Study on the Safety and Effectiveness of Fedratinib and Azacitidine for Patients with Accelerated Phase Myelofibrosis

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What is this study about?

This clinical trial is focused on studying a condition known as myelofibrosis, which is a type of blood cancer that affects the bone marrow. The study is particularly interested in patients whose myelofibrosis has progressed to an accelerated phase, meaning the disease has become more severe. The treatment being tested involves a combination of two medications: fedratinib and CC-486. Fedratinib is taken in the form of hard capsules, while CC-486 is provided as film-coated tablets. The purpose of the study is to evaluate the safety and effectiveness of this combination therapy in improving the condition of patients with accelerated phase myelofibrosis.

Participants in the study will receive the combination of fedratinib and CC-486, or a placebo, to compare the effects. The study will be conducted in two phases. In the first phase, the focus will be on determining how safe and tolerable the combination is for patients. In the second phase, the study will assess how well the treatment works in terms of achieving the best possible response, which could include clinical improvement, partial remission, or complete remission of the disease. Throughout the study, participants will undergo regular check-ups, including physical exams and laboratory tests, to monitor their health and the effects of the treatment.

The study aims to gather information over a period of time to understand the duration of response to the treatment and any changes in symptoms associated with myelofibrosis. It will also look at how the treatment affects the size of the spleen, a common issue in myelofibrosis, and track the time until any disease progression or relapse occurs. The study is expected to continue until 2027, providing valuable insights into the potential benefits of combining fedratinib and CC-486 for patients with this challenging condition.

1 initial assessment

Upon joining the study, an initial assessment will be conducted. This includes a physical examination, laboratory tests, and an evaluation of your medical history.

You will be asked to sign an informed consent form, confirming your understanding and willingness to participate in the study.

2 treatment phase i

In Phase I, the main goal is to determine the safety and tolerability of the drug combination. You will receive fedratinib and CC-486.

The dosage and schedule will be determined based on safety data. Regular monitoring will include physical exams, laboratory tests, and vital signs checks.

3 treatment phase ii

In Phase II, the focus is on evaluating the best response to the combination therapy. This includes clinical improvement, partial remission, or complete remission.

The treatment will continue for up to 24 weeks, with regular assessments to monitor your response to the therapy.

4 ongoing monitoring

Throughout the trial, you will be monitored for any side effects or adverse events. This includes regular check-ups and communication with the study team.

You will be asked to report any symptoms or changes in your health promptly.

5 end of trial assessment

At the end of the trial, a final assessment will be conducted. This includes a comprehensive evaluation of your health and the effectiveness of the treatment.

You will receive information about the next steps and any follow-up care that may be necessary.

Who Can Join the Study?

Participants must be males or females who are at least 18 years old.
Participants must have a diagnosis of Myeloproliferative neoplasm in accelerated phase (MPN-AP). This means having a certain percentage (10%-19%) of immature blood cells, called blasts, in the blood or bone marrow.
Participants must have an Eastern Cooperative Oncology Group (ECOG) Performance Score of 0, 1, or 2. This score measures how well a person can perform daily activities, with 0 being fully active and 2 being able to do all self-care but unable to work.
Any side effects from previous treatments must have improved to a mild level (Grade 1) or returned to the level they were before the last treatment.
Participants must be willing and able to follow the schedule of study visits and procedures.
Participants must understand and voluntarily sign an Informed Consent Form (ICF) before any study-related assessments or procedures are conducted.

Who Cannot Join the Study?

Patients with a myeloproliferative neoplasm in accelerated phase (MPN-AP) cannot participate. This is a condition where the bone marrow makes too many blood cells too quickly.
Patients who are not within the specified age range for the study cannot participate.
Patients who are not part of the specified clinical trial groups cannot participate.
Patients who are not female or male cannot participate.
Patients who are considered part of a vulnerable population cannot participate. This means people who might be at a higher risk of harm or exploitation.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Technische Universitaet Dresden	Dresden	Germany
University Hospital Jena KöR	Jena	Germany
University Medicine Greifswald	Greifswald	Germany
Rostock University Medical Center	Rostock	Germany

Other Sites

Site Name	City	Country
Universitaetsklinikum Aachen AöR	Aachen	Germany
Muehlenkreiskliniken AöR	Minden	Germany
Klinikum Chemnitz gGmbH	Chemnitz	Germany
Mxlmfsjitcgivwmbhvkfycruyt Hujnhibshiiqlhvn	Halle (Saale)	Germany

Trial status

Country	Status	Recruitment Start
Germany	Not recruiting	03.11.2022

Trial locations

Investigated drugs:

Fedratinib is a medication used in this clinical trial to help treat a condition called accelerated phase myelofibrosis. Myelofibrosis is a type of bone marrow disorder that affects blood cell production. Fedratinib works by blocking certain proteins in the body that are involved in the growth of abnormal blood cells. This can help slow down the progression of the disease and improve symptoms.

CC-486 is another medication used in combination with fedratinib in this trial. It is known as a hypomethylating agent, which means it helps to modify the way certain genes are expressed in the body. By doing this, CC-486 can help restore normal function to bone marrow cells and improve blood cell production. This combination therapy aims to enhance the overall treatment effect for patients with accelerated phase myelofibrosis.

Investigated diseases:

Myelofibrosis

Myeloproliferative Neoplasm in Accelerated Phase (MPN-AP) – This disease is a type of blood cancer characterized by the overproduction of blood cells in the bone marrow. In the accelerated phase, the disease progresses more rapidly than in its chronic phase, with an increase in the number of immature blood cells, known as blasts, in the blood and bone marrow. Patients may experience symptoms such as fatigue, fever, weight loss, and an enlarged spleen. The accelerated phase indicates a transition towards a more aggressive form of the disease, potentially leading to further complications. The condition can cause significant changes in blood cell counts, affecting red blood cells, white blood cells, and platelets. This phase requires careful monitoring due to its potential to evolve into acute leukemia.

Trial ID:

2024-519227-22-00

Protocol code:

KKSH178

Trial Phase:

Human Pharmacology (Phase I) – Other

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Study on the Safety and Effectiveness of Fedratinib and Azacitidine for Patients with Accelerated Phase Myelofibrosis

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?