ongoing monitoring and immediate notification by the investigator to the Sponsor was required. | From first dose of study intervention up to 9 weeks after the last dose of study intervention (maximum duration: 49 weeks) |