Study on Long-Term Safety of Pelabresib for Patients with Myelofibrosis or Essential Thrombocythemia

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What is this study about?

This clinical trial is focused on studying the long-term effects of a treatment called pelabresib in patients with certain blood disorders known as myeloproliferative neoplasms, specifically myelofibrosis and essential thrombocythemia. These conditions involve the abnormal growth of blood cells in the bone marrow, which can lead to various health issues. The treatment being studied, pelabresib, is a type of medication known as a small molecule bromodomain inhibitor, which works by targeting specific proteins involved in the growth of these abnormal cells.

The purpose of this study is to evaluate the long-term safety and potential benefits of pelabresib for patients who have already been receiving this treatment in previous studies. Participants will continue to take pelabresib in the form of a tablet, and their health will be monitored over time to assess any side effects and overall survival. The study will also look at how long patients continue to benefit from the treatment and how it affects their quality of life.

Throughout the study, patients will have regular check-ups to monitor their condition and any changes in their health. The study aims to provide valuable information about the safety and effectiveness of pelabresib for managing myeloproliferative neoplasms, helping to improve treatment options for these conditions in the future.

1 joining the study

Upon joining the study, eligibility is confirmed. This includes being at least 18 years old, having been enrolled in a previous study with pelabresib, and currently receiving treatment with it.

Participants must agree to comply with scheduled visits, treatment plans, and study procedures. They must also agree to avoid pregnancy or fathering children during the study and for a specified period after the last dose of pelabresib.

2 treatment phase

During the treatment phase, participants will continue receiving pelabresib as a tablet taken orally. The dosage and frequency will be determined by the study protocol and the investigator’s assessment.

The main objectives during this phase are to evaluate the long-term safety of pelabresib, assess survival, and determine clinical benefits.

3 monitoring and assessments

Participants will undergo regular monitoring to track any treatment-emergent adverse events and serious adverse events. This includes assessing overall survival and leukemia-free survival.

Time to event endpoints, such as duration of response and progression-free survival, will also be evaluated.

4 follow-up phase

After completing the treatment phase, participants will enter a follow-up phase. This involves ongoing monitoring to assess long-term outcomes and any potential late effects of the treatment.

Participants must continue to comply with follow-up visits and assessments as outlined in the study protocol.

Who Can Join the Study?

The patient must be able to give signed informed consent, which means they understand and agree to the study’s requirements and restrictions.
The patient must be at least 18 years old and the legal age of consent in the area where the study is taking place.
The patient must have been enrolled and still be receiving treatment with pelabresib in a previous clinical study.
The patient must be experiencing clinical benefits from the treatment with pelabresib, as determined by the study doctor.
The patient must be willing and able to attend scheduled visits, follow treatment plans, and participate in other study procedures.
The patient must agree to avoid pregnancy or fathering children during the study and for a specified period after the last dose of pelabresib. This includes using effective birth control methods:

Male patients and their female partners must use birth control methods that are at least 99% effective and avoid donating sperm during this time.
Women of childbearing potential must have a negative pregnancy test before joining the study and agree to regular pregnancy testing. They must also avoid breastfeeding and donating eggs during the study and for a specified period after the last dose.
Women who cannot have children, either due to surgery or menopause, are eligible.

Who Cannot Join the Study?

Patients who have a different medical condition than the ones being studied, which are Myeloproliferative Neoplasms (a group of diseases where the bone marrow makes too many blood cells) like Myelofibrosis (a condition where scar tissue forms in the bone marrow) and Essential Thrombocythemia (a condition where the body produces too many platelets).
Patients who are not within the specified age range for the study.
Patients who are not part of the specified clinical trial group.
Patients who are not male or female, as both genders are included in the study.
Patients who are considered part of a vulnerable population, which means they might need special protection or care.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

Site Name	City	Country	Status
Azienda Ospedaliero Universitaria Careggi	Florence	Italy

Other Sites

Site Name	City	Country
Az St-Jan Brugge-Oostende A.V.	Brugge	Belgium
Gasthuiszusters Antwerpen	Antwerp	Belgium
Azienda Ospedaliero-Universitaria Maggiore Della Carita	Novara	Italy
Azienda Socio Sanitaria Territoriale Dei Sette Laghi	Varese	Italy
Ajbodhywd Unp	Amsterdam	The Netherlands

Trial status

Country	Status	Recruitment Start
Belgium	Recruiting	01.06.2024
Italy	Recruiting	01.06.2024
The Netherlands	Recruiting	01.06.2024

Trial locations

Investigated drugs:

2-((4S)-6-(4-Chlorophenyl)-1-Methyl-4H-Benzo[C]Isoxazolo[4,5-E]Azepin-4-Yl)Acetamide Monohydrate

Pelabresib is a medication being studied for its long-term safety and effectiveness in patients. It is used to assess survival and clinical benefits as determined by the investigators. This study is an extension for patients who have previously participated in other studies involving this medication.

Investigated diseases:

Myeloproliferative Neoplasms – These are a group of diseases where the bone marrow makes too many red blood cells, white blood cells, or platelets. This overproduction can lead to various symptoms and complications, such as an increased risk of bleeding or clotting. Over time, these conditions can cause the bone marrow to become fibrotic, or scarred, which can affect blood cell production. The diseases can progress slowly, and symptoms may vary depending on the specific type of neoplasm. Common types include myelofibrosis and essential thrombocythemia, each with its own unique characteristics and progression patterns.

Myelofibrosis – This is a rare type of bone marrow cancer that disrupts the body’s normal production of blood cells. It leads to extensive scarring in the bone marrow, which can result in severe anemia, weakness, and fatigue. As the disease progresses, it may cause an enlarged spleen and liver due to the body’s attempt to produce blood cells outside the bone marrow. Patients may experience symptoms such as night sweats, fever, and bone pain. The progression of myelofibrosis can vary greatly among individuals, with some experiencing rapid changes and others having a more stable course.

Trial ID:

2023-508950-24-00

Protocol code:

CDAK539A12001B

NCT ID:

NCT06401356

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on Long-Term Safety of Pelabresib for Patients with Myelofibrosis or Essential Thrombocythemia

What is this study about?

Who Can Join the Study?

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