Study on the Safety of Roginolisib and Ruxolitinib for Patients with Myelofibrosis Unresponsive to JAK Inhibitors

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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 specifically for patients whose condition has not improved with a type of treatment known as JAK inhibitors. The trial will test the safety and how well patients tolerate a new combination of medications: Ruxolitinib and Roginolisib (also known as IOA-244).

The purpose of the study is to see if combining these two medications can help patients with Myelofibrosis who have not responded to previous treatments. Participants in the study will take these medications in the form of tablets. The study will monitor patients over time to see how their bodies react to the treatment and to check for any side effects. The study will also look at changes in symptoms and overall health during the treatment period.

Throughout the study, patients will have regular check-ups, which may include physical exams and tests like ECG (a test that checks the heart’s rhythm) and blood tests. The study aims to gather information on how the combination of Ruxolitinib and Roginolisib affects the size of the spleen, a common issue in Myelofibrosis, and whether it can improve symptoms and quality of life for patients. The trial will continue for several years to ensure comprehensive data collection and analysis.

1 introduction to the study

Upon joining the study, the patient will receive detailed information about the trial, including its purpose, procedures, and potential risks and benefits.

The patient will be required to sign an informed consent form, confirming understanding and willingness to comply with the study requirements.

2 initial assessment

The patient will undergo a comprehensive medical evaluation to confirm eligibility, including a review of medical history and current health status.

Tests may include blood tests, physical examinations, and imaging studies to assess the condition of the spleen and other relevant health parameters.

3 treatment initiation

The patient will begin treatment with two medications: ruxolitinib and roginolisib, both administered orally in tablet form.

The dosage and frequency of administration will be determined by the study protocol and the patient’s specific health needs.

4 ongoing monitoring

Throughout the study, the patient will attend regular follow-up visits to monitor health status and response to treatment.

These visits will include physical examinations, blood tests, and possibly imaging studies to evaluate changes in spleen size and symptom severity.

5 evaluation of treatment response

The patient’s response to treatment will be assessed at specific intervals, such as 12 and 24 weeks, using various measures including spleen volume reduction and symptom score changes.

The study will also monitor for any side effects or adverse events related to the treatment.

6 completion of study participation

Upon completion of the study period, the patient will undergo a final evaluation to assess overall health and treatment outcomes.

The patient will receive guidance on any necessary follow-up care or additional treatment options.

Who Can Join the Study?

Must be 18 years of age or older at the time of signing the informed consent.
Must take steps to avoid pregnancy or fathering children during the study and for at least 1 month after the last dose of study treatment. This includes:
- Women who cannot have children (e.g., due to surgery or menopause) or women who can have children but have a negative pregnancy test and agree to use effective birth control methods.
- Men who agree to use effective methods to prevent fathering a child.
Must be able to give signed informed consent, agreeing to follow the study requirements.
Must have an ECOG performance status of 0 to 2, which is a scale used to assess how a disease affects a patient’s daily living abilities.
Must have a diagnosis of Myelofibrosis (MF), Post-Polycythaemia Vera Myelofibrosis (PPV-MF), or post-essential thrombocythemia MF (PET-MF).
Must have a DIPSS risk category of intermediate-1, intermediate-2, or high. This is a scoring system used to determine the risk level of the disease.
Must have been treated with ruxolitinib for at least 3 months, with a stable dose of at least 10 mg for a minimum of 8 weeks before starting the study. The spleen must not have reduced by more than 25% and must be felt at least 10 cm below the left rib margin during a physical exam.
Must not have received any other experimental drug therapy for MF or any other effective treatment for MF, except for ruxolitinib, within 3 months before starting the study drug. If other treatments were used, they must have been completed 6 months before starting ruxolitinib.
Must have active symptoms of MF at the screening visit, shown by a Total Symptom Score (TSS) of 10 or more using the Screening Symptom Form.
Must have a peripheral blast count of less than 10%. This is a measure of immature blood cells in the bloodstream.

Who Cannot Join the Study?

Patients who have not responded to JAK inhibitors. JAK inhibitors are a type of medication used to treat certain blood disorders.
Patients who are not diagnosed with Myelofibrosis (MF). Myelofibrosis is a rare type of blood cancer that affects the bone marrow.
Patients who are not within the specified age range for the study.
Patients who are part of a vulnerable population, which means they might need special protection or care.

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
Hospital Universitario Hm Sanchinarro	Madrid	Spain
IRCCS Humanitas Research Hospital	Rozzano	Italy
Hospital Universitario De Salamanca	Salamanca	Spain
Azienda Ospedaliera Universitaria Federico II Di Napoli	Naples	Italy

Other Sites

Site Name	City	Country
Azienda Sanitaria Universitaria Friuli Centrale	Udine	Italy
Azienda Ospedaliera Ordine Mauriziano Di Torino	Turin	Italy
Grande Ospedale Metropolitano Bianchi Melacrino Morelli	Reggio Calabria	Italy
Iuhaklwc Cezizt Dzswzvkciuntxzhhi	L'hospitalet De Llobregat	Spain
Isotdodr Rkunvurss Poc Lq Sdephg Dbd Ttfcgg Djqa Aumtzzs Ieej Smbgcc	Meldola	Italy
Aiwbsqn Odytygkvycf Urkywcyotgkuk Cnjoahpzdstq Dprqj Sgkyom E Drgmr Shuftub Df Tnuucj	Turin	Italy
Aepcpnm Uwgsz Skspaheek Ljwbya Dm Bvczrqb	Bologna	Italy
Uyhsatkved Dtiua Smxtr Dl Rzru Lq Sqpixbjc	Rome	Italy
Hzjxdhzm Vbze dvlzdbnr	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Italy	Recruiting	02.01.2025
Spain	Recruiting	02.01.2025

Trial locations

Investigated drugs:

Roginolisib is a medication being studied for its potential to help patients with myelofibrosis, a type of bone marrow disorder. It is being tested to see if it can be safely used together with another medication to improve patient outcomes.

Ruxolitinib is a medication already used to treat myelofibrosis. It works by targeting specific pathways in the body that contribute to the disease. In this study, it is being combined with roginolisib to see if the combination is more effective for patients who have not responded well to other treatments.

Investigated diseases:

Myelofibrosis

Myelofibrosis – Myelofibrosis is a disorder where scar tissue forms in the bone marrow, disrupting the body’s ability to produce blood cells. This condition can lead to anemia, fatigue, and an enlarged spleen. Over time, the bone marrow becomes increasingly fibrotic, reducing its capacity to generate blood cells. Patients may experience symptoms such as weakness, weight loss, and night sweats. As the disease progresses, the spleen and liver may enlarge due to the body’s attempt to produce blood cells outside the bone marrow. Myelofibrosis can also cause a variety of other symptoms, including bone pain and a feeling of fullness in the abdomen.

Trial ID:

2024-515252-20-00

Trial Phase:

Human Pharmacology (Phase I) – Other

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Study on the Safety of Roginolisib and Ruxolitinib for Patients with Myelofibrosis Unresponsive to JAK Inhibitors

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?