CAD cold agglutinin disease
Conditions
Interventions
All participants will receive 3.5 g SAR445088 IV infusions starting day 1, day
29 and week 12. Then every 12 weeks.
Sponsors
Sanofi BV
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: * Male and female adult patients >=18 years of age with CAD who were previously treated with SAR445088 and met the below criteria for entry into Part 1: - met the eligibility criteria of a previous study evaluating SAR445088; - successfully enrolled and completed dosing in a previous study evaluating SAR445088; - successfully completed end of study procedures in a previous study evaluating SAR445088; and - per Investigator judgement, had a favorable benefit to risk profile after receiving SAR445088. OR were never treated with SAR445088 before entering Part 2, and met the below criteria to establish CAD diagnosis: • chronic hemolysis; • polyspecific direct antiglobulin test (DAT) positive status; • monospecific DAT strongly positive for C3d; • cold agglutinin [CAg] titer >=64 at 4°C; • IgG DAT
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 within one month of enrollment. * Clinical diagnosis of systemic lupus erythematosus (SLE). * 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 other than SAR445088 (eg, eculizumab) within three months prior to screening. * Concurrent treatment with systemic corticosteroids other than a stable daily dose equivalent to
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| - Number of participants with treatment-emergent adverse events (TEAE) | — |
Secondary
| Measure | Time frame |
|---|---|
| - Mean change from baseline in total bilirubin over time - Mean change from baseline in hemoglobin over time - Mean change from baseline in lactate dehydrogenase over time - Mean change from baseline in reticulocyte count over time - Complement System Classical Pathway Levels as Measured by WIESLAB Assay - Complement System Alternative Pathway Levels as Measured by WIESLAB Assay - Mean change in CH50 over time - Total Complement Factor C4 Levels - PK parameters Cmax, AUC - Number of participants with anti-SAR445088 antibodies | — |
Countries
Netherlands
Outcome results
None listed