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

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What is this trial about?

A plain-language summary of the goals, design and what participants do

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.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 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.
Step 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.
Step 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.
Step 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 trial?

9 criteria

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 trial?

5 criteria

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.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Verified
Amsterdam UMC
Amsterdam, The Netherlands
Verified
Az St-Jan Brugge-Oostende A.V.
Brugge, Belgium
Responsive
Azienda Ospedaliero Universitaria Careggi
Florence, Italy

Trial locations

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Investigated drugs

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.

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

What is already known about the treatment

Pelabresib – Pelabresib is administered orally in the form of tablets. It is currently being studied in clinical trials, particularly in an open-label, multicenter extension study, to assess its long-term safety and effectiveness. The main therapeutic indications for Pelabresib include myeloproliferative neoplasms such as myelofibrosis and essential thrombocythemia. At the molecular level, Pelabresib works by inhibiting certain proteins that are involved in the regulation of gene expression, which can help control abnormal cell growth. It is classified pharmacologically as a BET inhibitor, targeting bromodomain and extra-terminal domain proteins.

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.