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Study on Improving Treatment for Sickle Cell Disease Patients Using Hydroxycarbamide

Verified siteRegistered drugNo placebo
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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 management of patients with sickle cell disease, a genetic condition that affects the shape and function of red blood cells. The treatment being investigated is hydroxyurea, a medication that helps to reduce the frequency of painful episodes and other complications associated with sickle cell disease. The study involves different forms of hydroxyurea, including HYDREA 500 mg capsules and Siklos film-coated tablets in various dosages.

The purpose of the study is to optimize the treatment of sickle cell disease by comparing two different methods of monitoring patients who are taking hydroxyurea. Participants will be divided into two groups, each receiving a different approach to therapeutic follow-up. The study will observe how quickly patients reach a stable treatment level and will monitor various health parameters, such as the number of painful episodes, hospitalizations, and any side effects related to the medication.

Throughout the study, participants will take hydroxyurea orally, and their progress will be tracked over a period of time. The study aims to provide insights into the best ways to manage sickle cell disease with hydroxyurea, potentially improving the quality of life for those affected by this condition. The trial is expected to continue until 2025, with the goal of gathering comprehensive data on the effectiveness and safety of hydroxyurea in treating sickle cell disease.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, the patient will be informed about the objectives and potential risks associated with the research. The patient or their legal guardian must provide informed consent, which includes understanding the study's purpose and agreeing to participate.

  2. Step 2

    Initial assessment

    An initial medical examination will be conducted to confirm eligibility. This includes verifying the sickle cell genotype (HbSS) and ensuring the patient has been hospitalized for a vaso-occlusive crisis in the last three months or requires treatment intensification.

  3. Step 3

    Medication administration

    The patient will begin treatment with hydroxycarbamide, which is administered orally. The specific product used may vary, including options such as HYDREA 500 mg capsules or Siklos film-coated tablets in 100 mg or 1,000 mg dosages. The dosage will be determined based on the patient's needs and medical condition.

  4. Step 4

    Monitoring and follow-up

    Throughout the study, the patient's response to the medication will be closely monitored. This includes regular assessments of clinical efficacy, such as the number of vaso-occlusive seizures and any complications related to sickle cell disease or hydroxycarbamide.

    Biological parameters will also be evaluated, including blood counts, renal and liver function tests, and the percentage of fetal hemoglobin (HbF).

  5. Step 5

    Pharmacokinetic analysis

    The study will involve pharmacokinetic analysis to understand how the drug is processed in the body. This includes measuring drug concentration over time and assessing factors like renal function, age, weight, and body surface area to predict individual dosage needs.

  6. Step 6

    Completion of the study

    The study is expected to conclude by August 7, 2025. Upon completion, the data collected will be analyzed to determine the effectiveness and safety of the treatment regimen.

Who can join the trial?

12 criteria

  • Age between 2 and 35 years.
  • Able to understand the study's goals and risks and provide signed consent.
  • Signed consent from the patient and/or their legal guardians if the patient is a minor.
  • Has a specific type of sickle cell disease called HbSS.
  • Hospitalized for a vaso-occlusive crisis (a painful episode) in the last 3 months and needs treatment adjustment.
  • For women who can have children:
    • Negative pregnancy test at the start of the study.
    • Agree to use effective birth control during the study and for 182 days after.
    • For men who can have children:
      • Agree to use effective birth control during the study and for 92 days after.
      • Starting or needing more intense treatment for sickle cell disease.
      • Currently hospitalized or has unbalanced treatment for sickle cell disease.
      • Has health insurance or is covered by a social protection scheme.
      • Informed about the results of a prior medical examination.
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Investigated drugs

Hydroxyurea is a medication used in the treatment of sickle cell disease, also known as drepanocytosis. It works by helping to reduce the frequency of painful crises and the need for blood transfusions. In this clinical trial, hydroxyurea is being studied to optimize its management in patients, focusing on how therapeutic follow-up can improve treatment outcomes.

What is already known about the treatment

Hydroxyurea – This medication is typically administered orally in the form of capsules or tablets. It is currently well-established in medical literature and is used primarily for the treatment of drepanocytosis, also known as sickle cell disease. Hydroxyurea works by increasing fetal hemoglobin production, which helps to reduce the sickling of red blood cells and improve blood flow. It is classified as an antineoplastic agent, although in this context, it is used for its ability to modify the disease process rather than for cancer treatment.

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 causes red blood cells to become rigid, sticky, and shaped like sickles or crescent moons. These misshapen cells can block blood flow, leading to pain episodes called vaso-occlusive crises. Over time, the disease can cause damage to organs and increase the risk of infections. Patients may experience anemia due to the rapid breakdown of the sickle cells. The severity and frequency of symptoms can vary widely among individuals.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-515562-14-00Estimated enrolment30 patientsSponsorLes Hopitaux Universitaires De Strasbourg

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