Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

A multicentre trial evaluating the efficacy and safety of oral decitabine-tetrahydrouridine (NDec) in patients with sickle cell disease

This study is looking into a new combination medication called NDec for people with sickle cell disease. The main goal is to find out if this medicine can help improve the amount of healthy red blood cells (haemoglobin) in the body after 24 weeks. Researchers will also be checking how safe the medicine is and if it reduces common problems like pain crises or the need for blood transfusions over a year. Participants will either receive the new medicine, an existing sickle cell medication (Siklos), or a dummy pill (placebo). This is a Phase II trial, meaning it's an early stage where scientists are still learning about the drug's effects and safety in a larger group of patients.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Novo Nordisk A/S

Enrolment target

Start

28 Jun 2024

Sickle cell disease

What is this study about?

This study is designed to learn more about a new combination medicine, called NDec, for people living with sickle cell disease. Sickle cell disease affects your red blood cells, making them a different shape and causing various health problems.

The main aim is to see if NDec can help increase the amount of healthy red blood cells (what doctors call 'haemoglobin') in your blood. Having more healthy haemoglobin can potentially reduce some of the difficulties people with sickle cell disease experience. Researchers will compare NDec to a commonly used sickle cell medication called Siklos, and also to a placebo – which looks like the real medicine but contains no active drug. This helps them understand the true effects of NDec.

They also want to understand how NDec works in the body and if it's safe. This involves looking at how the body processes the medicine and checking for any unwanted side effects. The study will also track important health markers over time to see if the medicine helps reduce painful episodes, other sickle cell complications, or the need for blood transfusions.

Key takeaways

This study is testing a new medicine (NDec) for adult patients with sickle cell disease.
It aims to see if NDec can increase healthy red blood cells (haemoglobin) and improve overall health.
Participants will receive NDec, another sickle cell medicine (Siklos), or a placebo.
The study will last about one year, with regular check-ups and blood tests.
The main goal is to understand the medicine's effectiveness and safety.

Who may be eligible?

To be part of this study, you need to be at least 18 years old. There's no upper age limit, so older adults can also take part.

Both men and women are welcome to join the study.

Beyond age and gender, doctors involved in the study will look at your specific health situation and medical history to make sure the study is a safe and suitable option for you. They will have a detailed list of health requirements that you would need to meet.

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?
Do you have a diagnosis of sickle cell disease?
Are you able to attend regular hospital or clinic appointments?
Are you willing to take a study medication as prescribed?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you would receive one of three treatments: the new NDec combination medicine, an existing medicine called Siklos, or a placebo (a dummy pill with no active drug). You would take this medicine regularly as instructed by the study team.

Throughout the study, you would come in for regular appointments. These visits would involve blood tests to check your haemoglobin levels, how the medicine is affecting your body, and other important health markers. You would also be asked about any symptoms you experience or any changes in your health. The total duration of the study, including follow-up, is about one year (52 weeks).

Potential risks and benefits

Taking part in any clinical trial comes with potential benefits and risks. You might find that the new medicine helps your sickle cell disease. However, it's also possible that you may not experience any improvement, or you could experience side effects. The study team will explain all known or expected risks. Remember, you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (4)

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

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Unverified
France
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Unverified
Italy
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Unverified
Greece
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Unverified
Spain

Common questions

What is sickle cell disease?

Sickle cell disease is a blood disorder where red blood cells become C-shaped (like a sickle) and break down easily, leading to anaemia, pain, and other issues.

What does 'haemoglobin' mean?

Haemoglobin is a protein in your red blood cells that carries oxygen from your lungs to the rest of your body. Healthy haemoglobin levels are important for your energy and overall health.

What is a 'placebo'?

A placebo is a 'dummy' pill that looks like the real medicine but doesn't contain any active drug. It helps researchers understand if the real medicine is truly having an effect.

What are 'vessel-occlusive crises'?

These are painful episodes that happen in sickle cell disease when sickle-shaped red blood cells block small blood vessels, stopping blood flow.

What is a 'Phase II' trial?

A Phase II trial is an early stage of research where doctors study a new medicine in a larger group of people to see if it works and to further check its safety.