Ongoing, recruitingTherapeutic use (Phase IV)Interventional

FAMODREP : Interest of Famotidine in Reducing Endothelial Expression of P-selectin in Children With Sickle Cell Disease: Pilot Study, Single-center, Prospective, Non-comparative

This study, called FAMODREP, is investigating whether a common medicine, famotidine, might help children with sickle cell disease. Famotidine is usually used to treat stomach issues, but researchers are exploring if it can reduce something in the blood called P-selectin. High levels of P-selectin are linked to problems in sickle cell disease. This is a small, early-stage study, meaning it’s one of the first times this idea is being tested. The aim is to see if famotidine can lower these markers after about a month of treatment. This information could help doctors understand if famotidine could be a useful treatment in the future.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic use (Phase IV)

Sponsor

Assistance Publique Hopitaux De Paris

Enrolment target

Start

20 Dec 2022

Sickle Cell Disease

What is this study about?

Sickle cell disease is a lifelong condition that affects red blood cells. These cells, which normally carry oxygen around the body, become stiff and C-shaped, like a sickle. This can block blood flow, leading to pain and other serious health problems. The FAMODREP study is exploring a new way to potentially help children with this condition using a medicine called famotidine.

Famotidine is a medication commonly used to reduce stomach acid, often prescribed for heartburn or indigestion. However, this study isn't about its usual use. Researchers are looking into whether famotidine can reduce a particular substance in the blood called P-selectin. In sickle cell disease, high levels of P-selectin are thought to contribute to blood cells sticking together and blocking vessels, which causes many of the symptoms. By lowering P-selectin, doctors hope to improve or prevent some of these issues.

This is a 'pilot study', which means it's a small, initial investigation to see if there's a strong enough effect to warrant bigger studies later. The main goal is to measure P-selectin levels in children with sickle cell disease before and after they take famotidine for 29 days. Researchers will also check other blood markers and overall health to get a broader picture of how the medicine might affect the body. The findings from this study will help guide future research into potential new treatments for sickle cell disease.

Key takeaways

This study explores a new use for a common medicine, famotidine, in children with sickle cell disease.
Researchers want to see if famotidine can reduce a blood marker called P-selectin.
P-selectin is thought to play a role in blood vessel blockages in sickle cell disease.
It's a small, early-stage study to gather initial information.
Participation involves taking famotidine for 29 days and having blood tests before and after.
The results could help guide future research into new treatments.

Who may be eligible?

This study is looking for children with sickle cell disease to take part. There are no specific age limits mentioned, so children of all ages might be able to participate. Both boys and girls are welcome to join.

To find out if your child can take part, doctors will need to check their medical history and current health carefully. This is to make sure the study is safe and appropriate for them. They'll also explain everything about the study in detail so you can make an informed decision.

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.

Does my child have sickle cell disease?
Is my child currently receiving standard care for their condition?
Am I comfortable with my child taking a new medication (famotidine) for nearly a month?
Am I able to bring my child to the hospital for blood tests at the start and end of the study?

Answer every question to see your result.

What does participation involve?

If your child takes part in this study, they will receive famotidine medication for 29 days. During this time, they will have blood tests. These tests will be done before starting the medication and again after the 29 days of treatment. Researchers will measure specific markers in the blood to see if the famotidine has had an effect. They will also keep track of your child's general health, including any changes in their blood counts and if they experience any side effects. All blood tests and health checks will likely happen at the hospital, and the total duration of active participation (taking medication and having tests) will be just over one month.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. A potential benefit of this study is that famotidine might reduce certain markers in the blood related to sickle cell disease, which could lead to better understanding and potentially new treatments in the future. However, as this is an early-stage study, it's not guaranteed that your child will personally benefit from taking famotidine, or that it will improve their condition. Potential risks include side effects from the famotidine medication, although it is generally well-tolerated. Blood tests can also cause minor discomfort. You have the right to withdraw your child from the study at any time, for any reason, without it affecting their usual medical care.

Locations (1)

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

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Unverified
France

Common questions

What is famotidine normally used for?

Famotidine is usually prescribed to reduce stomach acid, helping with conditions like heartburn or indigestion. This study is exploring a new potential use.

Will famotidine cure my child's sickle cell disease?

No, this study is not looking for a cure. It's investigating if famotidine can help manage certain aspects of the disease by reducing a specific blood marker.

How long will my child need to take the study medicine?

Your child will take famotidine for 29 days as part of this study.

Are there any age limits for children to join?

The study aims to include children of all ages, but specific health checks will determine if it's suitable for your child.

What happens if I want my child to stop participating?

You can withdraw your child from the study at any time, for any reason, without affecting their routine medical care.