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

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What is this trial about?

A plain-language summary of the goals, design and what participants do

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.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 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.
Step 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.
Step 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.
Step 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.
Step 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.
Step 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 trial?

11 criteria

Must be **18 years of age or older** at the time of signing the informed consent.
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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 trial?

4 criteria

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.

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Verified sites

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Responsive
Azienda Ospedaliera Ordine Mauriziano Di Torino
Turin, Italy
Responsive
Azienda Ospedaliero Universitaria Careggi
Florence, Italy
Responsive
Hospital Universitario Hm Sanchinarro
Madrid, Spain

Trial locations

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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.

What is already known about the treatment

Roginolisib
Roginolisib is administered orally and is currently being studied in combination with ruxolitinib for patients with myelofibrosis who do not respond to JAK inhibitors. It is in the clinical trial phase, specifically a Phase I/II study, to assess its safety and tolerability. The main therapeutic indication is for myelofibrosis, a type of bone marrow disorder. Roginolisib works by targeting specific pathways involved in cell growth and survival, aiming to reduce symptoms and improve quality of life. It is classified as an investigational drug in the context of this trial.
Ruxolitinib
Ruxolitinib is taken orally and is an established medication used to treat myelofibrosis, particularly in patients who have not responded well to other treatments. It is a well-documented medication in medical literature and is approved for use in this condition. The drug works by inhibiting certain enzymes known as Janus kinases (JAKs), which play a role in the signaling pathways that control blood cell production and immune function. Ruxolitinib is classified as a JAK inhibitor and is used to manage symptoms and improve the quality of life in patients with myelofibrosis.

Investigated diseases

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.