Primary Objective:
To assess the long-term safety and tolerability in patients with cold agglutinin disease (CAD), after multiple doses of SAR445088
Secondary Objective:
To assess, in patients with cold agglutinin disease (CAD), after multiple doses of SAR445088:
* The long-term effect of SAR445088 on complement mediated hemolysis
* The long-term pharmacodynamics (PD) effect of SAR445088 relating to complement inhibition
* The long-term pharmacokinetic (PK) profile of SAR445088
* The long-term immunogenicity of SAR445088
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Male and female adult patients ≥18 years of age with CAD who were previously treated with SAR445088 and met criteria 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 (vaccinations have to be initiated at least 14 days prior to dosing and completed before Week 5 of Part 2).
* 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.
Exclusion Criteria:
* Cold…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of participants with treatment-emergent adverse events (TEAE)
Timeframe: Day 1 or Day 1-IV to end of study, approximately 6 years