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

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

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.

Study Overview

• Status: Completed
• Conditions: Cold Agglutinin Disease
• Intervention / Treatment: Drug: sutimlimab

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Actual): 7
• Phase: Phase 3

Contacts and Locations

Study Locations

• Japan
  • Ishikawa, Japan, 920-8530
    • Investigational Site Number 3920005
  • Kanagawa, Japan, 259-1193
    • Investigational Site Number 3920004
  • Osaka, Japan, 565-0871
    • Investigational Site Number 3920003
  • Saitama, Japan, 350-0495
    • Investigational Site Number 3920002
  • Tokyo, Japan, 113-8431
    • Investigational Site Number 3920001

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 20 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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:

  1. Active, serious intercurrent illness which precluded enrolment until recovery was complete.
  2. 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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Sutimlimab; 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; Pharmaceutical form: solution for injection Route of administration: intravenous (IV)

What is the study measuring?

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)

Collaborators and Investigators

• Sponsor: Sanofi
• Investigators: Study Director: Clinical Sciences & Operations, Sanofi

Study record dates

Study Major Dates

• Study Start (Actual): November 11, 2021
• Primary Completion (Actual): November 15, 2022
• Study Completion (Actual): November 15, 2022

Study Registration Dates

• First Submitted: November 11, 2021
• First Submitted That Met QC Criteria: November 11, 2021
• First Posted (Actual): November 24, 2021

Study Record Updates

• Last Update Posted (Actual): October 3, 2023
• Last Update Submitted That Met QC Criteria: September 8, 2023
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Immune System Diseases; Autoimmune Diseases; Hematologic Diseases; Anemia; Anemia, Hemolytic; Anemia, Hemolytic, Autoimmune
• Other Study ID Numbers: LTS17352; U1111-1266-5421 (Registry Identifier: ICTRP)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Drug and device information, study documents

• 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