Active not recruitingPHASE3INTERVENTIONAL

A Study of Efficacy and Safety of Ianalumab in Previously Treated Patients With Warm Autoimmune Hemolytic Anemia

This research study is testing a new medicine called ianalumab for adults with a blood condition called warm autoimmune hemolytic anemia (wAIHA). This condition makes your body destroy its own red blood cells. We are looking for people who have already tried at least one other treatment for wAIHA that didn't work well enough. The study wants to find out if ianalumab is better than a dummy medicine (placebo) at increasing healthy red blood cells over time. It also aims to check if the medicine is safe. Participants will receive either ianalumab or a placebo, and the study will follow them closely to see how the treatment affects their health.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Novartis Pharmaceuticals

Enrolment target

Start

30 Dec 2022

Estimated completion

19 Dec 2028

Warm Autoimmune Hemolytic Anemia (wAIHA)

What is this study about?

This study is about a condition called warm autoimmune hemolytic anemia, or wAIHA for short. In wAIHA, your body's immune system, which usually fights off germs, mistakenly attacks and destroys your own red blood cells. Red blood cells are really important because they carry oxygen around your body. When they're destroyed too quickly, you can become anemic, which can make you feel tired, weak, and short of breath.

Doctors are testing a new medicine called ianalumab. They want to see if this medicine can help stop the immune system from attacking red blood cells, leading to more healthy red blood cells in your body. This particular study is for people with wAIHA who have already tried other treatments that haven't worked as well as hoped. By comparing ianalumab to a placebo (a dummy medicine), researchers can understand if ianalumab is truly effective and what its potential side effects might be.

The main goal is to see if ianalumab can help your body maintain a healthy level of red blood cells for a long time. The study will also carefully check for any side effects of the medicine. If ianalumab proves to be safe and effective, it could become a new treatment option for people living with wAIHA.

Key takeaways

Tests a new medicine, ianalumab, for wAIHA.
For wAIHA patients who haven't responded to other treatments.
Compares ianalumab to a placebo (dummy medicine).
Aims to increase red blood cells and check safety.
Involves regular clinic visits and long-term follow-up.
You can withdraw from the study at any time.

Who may be eligible?

This study is looking for adults aged 18 or older who have warm autoimmune hemolytic anemia (wAIHA). To be considered, you must have been diagnosed with wAIHA, and your red blood cells should show signs that your immune system is attacking them. Importantly, you should have already tried at least one other treatment for your wAIHA that either didn't work well enough, or the benefits didn't last, or you couldn't tolerate it. Your red blood cell levels at the start of the study also need to be within a specific range, and you should be experiencing symptoms related to your anemia.

There are also some reasons why you might not be able to join. For example, if your wAIHA is caused by certain other severe blood conditions or immune diseases that would require treatments not allowed in the study. You also cannot have other types of anemia where your body attacks its own cells, or have recently had certain strong immune-suppressing treatments. Your general health, including your kidney function and immune system, will be checked to make sure it's safe for you to participate.

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 warm autoimmune hemolytic anemia?
Have you already tried at least one other treatment for wAIHA that didn't work well enough?
Is your red blood cell level (hemoglobin) within a specific range, and are you feeling symptoms of anemia?
Do you have any other serious medical conditions that might exclude you from the study?
Have you recently received certain strong immune-suppressing medicines that aren't allowed?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will be assigned by chance (like flipping a coin) to receive one of two different doses of ianalumab, or a placebo (a dummy medicine). You won't know which one you're getting, and neither will your study doctor, during the main treatment period. If you are on the placebo and your condition doesn't improve, you might then be offered the higher dose of ianalumab.

Throughout the study, you'll have regular visits to the clinic. At first, these will be every two weeks to monitor your health and check how the treatment is working. After the main treatment period, the visits will become less frequent, starting monthly and then moving to quarterly (every three months) for up to two years. Some participants might have even longer follow-up, up to 39 months, to see how long the treatment's effects last. You will also be allowed to take other standard supportive care medicines for your wAIHA if your doctor thinks they are necessary.

Potential risks and benefits

Taking part in this study may offer potential benefits, such as receiving a new investigational treatment that could improve your red blood cell levels and reduce your anemia symptoms. However, there's no guarantee the treatment will work for everyone, and you might receive a placebo. Every medicine has potential risks, and ianalumab may cause side effects, which will be carefully monitored throughout the study. You will have regular medical check-ups to ensure your safety. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (53)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Michigan Center of Medical Research
Verified postcode
Farmington Hills, United States
University of Minnesota Med Center
Verified postcode
Minneapolis, United States
Fred Hutchinson Cancer Center
Verified postcode
Seattle, United States
Novartis Investigative Site
Unverified
CABA, Argentina
Novartis Investigative Site
Unverified
Caba, Argentina
Novartis Investigative Site
Unverified
Caba, Argentina
Novartis Investigative Site
Verified postcode
Garran, Australia
Novartis Investigative Site
Verified postcode
Melbourne, Australia
Novartis Investigative Site
Verified postcode
Guangzhou, China
Novartis Investigative Site
Verified postcode
Hangzhou, China
Novartis Investigative Site
Verified postcode
Dalian, China
Novartis Investigative Site
Verified postcode
Tianjin, China

Common questions

What is wAIHA?

Warm autoimmune hemolytic anemia (wAIHA) is a condition where your body's immune system mistakenly attacks and destroys its own red blood cells.

What is ianalumab?

Ianalumab is a new medicine being tested in this study to see if it can help stop the immune system from destroying red blood cells.

What is a placebo?

A placebo is a 'dummy' medicine that looks like the real treatment but contains no active drug. It helps researchers compare the new medicine's effects.

Will I know if I'm getting the real medicine?

For most of the study, you won't know whether you're receiving ianalumab or the placebo, and neither will your study doctor. This is called 'double-blinded'.

How long will the study last for me?

The study involves a treatment period followed by regular check-ups. Overall, your participation could last up to two years or even longer in some cases, depending on how you respond.