Active not recruitingPHASE2INTERVENTIONAL

A Single-arm, Multicenter Study to Assess the Efficacy, Safety, and Tolerability of P1101 in Adults With ET

This research study is looking into a new treatment called P1101 for adults living with Essential Thrombocythaemia (ET). ET is a rare blood condition where your body makes too many platelets. We want to find out how effective P1101 is at managing the condition, whether it's safe to use, and how well people tolerate it. P1101 is a type of medicine designed to help control blood counts. The hope is that it could offer a more lasting and effective way to manage ET, potentially preventing it from getting worse over time or developing into other related conditions. Participants will take P1101 for over a year, with a follow-up period, and researchers will closely monitor their health and blood test results throughout the study.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

PharmaEssentia

Enrolment target

Start

29 Sep 2022

Estimated completion

31 Mar 2027

Essential Thrombocythemia

What is this study about?

This study is about a new medicine called P1101, which is being developed to treat a blood condition called Essential Thrombocythaemia, often shortened to ET. If you have ET, your body makes too many blood cells called platelets. This can sometimes lead to problems like blood clots or bleeding. Currently, there aren't many treatments that can truly change the course of ET, so researchers are looking for new options.

P1101 is a special type of medicine that doctors hope will not only control the number of platelets but also lead to a more in-depth improvement in the condition. The idea is that it might help manage ET more effectively over a longer period, possibly stopping it from getting worse or turning into other serious conditions like myelofibrosis or acute myeloid leukaemia. This study is an important step in understanding if P1101 can be a good treatment choice for people with ET.

This particular study is carefully looking at how well P1101 works, if it's safe, and if patients find it easy to take. It's a 'Phase 2' study, which means it's still in the earlier stages but is building on previous research. By taking part, you would be helping doctors and scientists learn more about ET and potential new ways to treat it, which could benefit many others in the future.

Key takeaways

This study is for adults with Essential Thrombocythaemia (ET).
It's testing a new medicine, P1101, to see if it's safe and effective.
P1101 aims to control blood counts and potentially improve ET over time.
Participants will take the medicine for over a year and be closely monitored.
You might be eligible even if you've taken other ET medicines before.
The study helps researchers find better treatments for ET.

Who may be eligible?

To join this study, you need to be an adult, at least 18 years old, and have been diagnosed with Essential Thrombocythaemia (ET) using specific official guidelines. Your doctor will need to confirm this.

You might be suitable if you haven't had any cytoreductive treatments for ET before, but your doctor believes you need treatment because your ET is progressing, you're experiencing noticeable symptoms like itching or severe tiredness, or you have other factors that put you at higher risk. If you have previously taken a medicine called hydroxyurea (HU) for ET, you could still be eligible. This includes if HU wasn't working well enough, or if you had to stop taking it due to side effects.

There are also some important medical checks. Your liver and kidney function need to be healthy enough for you to safely take the study medicine. If you are a woman who could become pregnant, or a man whose partner could become pregnant, you'll need to agree to use effective birth control during the study and for a short time afterwards. Women who are breastfeeding cannot take part. Your platelet count must be above a certain level when you are checked for the study.

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 a diagnosis of Essential Thrombocythaemia (ET)?
Is your liver and kidney function generally healthy?
Are you able to use effective birth control if applicable?
Are your platelet counts currently above a specific level?

Answer every question to see your result.

What does participation involve?

If you decide to join this study, you'll receive the study medicine, P1101, over a period of about 13 months. This will be followed by an 'extension period,' meaning you'll continue to be monitored for a longer time after the initial treatment phase. During this time, you'll have regular hospital visits for check-ups and a range of tests. These will include blood tests to see how your body is responding to the medicine and to monitor for any side effects. The medical team will also ask you about your symptoms and how you are feeling to understand how the treatment affects your daily life. The total duration of your participation, including the follow-up, will be decided by the study doctors.

Potential risks and benefits

Taking part in this study could offer some potential benefits. You would receive a new, experimental treatment for your Essential Thrombocythaemia which might help manage your condition more effectively than current options, possibly reducing symptoms or preventing its progression. However, like all medicines, P1101 could also have side effects, which might be mild or more serious. The study team will closely monitor you for any unwanted effects to ensure your safety. Participation also means frequent hospital visits and 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 (34)

University of Alabama at Birmingham
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Birmingham, United States
City of Hope National Medical Center
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Duarte, United States
Marin Cancer Care
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Greenbrae, United States
USC Norris Comprehensive Cancer Center
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Los Angeles, United States
Yale University School of Medicine - Yale Cancer Center
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New Haven, United States
Georgetown University Medical Center
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Washington D.C., United States
The Winship Cancer Institute Emory University
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Atlanta, United States
Fort Wayne Medical Oncology and Hematology
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Fort Wayne, United States
Mercy Health - Paducah Medical Oncology and Hematology
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Paducah, United States
Tulane University Medical Center
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New Orleans, United States
Greater Baltimore Medical Center
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Baltimore, United States
Massachusetts General Hospital
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Boston, United States

Common questions

What is Essential Thrombocythaemia (ET)?

ET is a long-term blood condition where your body makes too many cells called platelets. This can sometimes make your blood too thick, leading to problems like blood clots or bleeding.

What is P1101?

P1101 is a new medicine being tested in this study. It's a type of treatment designed to help control the number of platelets in your blood if you have ET.

What does 'cytoreductive treatment-naïve' mean?

This means you haven't previously had treatments specifically aimed at lowering your excessive blood cell counts for your ET. However, if your doctor thinks you now need such treatment, you may still be able to join.

Can I join if I've taken other ET medicines before?

Yes, if you've taken a medicine called hydroxyurea (HU) in the past, you might still be eligible, especially if it didn't work well for you or caused side effects.

How long will I be involved in the study?

You'll take the study medicine for about 13 months, and then there will be a follow-up period where your health will continue to be monitored.