Study on the Effect of Famotidine in Reducing P-selectin Levels in Children with Sickle Cell Disease

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What is this study about?

This clinical trial is focused on studying the effects of a medication called Famotidine in children with Sickle Cell Disease. Sickle Cell Disease is a genetic condition that affects the red blood cells, causing them to become misshapen and leading to various health complications. The study aims to explore whether Famotidine can help reduce the expression of a protein called P-selectin, which is involved in the inflammation and blockage of blood vessels, a common issue in Sickle Cell Disease.

Participants in the study will take Famotidine, which is administered as an oral suspension, for a period of 29 days. The study will monitor changes in the levels of P-selectin and other related molecules in the blood before and after the treatment. This will help researchers understand the potential benefits of Famotidine in managing Sickle Cell Disease symptoms. The study is designed as a pilot, meaning it is a preliminary investigation to gather initial data on the treatment’s effects.

Throughout the study, researchers will also observe any changes in blood values, such as hemoglobin and other markers, to assess the overall impact of Famotidine on the participants’ health. Additionally, the occurrence of any adverse events will be recorded to ensure the safety of the treatment. This trial is an important step in exploring new ways to improve the quality of life for children affected by Sickle Cell Disease.

1 initial visit

Upon joining the study, an initial visit is scheduled. During this visit, eligibility is confirmed based on specific criteria, such as age and medical history related to sickle cell disease.

A blood test is conducted to measure baseline levels of certain proteins and blood components. This includes P-selectin, which is a protein involved in blood cell interactions.

2 medication administration

The medication famotidine is administered orally. The dosage and frequency are determined by the study protocol, and the treatment lasts for 29 days.

Famotidine is intended to reduce the expression of P-selectin in the blood, which may help in managing symptoms of sickle cell disease.

3 follow-up visits

Regular follow-up visits are scheduled throughout the 29-day treatment period. These visits are used to monitor health status and any potential side effects.

Blood tests are repeated to measure changes in the levels of P-selectin and other blood components, such as hemoglobin and reticulocytes.

4 final assessment

At the end of the 29-day treatment period, a final assessment is conducted. This includes a comprehensive review of any changes in health status and blood test results.

The primary focus is on the difference in P-selectin levels before and after treatment. Secondary assessments include changes in other blood markers and the occurrence of any adverse events.

Who Can Join the Study?

The patient must be a child or adolescent aged from 1 year to at most 17 years and 10 months.
The patient must have Sickle Cell Disease, specifically the SS or Sβ0 type.
The patient must have had at least one vaso-occlusive crisis in the year before joining the study. (A vaso-occlusive crisis is a painful event that occurs when blood flow is blocked in small blood vessels.)
For young girls who are of childbearing age (15 years or older), a negative pregnancy test is required.
Both parents or legal representatives must provide signed informed consent, and the child or adolescent, if able to express themselves, should also give oral and, if possible, signed consent.
The patient must be a beneficiary of social security coverage or entitled to it (excluding SMA).

Who Cannot Join the Study?

Patients who do not have Sickle Cell Disease cannot participate.
Patients who are not within the specified age range cannot participate.
Patients who are part of a vulnerable population cannot participate.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

Site Name	City	Country	Status
Hopital Necker Enfants Malades	Paris	France

Other Sites

No sites found in this category

Trial status

Country	Status	Recruitment Start
France	Not recruiting	12.01.2022

Trial locations

Investigated drugs:

Famotidine

Famotidine is a medication that is being studied for its potential to reduce the expression of a protein called P-selectin in children with sickle cell disease. P-selectin is involved in the process that causes blood cells to stick to the walls of blood vessels, which can lead to complications in sickle cell disease. By reducing the levels of P-selectin, famotidine may help improve blood flow and reduce the risk of these complications. This study aims to see how effective famotidine is in achieving this effect after 29 days of treatment.

Investigated diseases:

Sickle cell disease

Sickle Cell Disease – Sickle Cell Disease is a genetic blood disorder characterized by the production of abnormal hemoglobin, known as hemoglobin S. This abnormal hemoglobin causes red blood cells to become rigid, sticky, and shaped like a crescent or sickle. These sickle-shaped cells can block blood flow in small vessels, leading to pain and potential organ damage. Over time, the disease can cause anemia, as the sickle cells break down prematurely. The condition can also lead to episodes of pain, known as pain crises, and increase the risk of infections. The disease progresses with varying severity among individuals, often influenced by genetic and environmental factors.

Trial ID:

2022-502144-12-00

Protocol code:

APHP201133

NCT ID:

NCT05084521

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on the Effect of Famotidine in Reducing P-selectin Levels in Children with Sickle Cell Disease

What is this study about?

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