Optimizing Hydroxyurea Management for Sickle Cell Patients
Optimizing the Management of Sickle Cell Patients on Hydroxyurea: The Value of Therapeutic Pharmacological Monitoring
NA · University Hospital, Strasbourg, France · NCT06464458
This study is testing how better management of hydroxyurea treatment can help young people with sickle cell disease have fewer painful crises and complications.
Quick facts
| Phase | NA |
|---|---|
| Study type | Interventional |
| Enrollment | 30 (estimated) |
| Ages | 2 Years to 35 Years |
| Sex | All |
| Sponsor | University Hospital, Strasbourg, France (other) |
| Drugs / interventions | chemotherapy |
| Locations | 1 site (Strasbourg) |
| Trial ID | NCT06464458 on ClinicalTrials.gov |
What this trial studies
This clinical trial focuses on improving the management of patients with sickle cell disease through therapeutic pharmacological monitoring of hydroxyurea treatment. It aims to assess the effectiveness of hydroxyurea in reducing vaso-occlusive crises and other complications associated with the disease. Patients aged 2 to 35 years who have experienced recent hospitalizations for vaso-occlusive crises will be monitored to ensure optimal dosing and treatment efficacy. The study involves obtaining informed consent from participants and their guardians, with a structured approach to patient education and engagement.
Who should consider this trial
Good fit: Ideal candidates are individuals aged 2 to 35 years with severe sickle cell disease who have been hospitalized for vaso-occlusive crises within the last three months.
Not a fit: Patients with sickle cell genotypes other than HbSS or those who do not require intensification of hydroxyurea treatment may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to better management strategies for sickle cell disease, reducing complications and improving patient quality of life.
How similar studies have performed: Other studies have shown promising results with therapeutic monitoring of hydroxyurea in sickle cell disease, indicating that this approach may be beneficial.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion criteria: * Subjects aged between 2 and 35 years. * For very young children hydroxyurea is only initiated in cases of severe sickle cell disease. * Sickle cell genotype: HbSS * Subjects who have been hospitalised for CVO in the last 3 months and for whom HU treatment is to be initiated and/or whose treatment is not balanced or is less than 30 mg/kg, regardless of the length of treatment * For a woman of childbearing potential: * Negative blood pregnancy test at the inclusion visit * Patient accepting highly effective contraception for the duration of study participation and 182 days after discontinuation of the study or treatment. * Initiation of HU treatment in a patient requiring intensification of therapy in the context of sickle cell disease * Patient hospitalised (e.g. vaso-occlusive crisis) and/or whose HU treatment is not balanced (MTD not reached) * Informed consent signed, as appropriate, by : * The patient and/or * The holder(s) of parental authority and the minor subject if capable of discernment * Subject affiliated to a social health insurance scheme or beneficiary * Subject who has been informed of the results of the prior medical examination, and/or of whom the holder(s) of parental authority has (have) been informed * Subject able to understand the objectives and risks of the research and to give dated and signed informed consent Exclusion criteria: * HU patient who has achieved MTD (based on haematological criteria) or is not therapeutically ineffective or hydroxyurea dosage \> 35 mg/kg/day. * Refusal to accept the use of a highly effective contraceptive method as defined during HU treatment and for 182 days for females and 92 days for males following such treatment (fertile patients only) * Patient with a parental plan within 18 months * Hypersensitivity to the active substance or to any of the excipients of the medicinal product * Severe hepatic impairment * Severe renal insufficiency * Toxic signs of myelosuppression * Neutrophils \< 1500/mm3 * Platelets \< 80 000/mm3 * Haemoglobin \< 4.5 g/dL * Reticulocytes \< 80,000/mm3 if haemoglobin concentration \< 9 g/dL * Patient with transfusion, exchange transfusion or erythropoietin administration within 3 months of inclusion * Patient with HIV * Unable to give subject informed information (subject in emergency situation) * Inability of the subject to undergo the medical follow-up of the trial for geographical, social or psychological reasons * Subject under guardianship or curatorship * Pregnancy or breastfeeding in the case of adolescents or adults * Subject in an exclusion period (determined by a previous or ongoing study) * Concurrent inclusion in another drug study * Subject under court protection
Where this trial is running
Strasbourg
- Strasbourg University Hospital — Strasbourg, France (RECRUITING)
Study contacts
- Principal investigator: Paillard — University Hospital, Strasbourg, France
- Study coordinator: Mariem DRIDI
- Email: mariem.dridi@chru-strasbourg.fr
- Phone: +33 3 88 11 60 08
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Sickle Cell Disease, Sickle cell, Hydroxyurea, Therapeutic drug monitoring, Pharmacokinetics, SIKLOS®, HYDREA®