A New Approach to Treating ITP

A New Approach to Treating ITP

Considering taking part in a clinical research study for primary immune thrombocytopenia (ITP)?

If you have been diagnosed with persistent/chronic primary ITP, we invite you to learn how the TAK-079-1004 Study may help manage your symptoms by treating the root cause of your condition.

Find a participating site

We are looking for adults at least 18 years of age who have been diagnosed with persistent/chronic primary ITP to take part in this clinical research study. You may qualify if you:

  • Are 18 years of age or older
  • Have a confirmed diagnosis of ITP, which has lasted for at least three months (“persistent”)
  • Have required platelet count levels on two blood measurements
  • Have previously taken and positively responded to an ITP therapy

Participants may remain on their current ITP treatments throughout the study. The study doctor will discuss medications and any possible changes with participants.

About the TAK-079-1004 Study

The purpose of the study is to evaluate the safety, tolerability, and effectiveness of an investigational medication, called TAK-079, compared to a placebo in managing symptoms of persistent/chronic primary ITP by treating the root cause of the condition.

Participation in the TAK-079-1004 Study can last between five and 13 months. This includes a screening period (up to 28 days), dosing period (eight weeks), safety follow-up period (eight weeks), long-term safety follow-up period (16 weeks), and optional open-label extension study (32 weeks) with active TAK-079 for participants who received the placebo during the dosing period. The length of your participation depends on whether or not you join the optional open-label extension study.

Eligible participants in the TAK-079-1004 Study will be required to attend approximately 18–32 study visits, which may occur at home or at the study site. The number of study visits depends on whether or not a participant joins the open-label extension study. At these visits, the study team will perform study-related exams and procedures, such as physical exams and blood sample collections, and participants will answer questions about their health.

About the Study Periods

Screening

This period lasts 28 days. During this period, the study team performs tests and procedures to find out if the study is a good fit for you. These procedures include blood sample collection, a physical exam, an electrocardiogram, and a review of your medical history.

Dosing Period

If the study doctor determines that you are eligible to enroll in this study and you provide consent to participate, you will enter the dosing period and will enroll in either Part A or Part B of the study. Part A will randomize participants at two different dose levels or placebo. Part B will randomize participants at one dose level or placebo. Part B is planned to open enrollment after all participants in Part A have completed the safety follow-up period.

In both parts of the study, this period lasts eight weeks and you will receive either the investigational medication or the placebo once a week. The study team will also perform tests, such as collecting blood samples, to check on your health while taking the investigational medication.

Safety Follow-Up Period

This period lasts approximately eight weeks to check on your health after you have stopped taking the investigational medication or placebo. After the Week 16 visit, the last visit in this period, the study will be unblinded. This means all participants will know what their assigned study treatment was.

Long-Term Follow-Up Period

Participants who were assigned to the placebo will have the option to either enroll in the open-label extension study or have a final visit during Week 20. Participants who were assigned to the investigational medication will have a follow-up visit every four weeks. The long-term follow-up period lasts approximately 16 weeks. During these visits, the study team will collect blood samples and ask questions about any side effects.

Open-Label Extension Study

This period lasts approximately 32 weeks. Participants who were assigned to the placebo will have the option to join this extension study and receive the investigational medication. This study consists of its own dosing period, safety follow-up period, and long-term safety follow-up period.

About the Investigational Medication

The TAK-079-1004 Study is evaluating an investigational medication, called TAK-079, for managing symptoms of ITP by treating the root cause of the condition. TAK-079 is being compared to a placebo, which looks just like the investigational medication but contains no active medicine. The placebo and TAK-079 are administered as subcutaneous injections (injections under the skin).

Participants in Part A will be chosen at random to receive one of the following study treatments:

  • TAK-079 100 mg added to stable doses of a participant’s current ITP treatment(s)
  • TAK-079 300 mg added to stable doses of a participant’s current ITP treatment(s)
  • Placebo added to stable doses of a participant’s current ITP treatment(s)

Participants in Part B will be chosen at random to receive one of the following study treatments:

  • TAK-079 600 mg added to stable doses of a participant’s current ITP treatment(s)
  • Placebo added to stable doses of a participant’s current ITP treatment(s)

To help with main treatment dosing, on each dosing day, participants will receive acetaminophen and an oral or IV antihistamine before the study treatment. Methylprednisolone steroid will also be administered after the study treatment for at least the first day of study dosing.

At the end of the safety follow-up period, all participants who received the placebo will have the option to enroll in an open-label extension study where they will be able to receive the investigational medication.

About Primary Immune Thrombocytopenia1

Primary ITP is an autoimmune bleeding disorder that is characterized by abnormally low levels of blood cells called platelets. Platelets are specialized blood cells that help stop bleeding by accelerating blood clotting. They also help maintain the structure of our blood vessels’ walls. A normal platelet count ranges from approximately 150,000 to 400,000 per microliter of blood. If someone has a platelet count lower than 100,000 per microliter of blood with no other reason for low platelets, that person might have ITP.

Our immune systems normally produce proteins called antibodies, which attach to specific foreign particles and germs, marking them for destruction. But for someone with ITP, the body’s immune system malfunctions and produces antibodies that will attack the body’s own platelets. Some of these antibodies affect the cells in the bone marrow that produce platelets, which also leads to a decrease in platelet production, further reducing the number of platelets in the blood. Some people with ITP tend to bruise and bleed easily when they have a low platelet count.

It is unknown why the immune system responds this way, and there is no known cure for ITP, which is why clinical research studies are vital. Through clinical trials, researchers are able to learn more about this condition and potentially develop future treatment options.

1. rarediseases.org/rare-diseases/immune-thrombocytopenia/

What You Should Know about Clinical Research Studies

Clinical research studies, also called clinical trials, look closely at an investigational medication or medical device to see if it is safe, how it works in the body, and if it works to treat a specific disease. Clinical research studies are conducted by doctors who are responsible for the study participants’ study-related care.

In most countries, the regulatory health authority, such as the Food and Drug Administration (FDA) in the United States, requires that several phases of clinical research be performed to better understand the safety and effectiveness of new investigational medications and certain medical devices.

Clinical research studies must be approved by an institutional review board (IRB) or ethics committee (EC). An IRB/EC is a group that is responsible for helping to protect the rights and welfare of study participants. In addition, every study participant is monitored with study-related medical tests and exams before, during, and sometimes even after the study.

Participation in any clinical research study is completely voluntary, and participants may choose to leave the study at any time for any reason. If you would like to leave the study, you should discuss this with your study doctor, who will give you information about how to do this safely.

Before you can take part in the TAK-079-1004 Study, you will first need to attend the screening visit(s) for initial tests and assessments to see if you are eligible to participate. After all necessary tests and assessments have been completed, and if you are eligible to participate, you may enter the study and receive your assigned study treatment.

Participation in this study will range between five and 13 months, depending on whether or not participants continue in the optional open-label extension study.

Find a participating site