An open label, long term, safety and tolerability study of patients with cold agglutinin disease previously treated with SAR445088 or never treated with SAR445088
- Conditions
- CADcold agglutinin disease10003816
- Registration Number
- NL-OMON54300
- Lead Sponsor
- Sanofi BV
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Not specified
- Target Recruitment
- 2
* 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 <=1+;
• hemoglobin level <=10 g/dL;
• elevated bilirubin not attributable to liver disease;
* Documented vaccinations against encapsulated bacterial pathogens given within
five years of
enrollment and at least 14 days prior to dosing
* Contraception (with double contraception methods) for male and female
participants;
not pregnant or breastfeeding for female participants; no sperm donation for
male
participants.
* Having given written informed consent prior to undertaking any study-related
procedure.
* 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 <=10
mg/day prednisone within three months prior to screening.
* History of hypersensitivity to SAR445088 or any of its components.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>- Number of participants with treatment-emergent adverse events (TEAE)</p><br>
- Secondary Outcome Measures
Name Time Method <p>- Mean change from baseline in total bilirubin over time<br /><br>- Mean change from baseline in hemoglobin over time<br /><br>- Mean change from baseline in lactate dehydrogenase over time<br /><br>- Mean change from baseline in reticulocyte count over time<br /><br>- Complement System Classical Pathway Levels as Measured by WIESLAB Assay<br /><br>- Complement System Alternative Pathway Levels as Measured by WIESLAB Assay<br /><br>- Mean change in CH50 over time<br /><br>- Total Complement Factor C4 Levels<br /><br>- PK parameters Cmax, AUC<br /><br>- Number of participants with anti-SAR445088 antibodies</p><br>