NCT04269551

A Safety and Tolerability Study of BIVV020 in Adults With Cold Agglutinin Disease

Completed Phase 1 Last updated 22 September 2025

What this trial tests

Phase 1 trial testing BIVV020 in Autoimmune Haemolytic Anaemia in 12 participants. Completed in 6 January 2022.

Timeline

15 June 2020

Primary endpoint
6 January 2022

6 January 2022

Quick facts

Lead sponsor	Bioverativ, a Sanofi company
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	12
Start date	15 June 2020
Primary completion	6 January 2022
Estimated completion	6 January 2022
Sites	10 locations across Italy, Netherlands, United Kingdom, Germany, Norway, United States

Drugs / interventions tested

• BIVV020 — full drug profile →

Conditions studied

• Autoimmune Haemolytic Anaemia — all drugs for Autoimmune Haemolytic Anaemia →

Sponsor

Bioverativ, a Sanofi company — full company profile →

Who can join

18 and older, any sex, with Autoimmune Haemolytic Anaemia. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Primary Objective:

To assess the safety and tolerability in participants with cold agglutinin disease (CAD), after a single dose of intravenous (IV) BIVV020

Secondary Objectives:

To assess, in participants with cold agglutinin disease, after a single dose of intravenous (IV) BIVV020:

* The effect of BIVV020 on complement mediated hemolysis
* The pharmacodynamics (PD) of BIVV020 relating to complement inhibition
* The pharmacokinetics (PK) of BIVV020
* The immunogenicity of BIVV020

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Targeting the complement system in neuromyelitis optica spectrum disorder.
   Asavapanumas N, Tradtrantip L, Verkman AS. · · 2021 · cited 64× · PMID 33513036 · DOI 10.1080/14712598.2021.1884223
2. The choice of new treatments in autoimmune hemolytic anemia: how to pick from the basket?
   Berentsen S, Fattizzo B, Barcellini W. · · 2023 · cited 29× · PMID 37168855 · DOI 10.3389/fimmu.2023.1180509
3. Daratumumab monotherapy in refractory warm autoimmune hemolytic anemia and cold agglutinin disease.
   Jalink M, Jacobs CF, Khwaja J, Evers D, et al · · 2024 · cited 27× · PMID 38507742 · DOI 10.1182/bloodadvances.2024012585
4. Complement C1s as a diagnostic marker and therapeutic target: Progress and propective.
   Ye J, Yang P, Yang Y, Xia S. · · 2022 · cited 25× · PMID 36275687 · DOI 10.3389/fimmu.2022.1015128
5. Safety, tolerability, and activity of the active C1s antibody riliprubart in cold agglutinin disease: a phase 1b study.
   D'Sa S, Vos JMI, Barcellini W, Wardęcki M, et al · · 2024 · cited 13× · PMID 38085846 · DOI 10.1182/blood.2023022153
6. Halting targeted and collateral damage to red blood cells by the complement system.
   Jalink M, de Boer ECW, Evers D, Havinga MQ, et al · · 2021 · cited 13× · PMID 34191092 · DOI 10.1007/s00281-021-00859-8
7. Beneath the surface in autoimmune hemolytic anemia: pathogenetic networks, therapeutic advancements and open questions.
   Costa A, Mulas O, Mereu AM, Schintu M, et al · · 2025 · cited 2× · PMID 40821796 · DOI 10.3389/fimmu.2025.1624667
8. Confirmation of Fixed Quarterly Riliprubart Regimen in Patients with Cold Agglutinin Disease Using Population PK/PD and Exposure-Response Analyses.
   Chow T, Wardecki M, Storek M, Wong N. · · 2025 · PMID 40308078 · DOI 10.1002/cpt.3692