Study Comparing Fedratinib with Drug Combination for Myelofibrosis in Patients Previously Treated with Ruxolitinib

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What is this study about?

This clinical trial is focused on studying a condition called myelofibrosis, which can occur on its own or after other blood disorders like polycythemia vera or essential thrombocythemia. Myelofibrosis is a type of bone marrow cancer that affects the production of blood cells. The study is comparing a new treatment called fedratinib (also known by its code name BMS-847943) with the best available therapies currently used for this condition. Fedratinib is taken as a capsule by mouth.

The purpose of the study is to evaluate how well fedratinib works in reducing the size of the spleen, an organ that can become enlarged in people with myelofibrosis. Participants in the study will be randomly assigned to receive either fedratinib or one of the standard treatments. These standard treatments may include medications like ruxolitinib, glatiramer acetate, dimethyl fumarate, peginterferon alfa-2a, hydroxycarbamide, danazol, busulfan, or other therapies that are commonly used to manage myelofibrosis. Some participants may receive a placebo, which is a substance with no active medication.

The study will take place over a period of time, during which participants will have regular check-ups to monitor their health and the effects of the treatment. The main goal is to see if fedratinib can help reduce the spleen size by at least 35% compared to the other treatments. Participants will also be monitored for any changes in their symptoms and overall health. This study aims to provide more information on the effectiveness and safety of fedratinib for people with myelofibrosis who have previously been treated with ruxolitinib.

1 initial assessment

Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying age, health status, and previous treatment history.

A diagnosis of primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis is required, confirmed by a pathology report.

A measurable spleen size and symptom score are assessed using imaging techniques like MRI or CT-scan.

2 randomization

Participants are randomly assigned to receive either fedratinib or the best available therapy (BAT).

The goal is to compare the effectiveness of fedratinib with other standard treatments.

3 treatment phase

Participants receiving fedratinib take it orally in capsule form. The dosage and frequency are determined by the study protocol.

Participants in the BAT group receive standard treatments, which may include medications like ruxolitinib, hydroxycarbamide, or others, depending on individual needs and previous treatments.

4 monitoring and assessments

Regular monitoring is conducted to assess spleen size and symptom changes. This involves periodic imaging and symptom questionnaires.

Participants are monitored for any side effects or adverse reactions to the treatments.

5 end of treatment evaluation

At the end of the treatment period, a final evaluation is conducted to measure the reduction in spleen volume and symptom improvement.

The primary goal is to achieve at least a 35% reduction in spleen volume.

6 follow-up

After completing the treatment phase, participants undergo follow-up assessments to monitor long-term effects and any ongoing symptoms.

This phase ensures the safety and effectiveness of the treatment over time.

Who Can Join the Study?

The person must be at least 18 years old when signing the informed consent form.
The person must be willing and able to follow the study visit schedule and other study requirements.
If the person is a female who can have children, she must have two negative pregnancy tests before starting the study treatment. She must agree to ongoing pregnancy testing during the study and after the study treatment ends. This applies even if she chooses not to have sexual contact with men. She must either commit to not having sexual contact with men or agree to use highly effective birth control methods without interruption, starting 14 days before the study treatment, during the study, and for 30 days after stopping the study treatment.
If the person is a male, he must either not have sexual contact or agree to use a condom during sexual contact with a pregnant female or a female who can have children while in the study, during breaks in treatment, and for at least 30 days after stopping the study treatment, or longer if required by local rules, even if he has had a successful vasectomy.
The person must have an Eastern Cooperative Oncology Group (ECOG) Performance Score of 0, 1, or 2. This score is a way to measure how well a person can perform daily activities.
The person must have a diagnosis of primary myelofibrosis or post-essential thrombocythemia or post-polycythemia vera myelofibrosis according to specific medical criteria, confirmed by the latest local pathology report.
The person must have a DIPSS Risk score of Intermediate-2 or High. This score helps to assess the risk level of the disease.
The person must have a measurable splenomegaly, which means an enlarged spleen, during the screening period. This is shown by a spleen volume of at least 450 cm³ by MRI or CT-scan and by a palpable spleen measuring at least 5 cm below the left rib margin.
The person must have a measurable total symptoms score of at least 1, as measured by the Myelofibrosis Symptom Assessment Form (MFSAF).
The person must have been previously treated with ruxolitinib and meet at least one of the following conditions:
- Treatment with ruxolitinib for at least 3 months with inadequate response, meaning less than 10% spleen volume reduction by MRI or less than 30% decrease in spleen size by touch, or regrowth to these sizes after an initial response.
- Treatment with ruxolitinib for at least 28 days complicated by any of the following: needing at least 2 units of red blood cell transfusions per month for 2 months, or severe side effects like low platelet count, anemia, bruising, and/or bleeding while on treatment.
The person must have any side effects from previous treatment resolved to a mild level or back to the level before the last treatment before starting the study.
The person must understand and voluntarily sign an informed consent form before any study-related assessments or procedures are done.

Who Cannot Join the Study?

Patients with other serious health conditions that could interfere with the study.
Patients who have had a recent major surgery or are planning to have one during the study period.
Patients who are currently participating in another clinical trial.
Patients who have a history of allergic reactions to similar medications.
Patients who are pregnant or breastfeeding.
Patients who have not recovered from the side effects of previous treatments.
Patients with certain infections that are not well controlled.
Patients with severe liver or kidney problems.
Patients who have a history of drug or alcohol abuse.
Patients who are unable to follow the study procedures or attend the required visits.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Azienda Ospedaliero Universitaria Careggi	Florence	Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Rome	Italy
Medical University Of Vienna	Vienna	Austria
Hospital Universitario De Salamanca	Salamanca	Spain
Centre Hospitalier Universitaire De Bordeaux	Bordeaux	France
Centre Hospitalier Regional Et Universitaire De Brest	Brest	France
Katholieke Universiteit te Leuven	Leuven	Belgium
Uniwersytecki Szpital Kliniczny Im Jana Mikulicza Radeckiego We Wroclawiu	Wroclaw	Poland

Other Sites

Site Name	City	Country
Centre Hospitalier Universitaire De Nimes	Nimes	France
Azienda Ospedaliero-Universitaria Sant Andre	Rome	Italy
Centre Hospitalier Universitaire De Poitiers	Poitiers	France
Centre hospitalier universitaire de Liege	Liege	Belgium
Azienda Sanitaria Universitaria Friuli Centrale	Udine	Italy
Centre Hospitalier Lyon Sud	Pierre Benite	France
Hospital Universitario 12 De Octubre	Madrid	Spain
Hospital General Universitario Morales Meseguer	Murcia	Spain
Instytut Hematologii I Transfuzjologii	Warsaw	Poland
Muehlenkreiskliniken AöR	Minden	Germany
El Hospital Universitario De Gran Canaria Dr. Negrin	Las Palmas De Gran Canaria	Spain
Institut De Cancerologie Strasbourg Europe	STRASBOURG, Alsace	France
Somogy Varmegyei Kaposi Mor Oktato Korhaz	Kaposvar	Hungary
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico	Milan	Italy
Universita’ Degli Studi Di Verona	Verona	Italy
Hospital Clinic De Barcelona	Barcelona	Spain
Az St-Jan Brugge-Oostende A.V.	Brugge	Belgium
Hospital Universitario De Cruces	Barakaldo	Spain
Hanusch Krankenhaus Der Wiener Gebietskrankenkasse	Vienna	Austria
Szabolcs-Szatmar-Bereg Varmegyei Oktatokorhaz	Nyiregyhaza	Hungary
Hospital Universitari De Girona Doctor Josep Trueta	Girona	Spain
Centre Hospitalier Universitaire De Nice	Nice	France
Uniwersytecki Szpital Kliniczny W Poznaniu	Poznan	Poland
Hospital Universitario De Canarias	La Laguna	Spain
CHU Helora	La Louviere	Belgium
Hopital Beaujon	Clichy	France
Universita Degli Studi Di Brescia	Brescia	Italy
Centre Hospitalier Universitaire Dinant Godinne Sainte-Elisabeth-UCL-Namur	Namur	Belgium
Azienda Socio Sanitaria Territoriale Dei Sette Laghi	Varese	Italy
University Hospital Ostrava	Ostrava	Czechia
Hospital Universitario Virgen De La Victoria	Malaga	Spain
Ooiwrneiddczip Lcbz Ggwh	Linz	Austria
Mdbslmxbepdororemrblzpjnrb Huwiawbmrycdfwbb	Halle (Saale)	Germany
Cexi Uhsrvsvzlm Hqghtsqv	Cork	Ireland
Mwrrahe Umrlrssbrs Op Gfvr	Graz	Austria
Agzyvad Odqaqhoebxz Udqjoahorfnxk Ckihecatmdhs Dsrxu Srvvfx E Dapme Srrvlot Dp Txdwin	Turin	Italy
Gtkwbbquxqsqrjqkl Vapnrfitj Pycy Apdkpy Eopgthdm Otihjk Kxebzc	Gyor	Hungary
Ahflhca Urryy Svhydofts Lnbpjc Dx Bigpgik	Bologna	Italy
Uuintbtwcs Debkd Szcni Dm Rtsu Lw Sqamyzgo	Rome	Italy
Iozhcrem Ccfeue Dlbpqewnudoyphehq	L'hospitalet De Llobregat	Spain
Cpxcov Hlazmgzaqla Rbannhdy Dhmnikdspnsjnu	Angers	France

Trial status

Country	Status	Recruitment Start
Austria	Not recruiting	23.08.2019
Belgium	Not recruiting	23.08.2019
Czechia	Not recruiting	23.08.2019
France	Not recruiting	23.08.2019
Germany	Not recruiting	23.08.2019
Hungary	Not recruiting	23.08.2019
Ireland	Not recruiting	23.08.2019
Italy	Not recruiting	23.08.2019
Poland	Not recruiting	23.08.2019
Spain	Not recruiting	23.08.2019

Trial locations

Investigated drugs:

Fedratinib is a medication used in this trial to treat patients with certain types of myelofibrosis, a disorder that affects the bone marrow. It works by inhibiting specific enzymes that contribute to the disease, potentially reducing symptoms and improving quality of life.

Best Available Therapy (BAT) refers to the standard treatments that are currently used for managing myelofibrosis. This can include a variety of medications and approaches that are considered effective based on current medical knowledge and practice. The specific therapies used as BAT can vary depending on the patient’s condition and the healthcare provider’s judgment.

Investigated diseases:

Myelofibrosis

Primary Myelofibrosis – This is a rare bone marrow disorder where the marrow is replaced by fibrous tissue, leading to a decrease in blood cell production. Over time, this can cause anemia, fatigue, and an enlarged spleen. The disease progresses as the fibrous tissue continues to replace normal marrow, affecting the body’s ability to produce blood cells effectively.

Post-Polycythemia Vera Myelofibrosis – This condition occurs in some individuals who have had polycythemia vera, a disease where the body produces too many red blood cells. Over time, the bone marrow becomes fibrotic, leading to reduced blood cell production and an enlarged spleen. Symptoms may include fatigue, weakness, and abdominal discomfort due to spleen enlargement.

Post-Essential Thrombocythemia Myelofibrosis – This disease develops in individuals who have had essential thrombocythemia, a condition characterized by an overproduction of platelets. As the disease progresses, the bone marrow becomes fibrotic, reducing its ability to produce blood cells. This can result in symptoms such as fatigue, anemia, and an enlarged spleen.

Trial ID:

2024-511972-33-00

Protocol code:

FEDR-MF-002

NCT ID:

NCT03952039

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study Comparing Fedratinib with Drug Combination for Myelofibrosis in Patients Previously Treated with Ruxolitinib

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?