RecruitingPHASE3INTERVENTIONAL

Efficacy and Safety of Subcutaneous Belimumab or Placebo in Addition of Rituximab in Persistent or Chronic Immune Thrombocytopenia

This UK clinical trial is for adults with a condition called Immune Thrombocytopenia (ITP), where the body attacks its own blood platelets. If you’ve had ITP for a few months or longer, and your platelet count (which helps blood clot) is still low despite previous treatments, this study might be for you. It's testing if adding a drug called belimumab to rituximab, a common treatment, is more effective at raising platelet levels and keeping them stable over time. Participants will receive either rituximab plus belimumab, or rituximab plus a dummy drug (placebo), given as injections. Researchers want to see if this new combination helps more people with ITP.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Assistance Publique - Hôpitaux de Paris

Enrolment target

132

Start

14 Nov 2022

Estimated completion

14 Nov 2027

Primary Immune Thrombocytopenia (ITP)

What is this study about?

This study is looking into a condition called Primary Immune Thrombocytopenia, or ITP. If you have ITP, your body's immune system mistakenly attacks your platelets, which are tiny blood cells essential for blood clotting and stopping bleeding. This can lead to bruising, bleeding, or other more serious issues. While there are treatments like steroids or other medications, for many people, ITP can be long-lasting or keep coming back.

The trial wants to find out if combining an existing treatment called rituximab with a new drug called belimumab works better than rituximab alone. Rituximab helps by targeting certain immune cells. Researchers believe that belimumab might help by stopping other immune cells that cause problems in ITP. They've had some promising early results, and this larger study aims to confirm if this combination is a real step forward for ITP treatment.

Participants will be placed into one of two groups: one group will receive rituximab plus belimumab, and the other will receive rituximab plus a placebo (a dummy drug that looks like belimumab but has no active ingredient). This is a 'Phase 3' study, which means it’s a later stage of research, testing the new treatment on a larger group of people to confirm its effectiveness and safety. This approach helps us understand if the new drug combination can offer a better or more lasting response for people living with ITP.

Key takeaways

This study investigates a new combination treatment for long-lasting ITP.
It combines an existing drug (rituximab) with a new one (belimumab) to boost platelet counts.
Participants will receive weekly injections for 24 weeks and be monitored for a year.
The study is 'double-blind', meaning you won't know if you're getting the active drug or a placebo.
Your doctors will carefully monitor your health and platelet levels throughout the study.

Who may be eligible?

To join this study, you need to be an adult, aged 18 or over. You must have Primary ITP, which means your ITP isn't caused by another medical condition. Your ITP must have been diagnosed more than two months ago, but less than five years ago. Also, you should have previously responded to treatments like steroids or IVIg (a type of drip) but then your platelet count dropped again. Your platelet count must currently be low.

For safety, if you are over 60, doctors will check your bone marrow to make sure it's healthy. If you are a woman who could become pregnant, you'll need to have negative pregnancy tests throughout the study and use effective birth control. This could mean either not having sex at all during the study period and for a few months afterwards, or consistently using a reliable form of contraception like the pill, injection, or implant.

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 Primary ITP?
Has your ITP lasted for more than 2 months but less than 5 years?
Have your platelets dropped again after previous treatments like steroids?
Is your current platelet count lower than normal?
If you're a woman who could get pregnant, are you willing to use effective birth control?

Answer every question to see your result.

What does participation involve?

If you join this study, you will be randomly assigned to one of two groups: either rituximab plus belimumab or rituximab plus a placebo (a dummy drug). Both belimumab and the placebo will be given as injections under the skin every week for 24 weeks. You will also receive rituximab, which is typically given through a drip. The study is 'double-blind', meaning neither you nor your doctors will know which group you are in.

You will have regular appointments and tests over the course of the study to monitor your platelet counts and overall health. These will include blood tests and possibly other checks. The total duration of active treatment with injections is 24 weeks, but your progress will be followed for a full year (52 weeks) to see how effective the treatment is long-term. You are free to withdraw from the study at any time.

Potential risks and benefits

Participating in research studies like this can offer several potential benefits, such as access to a new treatment that might improve your condition if the belimumab combination is more effective than standard care. You'll also receive careful monitoring by a medical team. However, there are potential risks, including side effects from the study drugs, which will be fully explained by the study team. As with all medical treatments, there's no guarantee the treatment will work for you. You have the right to withdraw from the study at any time, for any reason, without affecting your usual medical care.

Locations (1)

Henri Mondor Hospital
Verified postcode
Créteil, France· Recruiting

Common questions

What is ITP?

ITP stands for Immune Thrombocytopenia. It’s a condition where your body's immune system attacks and destroys your own platelets, which help your blood clot.

What are platelets?

Platelets are tiny cells in your blood that help stop bleeding by forming clots when you get a cut or injury.

What is a placebo?

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

How long will the treatment last?

The injections (belimumab or placebo) will be given weekly for 24 weeks, but the study will keep track of your health for a full year to see long-term effects.

Is this a new drug?

Belimumab is already approved for another condition, but its use in combination with rituximab for ITP is being tested for the first time in this study.

How to find out more

Matthieu MAHEVAS, Professor of medicine

matthieu.mahevas@aphp.fr 0 1 49 81 20 76 Official trial record

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