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

A Multicenter, Phase 1b, Open Label, Nonrandomized, Single Dose Study Evaluating the Safety, Tolerability and Activity of BIVV020 in Adults With Cold Agglutinin Disease

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

Up to 23 weeks (screening period up to 8 weeks, treatment period 15 weeks)

Inclusion criteria :

• Male and/or female patients, ≥ 18 years of age with cold agglutinin disease as defined by:

  1. Chronic hemolysis per Investigator's judgement,
  2. Polyspecific direct antiglobulin test (DAT) positive,
  3. Monospecific DAT strongly positive for C3d,
  4. Cold agglutinin (CAg) titer ≥ 64 at 4 C; and,
  5. IgG DAT ≤1+.

• A hemoglobin level ≤11 mg/dL.

• A total bilirubin level above the normal reference range that is thought to be due to hemolysis.

• Documented vaccinations against encapsulated bacterial pathogens (Neisseria meningitidis, including serogroup B meningococcus and Streptococcus pneumoniae) within five years of screening or willing to complete protocol specified vaccinations.

• Having given written informed consent prior to undertaking any study-related procedure.

Exclusion criteria:

• Cold agglutinin syndrome secondary to infection, rheumatologic disease, or known high grade hematologic malignancy, or known solid organ tumor.
• Clinically relevant infection of any kind within one month preceding screening.
• Treatment with anti-CD20 monotherapy within three months or anti CD20 combination therapies within six months prior to screening.
• Concurrent treatment with systemic immunosuppressive agents targeting B- or T-cell function and/or cytotoxic agents within 3 months prior to screening. Concurrent treatment with other systemic immunosuppressants within 5.5 half-lives of the drug prior to screening.
• Any specific complement system inhibitor within three months prior to screening.
• Concurrent treatment with systemic corticosteroids other than a stable daily dose equivalent to ≤10 mg/day prednisone within three months prior to screening.
• If female, pregnant or lactating.

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.