2024-515562-14-00招募中4 期
Optimization of the management of drepanocytosis patients treated with hydroxyurea: Interest of the pharmacological therapeutic follow-up
相关药物
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Proportion of subjects with a time to reach LMA of less than 9 months
研究概览
简要总结
Compare the time to reach DMT in 2 groups of patients each with a different methodology of therapeutic follow-up
入排标准
- 年龄范围
- 0 years 至 64 years（18-64 Years, 0-17 Years）
- 接受健康志愿者
- 否
入选标准
- •Subject of age between 2 and 35 years.
- •Subject able to understand the objectives and risks related to the research and to give dated and signed informed conse
- •Informed consent signed as the case may be, by: o the patient and/or o the holder(s) of parental authority and the minor subject if he is capable of discernment
- •Sickle cell genotype: HbSS
- •Subject who has been hospitalized for CVO in the last 3 months in whom hu treatment is to be initiated and / or whose treatment is not balanced or less than 30 mg / kg regardless of the age of treatment
- •For a woman of childbearing age: o Negative blood pregnancy test at inclusion visit o Patient accepting highly effective contraception for the duration of participation in the study and 182 days after discontinuation of the study or treatment for a woman. The contraceptives considered highly effective are: ▪ Combined hormonal contraception (containing estrogen and progesterone) associated with ovulation inhibition: oral, intravaginal, transdermal ▪ Hormonal contraception progesterone alone associated with ovulation inhibition: oral, injectable, implantable ▪ intrauterine device ▪ intrauterine device with hormone release ▪ tubal ligation
- •For men of childbearing age: patient accepting effective contraception throughout the study and for 92 days after stopping the study or treatment, use of condoms in the included patient as well as taking contraception by the partner of childbearing age.
- •Initiation of HU treatment in a patient requiring therapeutic intensification in the context of sickle cell disease
- •Hospitalized patient (e.g. vaso-occlusive crisis) and / or whose treatment with HU is unbalanced (DMT not reached)
- •Subject affiliated to a social protection scheme for health insurance or beneficiary
- •Subject who has been informed of the results of the prior medical examination, and/or whose holder(s) of parental authority has been informed(s)
排除标准
- •Patient treated with HU who has reached DMT (hematological criteria) or who does not have therapeutic ineffectiveness or hydroxyurea dosage > 350 mg / kg / day.
- •Hypersensitivity to the active substance or to any of the excipients of the drug.
- •Severe hepatic impairment.
- •Severe renal failure.
- •Toxic signs of myelosuppression o Neutrophils < 1,500/mm3 o Platelets < 80,000/mm3 o Hemoglobin < 4.5 g/dL o Reticulocytes < 80,000/mm3 if the haemoglobin concentration is < 9 g/dL
- •Patient who received a transfusion, transfusion exchanges or administration of erythropoietin within 3 months before inclusion
- •Subject in period of exclusion (determined by a previous or ongoing study)
- •HIV-positive patient
- •Inability to give informed information about (subject in emergency situation)
- •Concomitant inclusion in another drug study
- •Subject under safeguard of justice
- •Impossibility for the subject to submit to the medical follow-up of the trial for geographical, social or psychological reasons
- •Subject under guardianship or curatorship
- •Pregnancy or breastfeeding in progress for teenagers or adults
- •Refusal to agree to use a highly effective contraceptive method as defined during a HU treatment and during the 182 days for women and 92 days for men following this treatment (fertile patients only).
- •Patient with a parental project within 18 months
结局指标
主要结局
Proportion of subjects with a time to reach LMA of less than 9 months
Proportion of subjects with a time to reach LMA of less than 9 months
次要结局
- Clinical parameters of efficacy in both arms: has. a. Number of vaso-occlusive seizures b. Number of complications related to sickle cell disease and/or hydroxyurea c. Number of hospitalizations d. Time elapsed before the need for transfusion e. Percentage of HbF
- Biological parameters of toxicity in both arms: has. a. Complete blood count, reticulocytes, ferritin b. Renal function (glomerular filtration rate estimated by cystatin C, plasma creatinine and urea) c. Liver function (AST, ALT, total and conjugated bilirubin).
- Non-compliance parameters: has. a. Mean blood cell volume (MCV) b. Percentage of HbF
- Population pharmacokinetic parameters of the 2 arms: a. AUC b. Clearance and volume of distribution of the drug.
- Pharmacokinetic analysis: has. a. Pharmacokinetic modeling of the population and identification of parameters involved in the inter- and intra-individual variability of the pharmacokinetics of the HU and allowing to better predict the individual dosage adaptation from our population study: i. Renal function ii. Age iii. Weight iv. Body surface v. % HbF b.Correlation between the concentrations obtained at the different sampling times and the AUC
研究者
Paillard
Scientific
Les Hopitaux Universitaires De Strasbourg
研究点 (1)
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