A Phase 1b, Open-Label, Single-Arm Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Clinical Effect of ANX1502 in Participants with Primary Cold Agglutinin Disease
This study is testing a new medicine called ANX1502 for people who have a rare blood condition called Primary Cold Agglutinin Disease (CAD). CAD is a condition where your immune system mistakenly attacks and destroys your red blood cells, especially when you're cold. This can make you feel very tired and unwell. This study is an early-stage trial, meaning it's one of the first times this medicine is being tested in people. The main goals are to check if ANX1502 is safe, to understand how it works in the body, and to see if it has any positive effects on the condition. Everyone in the study will receive the new medicine.
At a glance
What is this study about?
This research is about a new medicine called ANX1502 for a health problem called Primary Cold Agglutinin Disease, often shortened to CAD. CAD is a type of anaemia, which means your body doesn't have enough healthy red blood cells. In CAD, your immune system (your body's defence system) mistakenly attacks and damages your red blood cells, especially when you get cold. This can lead to symptoms like feeling very tired, weak, and sometimes having yellow skin or eyes.
The main purpose of this study is to learn more about ANX1502. Because it's a new medicine, the researchers want to firstly make sure it's safe for people to take. They also want to understand how the body uses the medicine, meaning how it's absorbed, how it works, and how it leaves the body. Finally, they will be looking to see if ANX1502 has any helpful effects on the symptoms of CAD or on the disease itself.
This is an "open-label, single-arm" study. "Open-label" means both you and the study team will know that you are receiving the new medicine. "Single-arm" means that everyone taking part will be given ANX1502; there isn't a group receiving a different treatment or a placebo (a dummy medicine) for comparison. This type of study is common in the early stages of testing a new medicine.
Key takeaways
- This study is testing a new medicine (ANX1502) for Primary Cold Agglutinin Disease (CAD).
- It's an early-stage study, mainly checking if the medicine is safe and how it works.
- All participants will receive the new medicine.
- It's for adults aged 18 and over with diagnosed CAD.
- Participation involves regular clinic visits and tests.
- There are potential benefits and risks; you can withdraw anytime.
Who may be eligible?
To be able to join this study, participants must be at least 18 years old. People of any gender can take part.
It's important to have a confirmed diagnosis of Primary Cold Agglutinin Disease (CAD) to be considered for this study. The study team will check your medical records and perform some tests to make sure you meet all the specific health requirements for taking part.
There might be other health conditions or medicines that would prevent you from joining, as the researchers need to ensure it's safe for everyone involved. The study doctor will discuss all these details with you.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you 18 years old or older?
- Do you have a diagnosis of Primary Cold Agglutinin Disease (CAD)?
- Are you able to attend regular clinic appointments?
- Are you willing to take the new study medicine as directed?
What does participation involve?
If you decide to take part in this study, you will receive the new medicine called ANX1502. The study team will ask you to visit the clinic regularly. At these visits, doctors and nurses will carry out various checks and tests. These might include blood tests to see how the medicine is affecting your body and the illness, and to check your general health. They will also ask you how you are feeling and if you have any side effects.
You will be involved in the study for a certain period, and there will be follow-up visits even after you finish taking the medicine. The total length of your participation, including all visits and follow-ups, will be clearly explained to you by the study team. This will allow them to monitor your health and the effects of the medicine over time.
Potential risks and benefits
Locations (1)
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Common questions
What is Primary Cold Agglutinin Disease (CAD)?
CAD is a rare condition where your immune system attacks your own red blood cells, especially when you are cold. This can lead to anaemia, making you feel very tired.
What is ANX1502?
ANX1502 is a new medicine being developed that researchers hope will help treat Primary Cold Agglutinin Disease. It's still in the early stages of testing.
Will I get a placebo (dummy medicine) in this study?
No, in this study, everyone who takes part will receive the new medicine, ANX1502. There isn't a group getting a placebo.
How long will I need to be in the study?
The exact duration of your participation, including how long you take the medicine and follow-up visits, will be explained by the study team. It will involve regular visits over a period of time.
Can I leave the study if I change my mind?
Yes, you can choose to leave the study at any time, for any reason. Your decision will not affect your future medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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