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RecruitingPHASE3INTERVENTIONAL

Study to Evaluate the Efficacy and Safety of Oral Rilzabrutinib in Adults With Immune Thrombocytopenia (ITP) Who Failed First-line Treatment

This study is exploring a new oral medicine, rilzabrutinib, for adults living with Immune Thrombocytopenia (ITP). ITP is a condition where your body doesn't have enough platelets, which help your blood clot. This trial is for people whose initial treatment for ITP didn't work as hoped. We want to understand if rilzabrutinib can effectively help manage ITP symptoms and if it’s safe to use. This is a Phase 3 study, which means it’s a later-stage trial designed to confirm the medicine's benefits and safety in a larger group of people before it can become widely available. The study involves a few stages, including an initial assessment, a main treatment period, and follow-up.

At a glance

Status
Recruiting
Phase
PHASE3
Sponsor
Sanofi
Enrolment target
60
Start
24 Oct 2025
Estimated completion
30 Dec 2028

What is this study about?

This research study is looking into a new oral medicine called rilzabrutinib. It's designed for adult patients who have Immune Thrombocytopenia (ITP). ITP is a condition where your immune system mistakenly attacks and destroys your platelets, which are tiny cells in your blood that help it to clot. This can lead to bruising, bleeding, and feeling tired.

Many people with ITP start with a treatment, but sometimes, for various reasons, that first treatment doesn't work well enough, or its effects don't last. This study focuses on these individuals, aiming to see if rilzabrutinib can be an effective and safe option when initial treatments haven't been successful.

The study is in 'Phase 3,' which means it's a critical stage of testing. Researchers are carefully observing a larger group of people to confirm how well the medicine works, what its side effects might be, and ultimately, if it could be a helpful new treatment option for ITP patients in the future.

Key takeaways

  • This study is testing a new oral medicine (rilzabrutinib) for adults with ITP.
  • It's for people whose first ITP treatment wasn't successful.
  • The study is in its final main testing stage (Phase 3) to check effectiveness and safety.
  • Participation involves screening, a treatment period, and follow-up appointments.
  • You can stop participating at any time if you change your mind.

Who may be eligible?

This study is looking for adult men and women, aged 18 and over, who have been diagnosed with a specific type of ITP called 'primary ITP.' You might be a good fit if you've already tried at least one initial treatment for your ITP, and while that treatment might have helped at first, it's no longer working as well, or you've had to continue taking steroids to manage your condition.

However, there are certain situations that would mean you couldn't join this study. For example, if your ITP is caused by another underlying health problem (we call this 'secondary ITP'), or if you've had certain other conditions like some types of cancer in recent years, or if you've had an organ transplant. People who have already had advanced treatments for ITP or had their spleen removed for ITP reasons would also not be able to participate.

Also, if you are pregnant or breastfeeding, this study would not be suitable for 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.

  1. Are you 18 years old or older?
  2. Do you have a diagnosis of primary ITP?
  3. Has your first ITP treatment not worked as well as you hoped, or are you still relying on steroids?
  4. Have you not had an organ transplant or certain types of cancer in the last 5 years?
  5. Are you not pregnant or breastfeeding?
Answer every question to see your result.

What does participation involve?

If you join this study, your journey would typically begin with a 'screening period' lasting up to 8 weeks. During this time, the study team will carry out various tests and checks to make sure the study is right for you. If you are eligible, you would then enter the 'primary analysis period,' which lasts for up to 28 weeks. During this time, you would take the study medicine, rilzabrutinib, as directed, and attend regular appointments for assessments and to monitor your health. Some participants might then be invited to continue in a longer 'extension period' for another 28 weeks. After finishing the treatment part of the study, there will be a 'follow-up period' for about 24 weeks, where the study team will check on your health and well-being. The total length of your involvement could vary depending on which parts of the study you participate in.

Potential risks and benefits

Taking part in a study like this might offer a potential benefit by giving you access to a new medicine that could help manage your ITP, especially if your current treatments aren't working well. However, like all medicines, rilzabrutinib might have side effects, and some might not be known yet. The study team will carefully monitor you for any potential issues. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (30)

  • Community Health Partners (CHP) - Community Medical Oncology Specialists (University Oncology Associates)
    Verified postcode
    Clovis, United States· Recruiting
  • Tulane University Health Sciences Center - Tulane Avenue-Investigational Site Number: 8400011
    Verified postcode
    New Orleans, United States· Recruiting
  • Massachusetts General Hospital Cancer Center
    Verified postcode
    Boston, United States· Recruiting
  • Beth Israel Deaconess Medical Center
    Verified postcode
    Boston, United States· Recruiting
  • University of Michigan Health - Michigan Medicine - University Hospital-Investigational Site Number: 8400001
    Verified postcode
    Ann Arbor, United States· Recruiting
  • Mayo Clinic_Investigational Site Number: 8400009
    Verified postcode
    Rochester, United States· Recruiting
  • New York Oncology Hematology-Investigational Site Number: 8400010
    Verified postcode
    Albany, United States· Recruiting
  • Montefiore Medical Center-Investigational Site Number: 8400012
    Verified postcode
    The Bronx, United States· Recruiting
  • Community Cancer Trials of Utah-Investigational Site Number: 8400002
    Verified postcode
    Ogden, United States· Recruiting
  • Investigational Site Number : 0400001
    Verified postcode
    Vienna, Austria· Recruiting
  • Investigational Site Number: 2500003
    Verified postcode
    Créteil, France· Recruiting
  • Investigational Site Number : 2500001
    Verified postcode
    Dijon, France· Recruiting

Common questions

What is ITP?

ITP stands for Immune Thrombocytopenia. It's a blood condition where your body doesn't have enough platelets, which are tiny cells that help your blood clot.

What does 'first-line treatment failed' mean?

It means the initial treatment you received for your ITP didn't work as well as hoped, or its benefits didn't last, or you still need to take steroid medicines to control your condition.

What is rilzabrutinib?

Rilzabrutinib is the name of the new oral (taken by mouth) medicine being tested in this study for ITP.

What is a 'Phase 3' study?

Phase 3 is an important stage of testing a new medicine. It means the medicine has already shown some promise, and now researchers want to confirm its benefits and safety in a larger group of people.

Will I have to pay to be in the study?

No, you will not have to pay to take part in this study or for the study medicine.

How to find out more

Trial Transparency email recommended (Toll free for US & Canada)

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

Interested in taking part?

Register your interest

Share your details and the research team for "Study to Evaluate the Efficacy and Safety of Oral Rilzabruti…" will contact you if you may be eligible. Always speak to your GP before agreeing to take part.

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