A Study of Efficacy and Safety of Ianalumab Versus Placebo in Addition to Eltrombopag in Primary Immune Thrombocytopenia Patients Who Failed Steroids
This research study is looking for adults with a bleeding disorder called primary Immune Thrombocytopenia (ITP). ITP causes your body to wrongly attack and destroy your platelets, which are tiny cells that help your blood clot. If your ITP hasn't gotten better after trying steroid medication, this study might be for you. The study aims to find out if adding a new drug, ianalumab, to an existing treatment called eltrombopag, can help keep your platelet count at a healthy level for a longer time. They also want to see how safe this combination of drugs is. Participants will receive either ianalumab or a placebo (a dummy drug) along with eltrombopag, and will be carefully monitored throughout the study.
At a glance
What is this study about?
Imagine your body's immune system as a protective shield. In primary Immune Thrombocytopenia (ITP), this shield mistakenly attacks your own platelets, which are crucial for stopping bleeding. When your platelet count drops too low, you can bruise easily or experience more serious bleeding problems. The usual first step to treat ITP is often steroid medication, but for many people, steroids don't work well or their ITP comes back.
This study is looking for a better way to help people whose ITP hasn't responded to steroids. It's focusing on a new drug called ianalumab, which is being tested alongside eltrombopag, a medication already approved for ITP. Eltrombopag works by encouraging your body to make more platelets. Researchers want to see if adding ianalumab can make eltrombopag even more effective at keeping platelet counts stable and preventing the need for other treatments or worsening symptoms. They also want to make sure the combination is safe.
The study is designed to be fair and unbiased. Half of the participants will receive ianalumab plus eltrombopag, and the other half will receive a placebo (dummy drug) plus eltrombopag. Neither you nor your doctor will know which treatment you are receiving, which helps ensure the results are as accurate as possible. This is a common and important way that medical research is done.
Key takeaways
- New study for adults with ITP not helped by steroids.
- Compares ianalumab with placebo, both given with eltrombopag.
- Aims to keep platelet counts stable for longer and check safety.
- Participation involves regular check-ups and blood tests.
- You won't know if you're getting ianalumab or the placebo (double-blind).
- You can stop participating at any time.
Who may be eligible?
To join this study, you need to be an adult (18 years or older) with a diagnosis of primary ITP. Your ITP must not have responded well to, or may have come back after, treatment with steroids. Your current platelet count needs to be quite low (below 30), and your doctor should agree that eltrombopag is a suitable treatment for you.
There are also some important reasons why you *wouldn't* be able to join. For example, if you've already had multiple treatments for your ITP (other than steroids), including surgery to remove your spleen, this study might not be right for you. They are focusing on people whose first-line steroid treatment hasn't worked.
Other reasons for not being able to join include having certain other serious health conditions like specific blood disorders, severe infections, active liver problems, or being pregnant or breastfeeding. The researchers want to make sure the study is safe for everyone involved and that the results are clear for those who fit the specific criteria.
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 ITP?
- Has your ITP not responded well to, or come back after, steroid treatment?
- Is your current platelet count lower than 30? (Your doctor can confirm this).
- Have you had your spleen removed or undergone other major ITP treatments (apart from steroids/IVIG)? (If yes, you might not be eligible).
- Are you pregnant or breastfeeding, or do you have any serious underlying health conditions like severe liver problems or uncontrolled infections? (If yes, you might not be eligible).
What does participation involve?
If you decide to take part, you'll first have a screening period where doctors will check if the study is right for you. Then, you'll be randomly assigned to one of two groups: one receiving ianalumab plus eltrombopag, and the other receiving a placebo (a dummy drug) plus eltrombopag. You'll take these medications for a certain period. After that, your eltrombopag dose might be slowly reduced while still being monitored.
Once the main treatment period is over, you'll enter a follow-up phase. During this time, the study team will continue to check your health, blood counts, and response to treatment. This is important to understand how the medicines worked in the long term and to ensure your safety. The total duration of your participation will vary depending on how you respond and how long the researchers need to follow your progress, but it will involve regular clinic visits to monitor your health and take blood samples.
Potential risks and benefits
Locations (77)
- Yuma Regional Medical CenterVerified postcodeYuma, United States
- University of Colorado AnschutzVerified postcodeAurora, United States
- NorthShore University Health SystemVerified postcodeEvanston, United States
- Boston Medical CenterVerified postcodeBoston, United States
- UMASS Memorial Medical CenterVerified postcodeWorcester, United States
- Michigan Center of Medical ResearchVerified postcodeFarmington Hills, United States
- St Vincent Frontier Cancer CenterVerified postcodeBillings, United States
- Hematology Oncology Association of RocklandVerified postcodeNyack, United States
- Montefiore Medical CenterVerified postcodeThe Bronx, United States
- Novartis Investigative SiteUnverifiedCABA, Argentina
- Novartis Investigative SiteVerified postcodeVienna, Austria
- Novartis Investigative SiteVerified postcodeYvoir, Belgium
Common questions
What is ITP?
ITP stands for Immune Thrombocytopenia. It's a blood condition where your body's immune system mistakenly attacks and destroys your platelets, which are important for blood clotting.
What is a 'placebo'?
A placebo is a 'dummy drug' that looks exactly like the real medication but doesn't contain any active ingredients. It's used in studies to compare the new drug's effects against having no active treatment.
Does this study mean I stop my current ITP treatment?
The study is for people whose ITP hasn't improved with steroids. You would receive eltrombopag, an existing ITP treatment, plus either the new drug (ianalumab) or a placebo. Your doctors will discuss your current treatment plan with you.
How long will I be in the study?
The exact duration can vary, as it depends on your response to treatment and the study design. It will involve a treatment period followed by a follow-up period to monitor your health and response.
Can I leave the study at any time?
Yes, you are free to withdraw from the study at any point, for any reason, without it affecting your current or 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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