#1 Clinical Trials Search in Europe

Study on Hydroxycarbamide Tablets for Children with Sickle Cell Disease

2 1 1

What is this study about?

This clinical trial is focused on studying Sickle Cell Disease in children. The treatment being tested is called Hydroxycarbamide, which is provided in the form of SIKLOS PAEDIATRIC 50mg dispersible tablets. The purpose of the study is to understand how this medication behaves in the body when given twice a day to children aged 9 months to 11 years.

During the study, children will receive the medication for up to 12 months. The study will monitor how the medication is absorbed and processed in the body over time. This includes checking the levels of the medication in the blood at different times and observing any changes in the children’s health. The study will also look at how the medication affects certain blood components, such as hemoglobin, which is important for carrying oxygen in the blood.

Throughout the study, researchers will keep track of any health events related to Sickle Cell Disease and any side effects that might occur. The study aims to gather information on how well children tolerate the medication and how easy it is for them and their caregivers to use the treatment. This information will help in understanding the safety and effectiveness of the medication for children with Sickle Cell Disease.

1 introduction to the study

Upon joining the study, the child will begin taking Siklos Paediatric 50mg dispersible tablets.

The medication is taken orally, twice daily, as part of the study’s dosing regimen.

2 initial treatment phase

The primary goal during this phase is to monitor how the body processes the medication.

Measurements will be taken at 1, 3, 6, 9, and 12 months to assess the medication’s concentration in the blood.

3 monitoring and assessment

Throughout the study, changes in blood parameters such as white blood cells and hemoglobin levels will be recorded.

The child’s response to the medication, including any side effects, will be closely monitored.

4 acceptability and compliance evaluation

At 3 months, the child and their parent or representative will evaluate the medication’s acceptability using a scale.

The ease of using the administration kit will also be assessed.

5 ongoing treatment and follow-up

The child will continue taking the medication for up to 12 months, with regular follow-up visits to ensure compliance and monitor health.

Any events related to sickle cell disease or adverse effects will be documented.

Who Can Join the Study?

  • Both parents or a legal representative must sign a written informed consent form, and if possible, the child should also agree to participate.
  • The child must have a specific type of Sickle Cell Disease known as HbSS or HbSβ0.
  • The child must be between 9 months and 11 years old.
  • The child should not have had any previous or current treatment with HC (Hydroxyurea).
  • The parents or legal representative must be able to communicate with the study team, understand the study requirements, and agree to follow them.
  • If applicable, sexually active female patients who can have children must agree to use a highly effective form of birth control during the study and for 3 months after stopping HC. Male patients with partners who can have children must also agree to use effective birth control during the study and for 3 months after stopping HC. Male patients with pregnant or breastfeeding partners should use a condom to prevent exposure to HC.
  • The child must be covered by a social security plan or a similar insurance plan.
  • The child must have certain laboratory values: Absolute Neutrophil Count of at least 1.0×109/L, Platelets of at least 75×109/L, and Haemoglobin (Hgb) greater than 5.5 g/dL.
  • For children over 18 months old, a Transcranial Doppler (TCD) test in the last 12 months must show a low risk for stroke.

Who Cannot Join the Study?

  • Children younger than 9 months or older than 11 years cannot participate.
  • Participants who do not have Sickle Cell Disease are not eligible. Sickle Cell Disease is a condition where red blood cells, which carry oxygen, are shaped like a sickle or crescent, causing blockages in blood flow.
  • Participants who are not able to take the study medication as prescribed, which is twice a day, are excluded.
  • Participants who are not part of the vulnerable population selected for the study cannot join. A vulnerable population might include groups like children or those with certain health conditions.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Centre Hospitalier Intercommunal Creteil Creteil France
Hopital Necker Enfants Malades Paris France

Other Sites

Site Name City Country Status
Centre Hospitalier De Cayenne Cayenne France
Grand Hopital De L Est Francilien Meaux France
Centre Hospitalier Lyon Sud Pierre Benite France
Crv Klslgld Bchnpie Le Kremlin-Bicetre France

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
France France
Recruiting
01.09.2024

Trial locations

Investigated drugs:

Siklos® Paediatric Dispersible Tablets are used in this clinical trial to study their effects in children with Sickle Cell Disease. These tablets are designed to be dissolved in water, making them easier for children to take. The trial aims to understand how the medication is processed in the body and to evaluate its effectiveness and safety when given twice a day to children aged 9 months to 11 years.

Investigated diseases:

Sickle Cell Disease – Sickle Cell Disease is a genetic disorder that affects the red blood cells, causing them to become rigid and shaped like a sickle or crescent. These abnormally shaped cells can block blood flow in small blood vessels, leading to pain and potential organ damage. The disease is characterized by episodes of pain, known as sickle cell crises, which can vary in frequency and severity. Over time, the repeated blockages can lead to complications such as anemia, infections, and damage to organs like the spleen and liver. The condition is present from birth and is inherited when a child receives two sickle cell genes, one from each parent. It primarily affects individuals of African, Mediterranean, Middle Eastern, and Indian ancestry.

Trial ID:
2024-513750-30-00
Protocol code:
SIK-FR-24-1
Trial Phase:
Therapeutic exploratory (Phase II)

Other Trials to Consider

  • A study testing gene therapy with Dream01 and imatinib for patients with severe sickle cell disease to improve red blood cell production

    Recruiting

    2 1 1 1
    Investigated diseases:
    Investigated drugs:
    France

  • Study on Rilzabrutinib for Patients Aged 10-65 with Sickle Cell Disease

    Recruiting

    3 1
    Investigated drugs:
    Belgium France Germany Greece Italy The Netherlands +1