An Open-Label Clinical Trial of RVU120 as Monotherapy and in Combination with Ruxolitinib in Patients with Intermediate or High-Risk, Primary or Secondary Myelofibrosis (POTAMI-61)
Myelofibrosis is a rare type of blood cancer that causes scar tissue to build up in the bone marrow. This makes it difficult for the body to produce normal blood cells, often causing severe tiredness and an enlarged spleen. This clinical trial, called POTAMI-61, is looking at a new experimental medicine called RVU120. Researchers want to see how safe and effective it is for adults with intermediate or high-risk myelofibrosis. Participants take RVU120 either on its own or alongside ruxolitinib (also known as Jakavi), an existing treatment. The main aim is to see if the treatment shrinks the spleen by at least 35% after 24 weeks, measured using MRI or CT scans. Researchers will also check if it reduces bone marrow scarring, improves daily symptoms, and helps people live longer and feel better.
Em resumo
Sobre o que é este estudo?
Myelofibrosis is a rare and serious blood cancer. Inside our bones, bone marrow normally acts like a factory making red blood cells, white blood cells, and platelets. In myelofibrosis, scar tissue builds up in the marrow, preventing it from working properly. Because the bone marrow struggles, organs like the spleen try to take over making blood cells. This causes the spleen to swell, leading to pain, fullness in the tummy, and severe fatigue.
This phase II trial is exploring a new tablet called RVU120. It works by blocking specific proteins that help cancer cells grow and survive. The study is testing whether taking RVU120 alone, or in combination with an approved medicine called ruxolitinib (Jakavi), can give patients better control over their illness. Ruxolitinib is commonly used to treat myelofibrosis, but not everyone responds well to it over time.
During the trial, doctors will use MRI or CT scans to measure the size of participants' spleens after 24 weeks of treatment. They will also look at whether the treatment improves scar tissue in the bone marrow, boosts blood counts, and relieves everyday symptoms such as night sweats, bone pain, and fatigue. Finding new treatments could offer vital options for people living with higher-risk forms of this condition.
Pontos principais
- Tests a new medicine, RVU120, alone or with ruxolitinib for myelofibrosis.
- Aims to shrink enlarged spleens and relieve disease symptoms after 24 weeks.
- Open to adults aged 18 and older with intermediate or high-risk myelofibrosis.
- Requires regular clinic visits for blood tests, symptom surveys, and imaging scans.
- Taking part is completely voluntary, and you can stop at any time.
Quem pode ser elegível?
This trial is open to adults aged 18 and over who have been diagnosed with intermediate-risk or high-risk myelofibrosis. This includes primary myelofibrosis (which starts on its own) and secondary myelofibrosis (which develops after other blood conditions like polycythaemia vera or essential thrombocythaemia).
To take part, participants must be well enough to carry out daily activities and be able to swallow tablets. You will also need to have regular scans and clinic visits so doctors can monitor your health.
You may not be able to join if you have certain other serious health issues, such as severe kidney or liver problems, active severe infections, or if you are pregnant or breastfeeding. Your hospital doctor will carry out detailed checks to confirm whether this study is safe and suitable for you.
Este estudo pode ser adequado para você?
Responda a estas perguntas rápidas para ver se você pode ser elegível. Este é apenas um guia — a decisão final é da equipe de pesquisa.
- I am aged 18 or older.
- I have been diagnosed with intermediate or high-risk myelofibrosis.
- I am able and willing to swallow daily tablets.
- I can attend regular hospital visits for scans, blood tests, and check-ups.
- I am willing to discuss my full medical history and current medications with the trial team.
O que envolve a participação?
If you join this study, you will take RVU120 as a tablet by mouth, either on its own or combined with ruxolitinib tablets. Because this is an 'open-label' study, both you and your doctor will know exactly which medicines and doses you are taking.
You will need to visit the hospital regularly for health checks. These visits will include blood tests to check your blood counts, physical exams, and health questionnaires about your daily symptoms. You will have CT or MRI scans, especially around week 24, to see if your spleen has shrunk. Bone marrow samples may also be taken to see if the scarring inside your bones has improved.
The initial treatment period is 24 weeks (about six months). If the medicine is working well and you are not having difficult side effects, you may be able to stay on the treatment for longer.
Riscos e benefícios potenciais
Localizações (2)
- —Não verificadoPolônia
- —Não verificadoItália
Perguntas frequentes
What is an 'open-label' trial?
An open-label trial means that both you and your medical team know which study medicines you are taking. There is no dummy pill (placebo) used in this study.
How is the spleen measured?
Doctors use imaging scans, such as an MRI or CT scan, to take detailed 3D pictures of your tummy. This allows them to measure the exact size and volume of your spleen before and during treatment.
Will taking part cost me anything?
No. The study medications and trial-related tests are provided free of charge. In many cases, travel expenses to and from hospital appointments may also be reimbursed.
Can I take my usual medications during the trial?
Some medicines can interact with RVU120 or ruxolitinib. Your study doctor will review all of your regular medicines, including over-the-counter tablets and supplements, to ensure they are safe to take together.
What happens if the treatment stops working for me?
If your illness gets worse or if you experience unpleasant side effects, your doctor will discuss stopping the trial treatment and moving you onto the best available alternative care.
Como saber mais
Sempre consulte seu médico ou especialista antes de decidir participar de um estudo.
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