Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

Not recruitingRare disease

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

Verified siteRegistered drugNo placebo
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

A plain-language summary of the goals, design and what participants do

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.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 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. Step 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. Step 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. Step 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 trial?

6 criteria

  • 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 trial?

3 criteria

  • 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.
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

Not recruiting
Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

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.

What is already known about the treatment

Famotidine – Famotidine is administered orally in the form of tablets. It is currently being studied for its potential benefits in reducing the expression of P-selectin, a molecule involved in inflammation, in children with Sickle Cell Disease. Famotidine is primarily used to treat conditions related to excess stomach acid, such as ulcers and gastroesophageal reflux disease (GERD). It works by blocking histamine receptors in the stomach lining, which reduces acid production. Famotidine belongs to a class of drugs known as H2 blockers.

Investigated diseases

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 detailsLast updated 2 Oct 2026
Age0-17PhasePhase IIITrial ID2022-502144-12-00Protocol codeAPHP201133Estimated enrolment30 patientsSponsorAssistance Publique Hopitaux De Paris

sourced from the EU Clinical Trials Register and site verification

Want to learn more about this trial or check if you can participate?

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.