RecruitingPHASE1, PHASE2INTERVENTIONAL

A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia

This research study is looking into a new medicine, pirtobrutinib, for adults with Immune Thrombocytopenia (ITP). ITP is a condition where your immune system mistakenly attacks and destroys platelets, which are tiny blood cells that help your blood clot. The study has two main parts. The first part will check how well people tolerate pirtobrutinib and look for any unwanted side effects. The second part will compare different doses of pirtobrutinib against a dummy pill (placebo) to see how effective it is in increasing platelet counts and if it's safe. Taking part involves taking the study medicine by mouth and having regular blood tests.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

Eli Lilly and Company

Enrolment target

Start

30 Jul 2025

Estimated completion

01 Feb 2027

Immune Thrombocytopenia (ITP)

What is this study about?

This study is about a new medicine called pirtobrutinib for adults who have a condition called Immune Thrombocytopenia, or ITP. ITP happens when your body's defence system, called the immune system, gets confused and starts attacking your own platelets. Platelets are really important because they help your blood clot and stop bleeding. If you have ITP, your platelet count can be very low, which increases your risk of bruising and bleeding.

The main goal of this study is to find out if pirtobrutinib is a safe and effective treatment for ITP. The study will happen in two stages. First, we need to understand if the medicine is well-tolerated and what kind of side effects it might have. This helps us ensure patient safety. After that, we'll look more closely at whether different amounts (doses) of pirtobrutinib can help increase platelet counts compared to a 'dummy' pill that contains no medicine (a placebo).

If you take part, you'll be given the study medicine, pirtobrutinib, as a tablet to swallow. The researchers will also take blood samples regularly. These blood tests help them understand how much of the medicine gets into your body and how long it stays there. The study will last for different periods depending on which part you are in, but it could be up to about 16 weeks for the first stage or up to 28 weeks for the second stage, not including the initial checks to see if you can join.

Key takeaways

This study is testing a new medicine, pirtobrutinib, for adults with ITP.
It aims to check the safety and effectiveness of the medicine.
Participants will take the medicine as a tablet and have regular blood tests.
The study has two parts: one to find the right dose, and another to compare it with a dummy pill.
Taking part means frequent clinic visits and medical supervision.
You can stop participating at any time.

Who may be eligible?

To be able to join this study, you would need to be an adult (18 years or older) with a specific type of ITP called 'primary ITP'. This means your low platelet count isn't caused by another known illness. You should also have shown some improvement in your platelet count with at least one previous ITP treatment, even if that treatment no longer works for you now. Importantly, your ITP should have returned or not responded well to treatments, and your platelet count generally needs to be quite low (under 30,000) on a couple of recent occasions.

There are also some reasons why you might not be able to join. For example, if you've had a blood clot in the past year, or if you recently needed a blood transfusion. People with certain severe heart conditions, a history of blood cancers, or certain types of hepatitis (like Hepatitis B or C) would also not be able to take part. The study team will check your overall health, including your liver and kidney function, to make sure it's safe for you to participate.

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 (ITP not caused by another disease)?
Have you tried at least one ITP treatment before, and it helped at some point?
Is your ITP not responding well to current treatments or has it returned?
Are your platelet counts generally below 30,000?
Do you have suitable liver, kidney, and blood cell function (as determined by tests)?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will be taking the study medicine, pirtobrutinib, as a tablet by mouth. You will have regular visits to the clinic for medical check-ups, which will include physical exams and several blood tests. These blood tests help the study team monitor your platelet counts, check for any side effects, and see how your body handles the medicine. For women who can get pregnant, there will be rules about using contraception during the study. The total time you would be involved in the study could be up to around 16 weeks for the first part or up to 28 weeks for the second part, plus an initial assessment period.

Potential risks and benefits

Taking part in a clinical trial may offer potential benefits, such as access to a new treatment before it's widely available, and you would receive very close medical attention from the study team. However, there are also potential risks, including side effects from the study medicine, which the researchers will carefully monitor. The study will also involve regular clinic visits and blood tests, which can be time-consuming. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (45)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

USC Norris Comprehensive Cancer Center
Verified postcode
Los Angeles, United States· Recruiting
Stanford University
Verified postcode
Stanford, United States· Not yet recruiting
MedStar Georgetown University Hospital
Verified postcode
Washington D.C., United States· Recruiting
University of Miami Hospital and Clinics Sylvester Comprehensive Cancer Center
Verified postcode
Miami, United States· Not yet recruiting
Bleeding and Clotting Disorders Institute
Verified postcode
Peoria, United States· Not yet recruiting
Ochsner Clinic Foundation
Verified postcode
New Orleans, United States· Recruiting
Mayo Clinic
Verified postcode
Rochester, United States· Not yet recruiting
Clinical Research Alliance
Verified postcode
Westbury, United States· Recruiting
Texas Oncology - Central South
Verified postcode
Austin, United States· Not yet recruiting
MD Anderson Cancer Center
Verified postcode
Houston, United States· Recruiting
Texas Oncology Gulf Coast
Verified postcode
The Woodlands, United States· Not yet recruiting
Nanfang Hospital of Southern Medical University
Verified postcode
Guangzhou, China· Not yet recruiting

Common questions

What is ITP?

ITP stands for Immune Thrombocytopenia. It's a condition where your body's immune system accidentally attacks and destroys your own platelets, which are blood cells that help stop bleeding.

What is pirtobrutinib?

Pirtobrutinib is the new medicine being tested in this study. It's designed to help manage ITP.

What is a 'dummy pill' (placebo)?

A dummy pill, or placebo, looks just like the real medicine but contains no active drug. It helps researchers compare the effects of the actual medicine fairly.

How long will the study last if I join?

The study could last up to about 16 weeks for the first part or up to 28 weeks for the second part, depending on which stage you are in, plus an initial screening period.

Can I stop participating in the study if I change my mind?

Yes, you can leave the study at any time, for any reason. Your decision will not affect your medical care.

How to find out more

Trial questions or participation questions: 1-877-CTLILLY (1-877-285-4559) or

LillyTrials@Lilly.com 1-317-615-4559 Official trial record

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