Ongoing, recruitingPhase I and Phase II (Integrated)- OtherInterventional

Fedratinib in Combination with CC-486, a Hypomethylating Agent, in Patients with Accelerated Phase Myelofibrosis - FAMy

The FAMy study is investigating a new treatment approach for people diagnosed with accelerated phase myelofibrosis, a condition affecting the bone marrow. This research combines two existing medicines, Inrebic (fedratinib) and Onureg (CC-486), to see if they work better together. The study has two main parts: an initial phase to find the safest and most effective dose of the combined therapy, and a second phase to see how well patients respond to this new treatment over 24 weeks. Researchers will also be looking at changes in symptoms and other health markers to understand the overall impact of the treatment. Many people can join, as long as they are over 18 years old.

At a glance

Status

Ongoing, recruiting

Phase

Phase I and Phase II (Integrated)- Other

Sponsor

Martin-Luther-Universitaet Halle-Wittenberg

Enrolment target

Start

13 Dec 2024

Myeloproliferative neoplasm in accelerated phase (MPN-AP)

What is this study about?

This study, called FAMy, is for people who have a rare blood and bone marrow problem called accelerated phase myelofibrosis. In this condition, the bone marrow doesn't work properly and makes too many abnormal blood cells, which can lead to symptoms like feeling tired, a swollen spleen, and other problems. Current treatments can help, but researchers are always looking for better options.

This study is testing a new way of treating accelerated phase myelofibrosis by combining two medicines that are already available: Inrebic (fedratinib) and Onureg (CC-486). Inrebic is often used to treat myelofibrosis, and Onureg is used for other blood cancers. The idea is that using them together might be more effective than using either one alone.

The study has two main parts. The first part is to carefully find the right dose of these two medicines when they are given together. This is important to make sure the treatment is as safe as possible while still being effective. Once the best dose is found, the second part of the study will look at how well patients respond to this combined treatment over 24 weeks. Researchers will be checking if the treatment helps to improve their condition and ease their symptoms.

Key takeaways

Tests a new combination of two medicines for accelerated phase myelofibrosis.
Aims to find the safest and most effective dose of Inrebic and Onureg together.
Monitors patient response and symptoms over 24 weeks in the second phase.
Open to adults aged 18 and over.
Includes regular health checks and blood tests.
You can stop participating at any time.

Who may be eligible?

This study is open to both men and women.

To be considered for this study, you must be at least 18 years old. There is no upper age limit, meaning adults of all ages can potentially participate if they meet other health requirements.

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.

Have I been diagnosed with accelerated phase myelofibrosis?
Am I 18 years old or older?
Am I able to take daily oral medication (capsules/tablets)?
Am I willing to attend regular appointments for checks and tests?

Answer every question to see your result.

What does participation involve?

If you join this study, your health will be closely monitored. This includes regular check-ups, blood tests, and heart tracings (ECGs). The study involves taking two different medicines, Inrebic and Onureg, usually as capsules or tablets. The main aim of the first part of the study is to find the best and safest dose of these medicines when taken together. Once the best dose is determined, the second part will track your progress over 24 weeks to see how your body responds to the treatment. This will involve regular assessments of your symptoms, spleen size, and overall health. The total duration of your participation will depend on how you respond to the treatment and the study's plan.

Potential risks and benefits

By taking part in this study, you might have the chance to receive a new combination treatment for your condition, which could potentially improve your symptoms or slow down the disease. However, like all medicines, Inrebic and Onureg can have side effects, and combining them might lead to new or increased side effects such as nausea, diarrhoea, or vomiting. The research team will carefully monitor you for any side effects. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Germany

Common questions

What is accelerated phase myelofibrosis?

It's a serious condition where your bone marrow doesn't make enough healthy blood cells, and it's progressing more quickly than usual.

What are Inrebic and Onureg?

They are both medicines. Inrebic is often used for myelofibrosis, and Onureg is used for other blood conditions. This study is testing if they work well together.

What kind of tests will I have?

You'll have regular physical exams, blood tests, and heart tracings (ECGs) to monitor your health and the treatment's effects.

How long will the study last?

The study involves an initial phase to find the right dose, followed by a 24-week period to assess treatment response. The overall length will depend on individual progress.

Can I stop participating if I want to?

Yes, you can choose to leave the study at any time without having to give a reason, and it won't affect your future medical care.