CAD cold agglutinin disease
Conditions
Interventions
Dosing will begin with a single dose of 30 mg/kg IV. Dosing will not exceed 50
mg/kg or the highest tolerated dose tested in normal healthy volunteers in any
cohort.
Sponsors
Genzyme Europe BV
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: - Male and/or female patients, * 18 years of age with cold agglutinin disease as defined by: a) Chronic hemolysis per Investigator*s judgement, b) Polyspecific direct antiglobulin test (DAT) positive, c) Monospecific DAT strongly positive for C3d, d) Cold agglutinin (CAg) titer * 64 at 4°C; and, e) 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
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety * Assessment of adverse events (AE)/treatment-emergent adverse events (TEAE). * Clinical laboratory evaluations including hematology, biochemistry, systemic lupus erythematosus (SLE) panel testing, and urinalysis. * Electrocardiographic (ECG) intervals (heart rate, PR, QRS, QT, and QTcF). * Vital signs (blood pressure and heart rate). | — |
Secondary
| Measure | Time frame |
|---|---|
| Hematologic BA * Total bilirubin. * Hemoglobin. Pharmacodynamics * CP. * Complement alternative pathway (AP). * CH50. * Total C4. Pharmacokinetics * Parameters (not limited to): Cmax, tmax, tlast, AUClast, AUC0-*, t1/2z, CL, Vd. Immunogenicity * Anti-BIVV020 antibodies (ADA). | — |
Countries
Netherlands
Outcome results
None listed