Active not recruitingPHASE2INTERVENTIONAL

A Study of Ianalumab (VAY736) in Patients With Primary Immune Thrombocytopenia (ITP) Previously Treated With at Least Two Lines of Therapies

This research study is testing a new medication called ianalumab for adults who have a blood condition known as Primary Immune Thrombocytopenia (ITP). ITP causes low platelet counts, which help your blood clot. We are particularly interested in people whose ITP hasn't improved enough with at least two other types of treatment, including a steroid and a TPO-RA. The main goals are to find out if ianalumab is effective in raising platelet counts, how safe it is, and if people can take it without too many side effects. Participants will receive the medication, and their health will be closely monitored for around two years to understand the long-term effects.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Novartis Pharmaceuticals

Enrolment target

Start

16 Aug 2023

Estimated completion

19 Mar 2029

Primary Immune Thrombocytopenia (ITP)

What is this study about?

This study is for adults who have a condition called Primary Immune Thrombocytopenia, or ITP for short. ITP is a blood disorder where your body's immune system mistakenly attacks and destroys its own platelets. Platelets are tiny blood cells that help your blood clot, so low platelet counts can lead to bruising and bleeding problems.

You might be interested in this study if you've already tried at least two other types of ITP treatments, such as certain steroids and a medication called a TPO-RA, but your platelet counts are still low (under 30 G/L) and your doctor thinks you need more treatment. The medication being tested is called ianalumab. We want to understand if it can help improve platelet counts, if it's safe to use, and if people find it easy to tolerate.

This is a 'Phase 2' study, which means we're evaluating the medication in a small group of people to see if it works and to get more information about its safety before potentially moving to larger studies. Understanding if ianalumab can help people who haven't responded well to other treatments is very important for finding new ways to manage ITP.

Key takeaways

This study evaluates ianalumab for adults with ITP.
It's for those whose ITP hasn't responded to at least two prior treatments.
The study will check if ianalumab is effective and safe.
Participants will be closely monitored for around two years.
You can withdraw from the study at any time.
Talk to your doctor if you think this study might be right for you.

Who may be eligible?

You might be able to join this study if you are 18 years old or older and have been diagnosed with primary ITP. You must have already tried at least two different treatments for your ITP, including a steroid and a TPO-RA, and your platelet count is still low (below 30 G/L) and your doctor believes you need treatment. You will need to agree to take part by signing an informed consent form.

There are also some reasons why you might not be able to join. For example, if your low platelet count is due to another medical condition (secondary thrombocytopenia) or if you've had certain treatments very recently, like a blood transfusion or some types of strong immunosuppressant medicines. Also, if you've had your spleen removed, you won't be able to participate. Your medical team will check all these details carefully.

Quick self-check

Are you 18 years old or older?
Have you been diagnosed with Primary ITP?
Have you already tried at least two ITP treatments (like a steroid and a TPO-RA)?
Is your platelet count currently low (below 30 G/L) and your doctor thinks you need treatment?
Have you NOT had your spleen removed?
Have you NOT had treatments like blood transfusions or certain strong immunosuppressants very recently?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part, the study will start with a screening period of up to two weeks. During this time, doctors will do tests to make sure you're a good fit for the study. If you qualify, you will receive the study medicine, ianalumab, at the same dose as other participants.

After you start the medicine, you'll have regular check-ups to see how well the treatment is working and to monitor for any side effects. These check-ups will continue for a main assessment period. After this, if your platelet count is stable and you're not needing other ITP treatments, your health will continue to be monitored for safety and effectiveness. Some people who initially respond well but later see their platelet counts drop might be offered a second course of ianalumab. The study will follow all participants for up to two years after their last dose of ianalumab, meaning the total time in the study could be around two years or more.

Potential risks and benefits

Participating in a study like this could potentially offer you access to a new treatment (ianalumab) that isn't widely available yet, which might help improve your platelet count if other treatments haven't been effective. However, it's also possible that the treatment may not work for you, or you might experience side effects, some of which could be serious or unexpected. Your health will be closely monitored by medical staff throughout the study. Remember, joining is completely voluntary, and you are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (24)

Massachusetts General Hospital
Boston, United States
Beth Israel Deaconess Med Center
Boston, United States
Novartis Investigative Site
CABA, Argentina
Novartis Investigative Site
Garran, Australia
Novartis Investigative Site
Melbourne, Australia
Novartis Investigative Site
Wuhan, China
Novartis Investigative Site
Beijing, China
Novartis Investigative Site
Jinan, China
Novartis Investigative Site
Toulouse, France
Novartis Investigative Site
Dresden, Germany
Novartis Investigative Site
Jena, Germany
Novartis Investigative Site
Giessen, Germany

+12 more sites — see the official record for the full list.

Common questions

What is ITP?

ITP stands for Primary Immune Thrombocytopenia. It's a condition where your body's immune system attacks and destroys its own platelets, leading to a low platelet count and a higher risk of bleeding.

What is ianalumab?

Ianalumab is the new medicine being tested in this study. It's hoped to help increase platelet counts in people with ITP, especially those who haven't responded well to other treatments.

What does 'Phase 2 study' mean?

A Phase 2 study means that we are testing the new medicine in a small group of people to see if it works and to gather more information on its safety, before potentially moving on to larger studies.

How long will I be in the study?

You could be in the study for up to two years or possibly longer, depending on whether you receive a second course of treatment and when you had your last dose of ianalumab.

Can I stop participating if I want to?

Yes, absolutely. Taking part is entirely your choice, and you can withdraw from the study at any time without having to give a reason, and it won't affect your future medical care.