Cold Agglutinin Disease
Conditions
Brief summary
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
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.
Interventions
DRUGsutimlimab
Pharmaceutical form: solution for injection Route of administration: intravenous (IV)
Sponsors
Sanofi
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
20 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) | From first dose of study intervention up to 9 weeks after the last dose of study intervention (maximum duration: 49 weeks) | 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). |
| Number of Participants With Treatment-emergent Adverse Events of Special Interest (AESI) | From first dose of study intervention up to 9 weeks after the last dose of study intervention (maximum duration: 49 weeks) | 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. |
Countries
Japan
Participant flow
Recruitment details
The study was conducted at 5 active sites in Japan. A total of 7 participants were enrolled from 11 November 2021 to 07 December 2021. Participants with cold agglutinin disease (CAD) and who had completed Part B of CARDINAL (NCT03347396) or CADENZA (NCT03347422) study and benefitted from sutimlimab treatment were enrolled in the current study.
Pre-assignment details
Per protocol, enrolled participants were planned to receive sutimlimab based on their baseline body weight as: 6.5 grams for baseline body weight of greater than or equal to (\>=) 39 kilograms (kg) to less than (\<) 75 kg or 7.5 grams for baseline body weight of \>= 75 kg. As no participant had a Baseline body weight \>= 75 kg, per the study protocol, 7.5 grams dose was not administered.
Participants by arm
| Arm | Count |
|---|---|
| Sutimlimab Participants with body weight \>= 39 kg to \< 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 IV infusion on Day 0, Day 7, Day 21 and thereafter every 2 weeks (maximum duration: 49 weeks) in the current study. | 7 |
| Total | 7 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 1 |
Baseline characteristics
| Characteristic | Sutimlimab |
|---|---|
| Age, Continuous | 69.3 years STANDARD_DEVIATION 12.8 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 7 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 0 Participants |
| Sex: Female, Male Female | 5 Participants |
| Sex: Female, Male Male | 2 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 1 / 7 |
| other Total, other adverse events | 7 / 7 |
| serious Total, serious adverse events | 1 / 7 |
Outcome results
Primary
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.
Time frame: From first dose of study intervention up to 9 weeks after the last dose of study intervention (maximum duration: 49 weeks)
Population: Analysis was performed on safety population.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Sutimlimab | Number of Participants With Treatment-emergent Adverse Events of Special Interest (AESI) | 0 Participants |
Primary
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).
Time frame: From first dose of study intervention up to 9 weeks after the last dose of study intervention (maximum duration: 49 weeks)
Population: Analysis was performed on safety population.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Sutimlimab | Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) | TEAEs | 7 Participants |
| Sutimlimab | Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) | TESAEs | 1 Participants |