RecruitingPHASE2INTERVENTIONAL

Study of Ianalumab in Adults With Primary Immune Thrombocytopenia (ITP) and Warm-antibody Autoimmune Hemolytic Anemia (wAIHA) Who Have Previously Benefited From Ianalumab

This research study is for adults who have either Primary Immune Thrombocytopenia (ITP) or warm autoimmune hemolytic anemia (wAIHA). These are conditions where your immune system mistakenly attacks your blood cells. We're looking at people who previously took part in an ianalumab study and saw good results, but then their condition got worse again, or the treatment didn't fully work long-term. The main goal is to find out if giving a second course of ianalumab is safe and effective for these patients. Participants will receive the same dose of ianalumab that helped them before. The study will monitor their health and condition over time to see how they respond and to check for any side effects. It's an exploration to understand if retreatment offers a good option.

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

Novartis Pharmaceuticals

Enrolment target

Start

26 Jun 2026

Estimated completion

30 Aug 2030

Primary Immune Thrombocytopenia Warm Autoimmune Hemolytic Anemia

What is this study about?

This study is about a medication called ianalumab, specifically for adults who have one of two blood conditions: Primary Immune Thrombocytopenia (ITP) or warm autoimmune hemolytic anemia (wAIHA). Both of these conditions involve your body's immune system mistakenly attacking and destroying important blood cells – platelets in ITP and red blood cells in wAIHA. This can lead to issues like easy bruising, bleeding, or anaemia (low red blood cells) which causes tiredness and weakness.

We're inviting people who have previously taken part in an ianalumab study for their ITP or wAIHA. In those earlier studies, ianalumab helped them, but either the good effects didn't last as long as hoped, or their condition became problematic again. This new study wants to see if giving a second course of ianalumab could be a safe and helpful option for these individuals, potentially bringing their condition back under control.

Essentially, the study aims to understand if a ‘top-up’ or ‘re-treatment’ with ianalumab can provide benefits again for those who previously responded well to it. By observing how people react to this second course, researchers hope to learn more about how long the benefits can last and if it's a good treatment strategy for relapses or when initial treatment effects wear off.

Key takeaways

This study is for adults with specific blood conditions: ITP or wAIHA.
It's for people who previously benefited from ianalumab in an earlier study.
The study explores if a second course of ianalumab is safe and effective.
All participants will receive the active study drug, ianalumab.
Regular health monitoring will be part of the study.

Who may be eligible?

To join this study, you must be an adult aged 18 or older. A key requirement is that you must have been part of an earlier ianalumab study for either Primary Immune Thrombocytopenia (ITP) or warm autoimmune hemolytic anemia (wAIHA).

If you have ITP, you would have previously received ianalumab (or a placebo) in one of the specific earlier studies (CVAY736I12301 or CVAY736Q12301). You would also need to have seen good results from that treatment, but then your condition worsened again at least two years after your last dose in the original study.

If you have wAIHA, you would also have participated in a specific previous ianalumab study (CVAY736O12301) and had a lasting positive response. However, your wAIHA must have come back, and your haemoglobin (red blood cell count) needs to be within a certain low range, and you might be feeling symptoms related to anaemia. Some temporary treatments might be allowed shortly before joining the study to manage your symptoms.

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?
Have you been diagnosed with Primary Immune Thrombocytopenia (ITP) or warm autoimmune hemolytic anemia (wAIHA)?
Did you previously take part in an ianalumab study for your ITP or wAIHA?
Did that ianalumab treatment help you, but your condition has now worsened again?
For wAIHA, is your red blood cell count (haemoglobin) currently low and causing symptoms?

Answer every question to see your result.

What does participation involve?

If you join this study, you will receive a second course of the study medicine, ianalumab. The dose you receive will be the same dose that you benefited from in the earlier ianalumab study.

You will have several check-ups and assessments during the study period. If your treatment is working well, you'll continue to be monitored for both safety and how well the treatment is working. If, during the study, the treatment doesn't seem to be working for you, you will continue to be monitored for safety but might not need as many follow-up visits related to tracking the treatment's effectiveness.

The study will continue until all participants have completed their safety and/or effectiveness follow-up visits after their last dose of ianalumab, or if they decide to leave the study earlier. The exact total duration will vary depending on your individual response and follow-up needs.

Potential risks and benefits

Participating in this study might offer a potential benefit if a second course of ianalumab helps to improve or control your ITP or wAIHA again, especially if your condition has worsened after an initial good response. However, like all medications, ianalumab can have side effects, and some are not yet fully known, so there are potential risks. The research team will carefully monitor you for any side effects and your overall health. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (18)

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

Novartis Investigative Site
Verified postcode
Roeselare, Belgium· Recruiting
Novartis Investigative Site
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Guangzhou, China· Recruiting
Novartis Investigative Site
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Tianjin, China· Recruiting
Novartis Investigative Site
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Ostrava, Czechia· Recruiting
Novartis Investigative Site
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Caen, France· Recruiting
Novartis Investigative Site
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Jena, Germany· Recruiting
Novartis Investigative Site
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Debrecen, Hungary· Recruiting
Novartis Investigative Site
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Florence, Italy· Recruiting
Novartis Investigative Site
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Roma, Italy· Recruiting
Novartis Investigative Site
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Trieste, Italy· Recruiting
Novartis Investigative Site
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Vicenza, Italy· Recruiting
Novartis Investigative Site
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George Town, Malaysia· Recruiting

Common questions

What is Ianalumab?

Ianalumab is a medication being studied for conditions where your immune system attacks your own body, like ITP and wAIHA.

Why is this study being done?

This study aims to see if a second course of ianalumab is safe and effective for people whose ITP or wAIHA improved with it before, but then got worse again.

Will I get a placebo this time?

No, in this study, all participants will receive ianalumab, not a placebo.

How long will I be in the study?

The study length will vary for each person, depending on how they respond and how long their safety and effectiveness check-ups are needed after their last dose.

Can I still take other medicines while in the study?

Some 'rescue' or 'bridging' medications might be allowed before or during screening, but this needs to be discussed with the study team.

How to find out more

Novartis Pharmaceuticals

novartis.email@novartis.com 1-888-669-6682 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.