Study on Improving Treatment for Sickle Cell Disease Patients Using Hydroxycarbamide

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What is this study about?

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.

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 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 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 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 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 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 Study?

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.

Who Cannot Join the Study?

Where you can join this trial?

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Site Name	City	Country	Status
Hvskqwpl Utqmuszcnbvkli Sfiyydfkxs &tvpahk Hthfltc dn Hvlctvbxzbo	STRASBOURG, Alsace	France

Trial status

Country	Status	Recruitment Start
France	Not recruiting	08.02.2023

Trial locations

Investigated drugs:

Hydroxycarbamide

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.

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 ID:

2024-515562-14-00

NCT ID:

NCT06464458

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on Improving Treatment for Sickle Cell Disease Patients Using Hydroxycarbamide

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