Study on the Effects of Navitoclax and Ruxolitinib for Patients with Myelofibrosis

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What is this study about?

This clinical trial is focused on studying a condition called Myelofibrosis, which is a type of blood cancer that affects the bone marrow. The study is testing a treatment using a medication called Navitoclax, either by itself or in combination with another medication called Ruxolitinib. Navitoclax is a type of drug known as a BCL-2 family protein inhibitor, which means it works by blocking certain proteins that help cancer cells survive. Ruxolitinib is a Janus Associated Kinases (JAKs) inhibitor, which helps reduce inflammation and control the growth of cancer cells.

The purpose of this study is to evaluate how well Navitoclax, alone or with Ruxolitinib, can reduce the size of the spleen in patients with Myelofibrosis. The study will observe participants over a period of time to see how their spleen size changes and to monitor any side effects they might experience. Participants will take the medication in tablet form, and the study will involve regular check-ups to assess their health and the effectiveness of the treatment.

Throughout the study, participants will be monitored for changes in their symptoms and overall health. The study aims to see if there is a significant reduction in spleen size and improvement in symptoms, such as fatigue and discomfort. The trial will also look at other factors like anemia response and changes in bone marrow condition. This research hopes to provide valuable information on the potential benefits of Navitoclax and Ruxolitinib for people living with Myelofibrosis.

1 joining the study

Upon joining the study, eligibility is confirmed based on specific criteria such as age, symptoms, and medical history related to myelofibrosis.

Participants must have a documented diagnosis of intermediate or high-risk primary myelofibrosis or related conditions.

2 initial assessment

An initial assessment is conducted to measure spleen size and evaluate symptoms using MRI or CT scans.

Laboratory tests are performed to ensure adequate bone marrow, kidney, and liver function.

3 medication administration

Participants receive navitoclax and/or ruxolitinib orally in tablet form.

For those in Cohort 1a, a stable dose of at least 10 mg of ruxolitinib twice daily is required for at least 8 weeks before starting navitoclax.

The treatment duration and specific dosage depend on the cohort and individual response.

4 ongoing monitoring

Regular monitoring of spleen volume and symptom severity is conducted throughout the study.

Participants are assessed for any side effects or changes in health status.

5 evaluation of outcomes

The primary goal is to achieve a reduction in spleen volume by at least 35% from the baseline at Week 24.

Secondary outcomes include a reduction in symptom scores, improvement in anemia, and changes in bone marrow fibrosis.

6 completion of study

The study is estimated to conclude by March 2029.

Final assessments are conducted to evaluate the overall effectiveness and tolerability of the treatment.

Who Can Join the Study?

Participants must be 18 years or older.
For certain groups (Cohorts 1b and 3), participants must have at least 2 symptoms with a score of 3 or more, or a total score of 12 or more on a symptom assessment over 4 out of 7 days before starting the study medication.
Participants must have splenomegaly, which means an enlarged spleen. This is defined as the spleen being 5 cm or more below the rib cage or having a spleen volume of 450 cm³ or more as measured by MRI or CT scan.
Participants must have adequate bone marrow, kidney, and liver function as specified in the study protocol.
Participants must have a confirmed diagnosis of Intermediate or High-risk Myelofibrosis, which includes different types like primary myelofibrosis, post-polycythemia vera myelofibrosis, or postessential thrombocythemia myelofibrosis.
Participants must be classified as intermediate-2 or high-risk myelofibrosis according to a scoring system called the Dynamic International Prognostic Scoring System (DIPSS).
Participants must be unable or unwilling to undergo a stem cell transplant due to age, other health conditions, or personal choice.
Participants must have an ECOG performance status of 0, 1, or 2, which is a scale used to assess how a disease affects a patient’s daily living abilities.
For Cohort 1a, participants must have been on ruxolitinib therapy for at least 12 weeks and on a stable dose of 10 mg or more twice daily for at least 8 weeks before the first dose of the study drug, navitoclax.
For Cohort 1b, participants must have been treated with ruxolitinib for 24 weeks or more with no improvement, or for less than 24 weeks with disease progression, or for 28 days or more with intolerance, which means needing new red blood cell transfusions.
For Cohort 2, participants must have had prior treatment with a JAK-2 inhibitor for at least 12 weeks, or for 28 days or more if it led to needing red blood cell transfusions or severe side effects like low platelet count, anemia, bruising, or bleeding.
For Cohort 3, participants must not have had previous treatment with a JAK-2 or BET inhibitor.

Who Cannot Join the Study?

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Azienda Ospedaliero Universitaria Careggi	Florence	Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Rome	Italy

Other Sites

Site Name	City	Country
Azienda Ospedaliero-Universitaria Policlinico G. Rodolico-San Marco Di Catania	Catania	Italy
Hospital General Universitario Gregorio Maranon	Madrid	Spain
Hospital Universitario 12 De Octubre	Madrid	Spain
Grande Ospedale Metropolitano Bianchi Melacrino Morelli	Reggio Calabria	Italy
KBC Split	Split	Croatia
Universita Degli Studi Di Brescia	Brescia	Italy
Azienda Socio Sanitaria Territoriale Dei Sette Laghi	Varese	Italy
Hospital Universitario Virgen De La Victoria	Malaga	Spain
Lipja Gujgxsf Hkudddcb Ou Avlpnd	Athens	Greece
Axpnfiw Uincu Sfnvdhvzs Lafyoq Do Byussjw	Bologna	Italy
Hjkdlbzl Uyepbwqosjhxa Hzpecgrs Trzsd y Pghjar Ijejqdej Ctqdgh dxhvrlsgcfqdhemlo (tgmj	Badalona	Spain
Hnvqvjur Vtkb dyveycsk	Barcelona	Spain
Cscoupfk Huwggnxy Dmxbneb	Zagreb	Croatia

Trial status

Country	Status	Recruitment Start
Croatia	Not recruiting	18.06.2020
Greece	Not recruiting	18.06.2020
Italy	Not recruiting	18.06.2020
Spain	Not recruiting	18.06.2020

Trial locations

Investigated drugs:

Ruxolitinib

Navitoclax is a medication being studied for its ability to reduce spleen size in patients with myelofibrosis. It works by targeting certain proteins in the body that help cancer cells survive, potentially leading to the death of these cells.

Ruxolitinib is another medication used in this study, often in combination with Navitoclax. It is already used to treat myelofibrosis and works by blocking certain enzymes that contribute to the disease, helping to reduce symptoms and spleen size.

Myelofibrosis – Myelofibrosis is a rare bone marrow disorder where the marrow is replaced by fibrous tissue, leading to a decrease in the production of blood cells. This condition often results in an enlarged spleen and can cause symptoms such as fatigue, weakness, and anemia. Over time, the fibrous tissue can disrupt normal blood cell production, leading to a variety of complications. Patients may experience a range of symptoms, including night sweats, fever, and weight loss. The disease can progress slowly or rapidly, varying significantly from person to person.

Trial ID:

2023-507276-53-00

Protocol code:

M16-109

NCT ID:

NCT03222609

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on the Effects of Navitoclax and Ruxolitinib for Patients with Myelofibrosis

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