Long-Term Safety Study of Riliprubart in Patients with Cold Agglutinin Disease

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What is this study about?

This clinical trial is focused on studying a condition known as Cold Agglutinin Disease (CAD), which is a type of autoimmune hemolytic anemia. In this condition, the body’s immune system mistakenly attacks and destroys its own red blood cells, leading to anemia. The study is investigating a treatment called riliprubart, also known by its code name SAR445088. This treatment is administered as a solution for injection or infusion.

The purpose of the study is to assess the long-term safety and tolerability of riliprubart in individuals with CAD. Participants in the study may have been previously treated with riliprubart or may be receiving it for the first time. The study will involve multiple doses of the medication, and participants will be monitored over an extended period to observe any side effects or changes in their condition.

Throughout the study, researchers will keep track of any new health issues that arise in participants, known as treatment-emergent adverse events. They will also measure changes in various blood components, such as bilirubin and hemoglobin levels, to understand how the treatment affects the body. The study aims to provide valuable information on the long-term use of riliprubart for managing Cold Agglutinin Disease.

1 joining the study

Upon joining the study, participants are required to provide written informed consent. This is a formal agreement to participate in the study after understanding its nature and potential risks.

2 initial assessment

Participants undergo an initial assessment to confirm eligibility. This includes verifying a diagnosis of cold agglutinin disease (CAD) and ensuring that all necessary vaccinations against encapsulated bacterial pathogens have been administered at least 14 days before starting the treatment.

3 treatment administration

Participants receive the medication riliprubart as a solution for injection. The frequency and dosage are determined by the study protocol and the healthcare provider overseeing the treatment.

4 monitoring and follow-up

Participants are monitored for any treatment-emergent adverse events (TEAE). Regular follow-up visits are scheduled to assess the safety and tolerability of the treatment.

During these visits, various health parameters are measured, including changes in bilirubin, hemoglobin, lactate dehydrogenase, and reticulocyte count. The levels of the complement system pathways are also evaluated.

5 completion of study

The study is estimated to conclude by September 18, 2028. Participants will have a final assessment to evaluate the long-term effects of the treatment and any potential development of anti-SAR445088 antibodies.

Who Can Join the Study?

Must be an adult, meaning 18 years or older.
Must have a condition called cold agglutinin disease (CAD).
If previously treated with the study drug SAR445088, must have completed a previous study with this drug and have a positive assessment from the doctor.
If never treated with SAR445088, must meet certain criteria to confirm CAD diagnosis, such as:
- Chronic hemolysis: Ongoing breakdown of red blood cells.
- Polyspecific direct antiglobulin test (DAT) positive: A test showing antibodies attached to red blood cells.
- Monospecific DAT strongly positive for C3d: A specific test result indicating a certain type of antibody.
- Cold agglutinin titer ≥64 at 4°C: A measure of antibodies that react at cold temperatures.
- IgG DAT ≤1+: A specific test result for another type of antibody.
- Hemoglobin level ≤10 g/dL: A measure of red blood cells that is lower than normal.
- Elevated bilirubin not due to liver disease: Higher levels of a substance in the blood, not caused by liver problems.
Must have received certain vaccinations within the last five years and at least 14 days before starting the study.
Must agree to use double contraception methods if sexually active, and females must not be pregnant or breastfeeding.
Males must agree not to donate sperm during the study.
Must provide written informed consent before participating in any study-related activities.

Who Cannot Join the Study?

Patients with autoimmune haemolytic anaemia cannot participate. This is a condition where the body’s immune system mistakenly attacks and destroys its own red blood cells.
Patients who do not have cold agglutinin disease (CAD) are excluded. CAD is a rare type of anaemia where the immune system attacks red blood cells at low temperatures.
Patients outside the specified age range cannot participate. The study is for adults, typically aged 18 and older.
Both male and female patients are eligible, but those who do not meet other criteria will be excluded.
Patients who are considered part of a vulnerable population may be excluded. This can include groups like children, pregnant women, or those unable to give consent.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

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Other Sites

Site Name	City	Country
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico	Milan	Italy
Utbbwxnqicbouivduljim Ehuzg Aii	Essen	Germany
Auqmthayx Ucr	Amsterdam	The Netherlands

Trial status

Country	Status	Recruitment Start
Germany	Not recruiting	22.03.2021
Italy	Not recruiting	22.03.2021
The Netherlands	Not recruiting	22.03.2021

Trial locations

Investigated drugs:

Riliprubart

SAR445088 is a medication being studied for its safety and tolerability in patients with cold agglutinin disease (CAD). This trial involves participants who have either been previously treated with SAR445088 or have never received this treatment before. The main goal is to understand how well patients can tolerate the medication over a long period and to ensure it is safe for use in managing CAD.

Investigated diseases:

Autoimmune haemolytic anaemia

Autoimmune Hemolytic Anemia – This is a condition where the immune system mistakenly attacks and destroys the body’s own red blood cells, leading to a shortage of these cells. The disease can cause symptoms such as fatigue, shortness of breath, and pale or yellow skin. It progresses as the immune system continues to target red blood cells, causing them to break down faster than they can be produced. This results in anemia, which is a deficiency of red blood cells or hemoglobin in the blood. The severity and progression can vary, with some individuals experiencing mild symptoms and others having more severe manifestations. The condition can be chronic or occur in episodes, with periods of remission and flare-ups.

Trial ID:

2023-510210-68-00

Protocol code:

LTS16637

Trial Phase:

Human Pharmacology (Phase I) – Other

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