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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

Sutimlimab (BIVV009) for the adult participants with cold agglutinin disease (CAD) who have completed Phase 3 studies (CARDINAL or CADENZA) in Japan (CARMEN-GC01)

7

All 7 participants who were from Japan completed the Part B of CARDINAL or CADENZA study and were eligible for this study. All 7 enrolled participants received at least 1 dose of sutimlimab and were included in the safety population. The participants had a median age o 73.0 years (range: 49 to 85) with a majority (5 [71.4%] participants) >=65 years of age. All participants were Asian, and a majority (5 [71.4%]) were female. More than half (4 [57.1%]) of participants had Raynaud's phenomenon, 3 (42.9%) had acrocyanosis, and 1 (14.3%) had disabling circulatory symptoms as cold agglutinin disease (CAD) circulatory symptoms at baseline.

- Treated: 7 - Completed: 6 - Not Completed: 1

Refer to "Outcome measures"

In the safety population, all 7 (100%) participants experienced at least 1 treatment-emergent adverse event (TEAE), with a total of 46 TEAEs reported. The most frequently (>=20% [ie, >=2 participants]) reported TEAEs were back pain (3 [42.9%] participants) and pyrexia (2 [28.6%]). One (14.3%) of the 7 participants had a TEAE assessed by the Investigator as related to sutimlimab. This event was nonserious urinary tract infection and resolved after medication. In total, 3 TESAEs were reported in 1 (14.3%) participant who died in the study. The reported TESAEs were cholangitis acute, spontaneous bacterial peritonitis, and chronic kidney disease (all during the treatment period). None of them were assessed as related to sutimlimab by the Investigator. Among 3 TESAEs, spontaneous bacterial peritonitis and chronic kidney disease led to discontinuation of the study intervention. This participant died of chronic kidney disease. There were no other participants who had serious TEAEs or TEAEs leading to treatment discontinuation. No adverse events of special interest (AESIs) were reported in the study.

All 7 participants who completed the previous studies in Japan were enrolled and received sutimlimab. All 7 participants experienced at least 1 TEAE. The most frequently reported TEAEs were back pain and pyrexia. In total, 3 TESAEs (cholangitis acute, spontaneous bacterial peritonitis, and chronic kidney disease) were reported in 1 participant who died. All of TESAEs were assessed as not related to sutimlimab. There were no other participants who had serious TEAEs. No AESIs were reported in the study.

Yes

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

https://jrct.mhlw.go.jp/latest-detail/jRCT2031210392

Tanaka Tomoyuki

Sanofi K.K.

Tokyo Opera City Tower, 3-20-2, Nishi Shinjuku, Shinjuku-ku, Tokyo 163-1488, Japan

clinical-trials-jp@sanofi.com

Clinical Study Unit

Sanofi K.K.

Tokyo Opera City Tower, 3-20-2, Nishi Shinjuku, Shinjuku-ku, Tokyo 163-1488, Japan

+81-3-6301-3670

clinical-trials-jp@sanofi.com

Complete

Oct. 29, 2021

Interventional

non-randomized controlled trial

open(masking not used)

uncontrolled control

single assignment

treatment purpose

- Participant must be adults.
- Participants who have been enrolled in, and has completed Part B of CARDINAL or CADENZA study
- Participants who have ongoing diagnosis of cold agglutinin disease (CAD)
- Participants who continue 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 have acceptable benefit/risk profile
- Participant who has acceptable infection risk
- Participants who have no available appropriate alternative therapy for CAD
- Body weight of >=39 kg
- Giving signed informed consent

Participants are excluded from the study if any of the following criteria apply:
- Clinical diagnosis of systemic lupus erythematosus (SLE) or immune complex mediated autoimmune disorders.
- Participants who meet recent Rituximab and/or immunosuppressive therapy.
- Any of the following medical conditions:
a) Active, serious intercurrent illness which will preclude enrolment until recovery is complete.
b) 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, contraindicates participation in the study.

Both

Cold Agglutinin Disease

Drug: sutimlimab (BIVV009)
Pharmaceutical Form: solution for injection, Route of Administration: intravenous(iv)

1. Number of Participants With Treatment-emergent Adverse Events (AEs) and Serious AEs (SAEs) [ Time Frame: Approximately 1 year ]

2. Number of Participants With Treatment-emergent Adverse Events of Special Interest (AESI) [ Time Frame: Approximately 1 year ]

N/A

+81-3-5923-3111

Sept. 07, 2021

none