C1131003 - AN INTERVENTIONAL PHASE 2, OPEN-LABEL, MULTI-CENTER STUDY TO EVALUATE SAFETY AND EFFICACY OF PF-06835375 IN ADULT PARTICIPANTS WITH MODERATE TO SEVERE PRIMARY IMMUNE THROMBOCYTOPENIA
Primary immune thrombocytopenia (ITP) is a blood condition that causes low levels of platelets, which are cells that help your blood clot. People with ITP often bruise easily or bleed more than usual. This Phase 2 study is testing an experimental medicine called PF-06835375. Researchers want to find out if this new injection can safely increase platelet counts in adults with moderate to severe ITP. Because this is an 'open-label' study, all participants will receive the study medicine at one of several planned doses—there is no dummy drug (placebo). Participants will also be given an allergy medicine (diphenhydramine) before their treatment to help prevent common injection reactions. Doctors will monitor participants closely over 12 to 16 weeks and beyond, checking their blood counts and watching for any side effects to see how well the treatment works.
At a glance
What is this study about?
Primary immune thrombocytopenia (ITP) is an autoimmune condition. In a healthy body, blood cells called platelets stick together to stop bleeding when you get a cut or bruise. If you have ITP, your immune system mistakenly attacks and destroys your own platelets. This leaves you with a low platelet count, which can lead to easy bruising, purple spots on the skin, nosebleeds, or more serious bleeding problems.
While there are existing treatments for ITP, they do not work for everyone, and some can cause difficult side effects. This clinical trial is testing a new injectable drug known as PF-06835375. Scientists have designed this medicine to calm the immune system's attack on platelets, aiming to let platelet numbers return to safer levels.
This study is a Phase 2 trial, which means researchers are working out how well the medicine works and finding the right dose. Participants will receive one of several dose amounts. Doctors will follow participants for several months, testing blood samples to see if their platelet counts rise and staying watchful for any unwanted side effects. Finding new and better ways to treat ITP could help people manage their condition with fewer bleeding risks.
Key takeaways
- Tests a new injection (PF-06835375) designed to raise platelet counts in adults with ITP.
- Open to adults aged 18 and older with moderate to severe primary ITP.
- All participants receive the study drug; there is no dummy treatment (placebo).
- Requires regular clinic visits for blood tests, injections, and safety checks over several months.
- Participation is completely voluntary and you can leave the study at any time.
Who may be eligible?
This study is open to adults aged 18 and older of any sex who have been diagnosed with moderate to severe primary immune thrombocytopenia (ITP). It is meant for people whose platelet counts remain low and who may need new treatment options.
To join, participants must be able to attend regular clinic appointments for injections, health checks, and blood tests.
Not everyone will be able to take part. People with certain other underlying medical conditions, active infections, or those taking specific medications that could clash with the study drug may not be eligible. The study doctor will review your full medical history and run tests to decide if the trial is safe 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.
- Are you aged 18 or older?
- Have you been diagnosed with primary immune thrombocytopenia (ITP)?
- Is your ITP currently considered moderate to severe?
- Are you willing to receive injections and have regular blood tests?
- Can you travel to a study centre for scheduled check-ups over several months?
What does participation involve?
If you take part, you will visit a specialist hospital clinic regularly. During your visits, doctors and nurses will check your overall health, measure your vital signs, and take routine blood samples to monitor your platelet counts and immune cells. You will receive the study medicine as an injection. Before the injection, you may receive an antihistamine called diphenhydramine to help stop any mild allergic or infusion reactions. The main treatment phase lasts around 12 to 16 weeks, depending on the dose group you are in. After this, there will be follow-up checks so the medical team can see how you are doing over time.
Potential risks and benefits
Locations (3)
- —UnverifiedPoland
- —UnverifiedCzechia
- —UnverifiedHungary
Common questions
What is primary immune thrombocytopenia (ITP)?
ITP is a blood disorder where your immune system mistakenly destroys your platelets, making it harder for your blood to clot and increasing the risk of bruising and bleeding.
Will I receive a placebo (dummy medicine)?
No. This is an 'open-label' study, which means every participant receives the real study medicine, and both you and your doctor will know the dose you are getting.
Why is diphenhydramine given before the injection?
Diphenhydramine is a common antihistamine medicine. It is given to help prevent mild allergic symptoms or reactions that can sometimes happen when receiving an injection of a new medicine.
How long will I be in the study?
The main part of the study lasts between 12 and 16 weeks, followed by further monitoring visits to check on your long-term health and platelet counts.
What happens if I want to stop taking part?
You can leave the trial at any time for any reason. Your regular doctors will support your decision and continue to provide standard treatment for your condition.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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