Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

A Multicenter, Open-Label Clinical Trial of RVU120 in Patients with Relapsed or Refractory High-Risk Myelodysplastic Syndrome or Acute Myeloid Leukemia with or without NPM1 Mutation (RIVER-52)

This important study, called RIVER-52, is testing a new medicine, RVU120, for people over 18 who have high-risk myelodysplastic syndrome (MDS) or acute myeloid leukaemia (AML). These are types of blood cancer, and the study focuses on cases where the cancer has returned or didn't get better with other treatments. Researchers want to find out how many patients achieve complete remission (where signs of the cancer disappear) after taking RVU120. They will also look at how long people respond to the treatment, if it helps reduce the need for blood transfusions, and their overall quality of life. This is a Phase II study, meaning it's exploring how well the drug works and its safety.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Ryvu Therapeutics S.A.

Enrolment target

174

Start

21 Dec 2023

Relapsed or Refractory High-Risk Myelodysplastic Syndrome Acute Myeloid Leukemia with or without NPM1 Mutation

What is this study about?

This study is called RIVER-52 and it's looking into a new treatment for two types of blood cancer: myelodysplastic syndrome (MDS) and acute myeloid leukaemia (AML). MDS and AML are serious conditions where the body doesn't make healthy blood cells properly. This study is specifically for patients whose cancer is considered 'high-risk' and has either come back after treatment (this is called 'relapsed') or hasn't responded well to previous treatments (this is called 'refractory'). The new medicine being tested is called RVU120.

The main goal of this study is to see how many patients achieve a 'complete remission' after taking RVU120. A complete remission means that tests don't show any signs of the cancer. This is a very important measure because it indicates the treatment has been effective in controlling the disease. Researchers will also look at other important things, like how long the treatment keeps the cancer under control, whether it helps people reduce their need for blood transfusions, and if it generally improves their health and how they feel day-to-day. They will also check if the medicine is helping them get ready for a stem cell transplant, if that's an option.

This is a 'Phase II' study, which means it builds on earlier research that showed some promise. Its purpose is to gather more information about how effective RVU120 is and to make sure it's safe for people to take. The hope is that this new medicine could offer another valuable treatment option for people living with these challenging blood cancers.

Key takeaways

This study tests a new drug, RVU120, for specific blood cancers.
It's for adults with high-risk MDS or AML that has returned or not responded to other treatments.
The main aim is to see if RVU120 can achieve complete remission.
All participants will receive the new medicine.
Regular monitoring and assessments are part of taking part.
You can stop participating at any time.

Who may be eligible?

To be considered for this study, you must be 18 years old or over – there's no upper age limit. Both men and women can take part. The study is looking for people who have been diagnosed with high-risk myelodysplastic syndrome (MDS) or acute myeloid leukaemia (AML).

Importantly, your cancer must be in a situation where it has either returned after you've already had treatment for it, or it hasn't responded well to the treatments you've received in the past. This is often referred to as 'relapsed or refractory' disease. Your doctors will be able to confirm if your specific medical situation fits these criteria.

There will also be other medical checks to make sure the treatment is suitable for you and that you are healthy enough to take part. For instance, your doctors will review your general health, organ function, and any other medical conditions you might have, to ensure your safety throughout the study.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Have you been diagnosed with high-risk myelodysplastic syndrome (MDS) or acute myeloid leukaemia (AML)?
Has your cancer come back after treatment, or not responded well to previous treatments?
Are you able to attend regular hospital visits for monitoring?

Answer every question to see your result.

What does participation involve?

If you decide to join this study, you'll be taking a new medicine called RVU120. You'll have regular hospital visits, where doctors and nurses will check your health, take blood and other samples, and ask you how you're feeling. These visits are important for monitoring how the medicine is working and to keep an eye on any side effects.

The medical team will conduct various tests, including blood tests and possibly bone marrow biopsies to see how your cancer is responding. They will also ask you to fill out questionnaires about your quality of life. The exact duration of your participation will depend on how well you respond to the treatment and your individual medical situation. The study will continue as long as the treatment is beneficial for you and you tolerate it well, followed by a period of follow-up.

Potential risks and benefits

Taking part in any clinical trial means there could be potential benefits and potential risks. The main hope is that the new medicine, RVU120, could help control your cancer, possibly leading to a complete remission or improving your quality of life. However, like all medicines, RVU120 may have side effects, which the study team will carefully monitor. You will be given detailed information about these potential side effects. It's very important to remember that you are free to leave the study at any time, for any reason, without it affecting your usual medical care. The study team will make sure you fully understand all aspects before you make a decision.

Locations (4)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Italy
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Unverified
Poland
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Unverified
France
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Unverified
Spain

Common questions

What is myelodysplastic syndrome (MDS)?

MDS is a type of blood cancer where your bone marrow doesn't make enough healthy blood cells. It can sometimes develop into acute myeloid leukaemia (AML).

What is acute myeloid leukaemia (AML)?

AML is a fast-growing cancer of the blood and bone marrow, where too many abnormal white blood cells are produced.

What does 'relapsed or refractory' mean?

It means the cancer has either come back after previous treatment (relapsed) or it didn't get better with earlier treatments (refractory).

What is a 'complete remission'?

Complete remission means that medical tests no longer show any signs of the cancer. It doesn't always mean the cancer is cured, but it's a very positive step.

Will I receive the new drug or a placebo?

This study focuses on giving the new drug (RVU120) to all participants. It's not comparing it to a placebo (a dummy treatment) in this phase.