NCT06578507招募中2 期
Pharmacokinetics, Efficacy and Safety of Twice Daily Dosing Regimen of Hydroxycarbamide Dispersible Tablets in Children With Sickle Cell Disease: a Single-group, Non-randomised, Open-label Study (KID-BID)
适应症
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- Theravia
- 入组人数
- 50
- 试验地点
- 6
- 主要终点
- Evaluate the PK exposure for Hydroxycarbamide Paediatric dispersible tablets administered BID through area under the curve (AUC)
研究概览
简要总结
The purpose of this interventional, phase II, national, multicentric, non-randomised, open-label study is to evaluate the pharmacokinetics (PK), efficacy and safety of Hydroxycarbamide Paediatric dispersible tablets with a twice daily dosing regimen in children with Sickle Celle Disease between 9 months to 11 years of age.
Participants will:
- Take Hydroxycarbamide twice a day every day for 12 months
- Visit the clinic at screening, baseline, 1, 3, 6, 9 and 12 months
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 9 Months 至 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Written informed consent, signed and dated by both parents or by the legally acceptable representative(s) of the children, and, if possible, assent from the children,
- •HbSS or HbSβ0 SCD,
- •Aged between 9 months and 11 years old,
- •Hydroxycarbamide naïve,
- •Parent(s) or legally acceptable representative(s) capable of communicating with the investigator and understanding the requirements and constraints of the study protocol and willing to comply with the study requirements,
- •Contraception criterion, if applicable: for patients who are sexually active
- •Affiliated to a social security plan or beneficiary of a similar insurance plan,
- •Patient must meet the following laboratory values : Absolute Neutrophil Count ≥ 1.0x109/L, Platelets ≥ 75x109/L and Haemoglobin (Hgb) > 5.5 g/dL,
- •Transcranial Doppler (TCD) in the last 12 months indicating low risk for stroke is required for children over 18 months of age.
排除标准
- •Participation in any other clinical study for any other pharmaceutical product within 4 weeks preceding the inclusion visit,
- •Patients who have had chronic blood transfusion or transfusion in the last 3 months preceding the inclusion visit,
- •Patients treated with other SCD-modifying therapies,
- •Patient with a stage 3, 4 or 5 chronic kidney disease,
- •Patients known to be infected with human immunodeficiency virus, hepatitis B virus, or hepatitis C virus,
- •Known hypersensitivity or allergy to the excipients,
- •Any surgical or medical condition or any significant illness that, in the opinion of the investigator, constitutes a risk or a contraindication to the participation of the patient to the study, or that may interfere with the objectives, conduct or evaluation of the study,
- •Female patients who are pregnant or lactating,
- •Any documented history of a clinical stroke or intracranial haemorrhage, or an uninvestigated neurologic finding within the past 12 months.
研究组 & 干预措施
Hydroxycarbamide Pediatric dispersible tablet
Experimental
Hydroxycarbamide Pediatric dispersible tablet will be administered twice daily during 12 months.
干预措施: Hydroxycarbamid (Drug)
结局指标
主要结局
Evaluate the PK exposure for Hydroxycarbamide Paediatric dispersible tablets administered BID through area under the curve (AUC)
时间窗: 1, 3, 6, 9 and 12 months after treatment initiation
Evaluate the PK exposure for Hydroxycarbamide Paediatric dispersible tablets administered BID through maximum plasma concentration (Cmax)
时间窗: 1, 3, 6, 9 and 12 months after treatment initiation
Evaluate the PK exposure for Hydroxycarbamide Paediatric dispersible tablets administered BID through time to obtain the maximum concentration (Tmax)
时间窗: 1, 3, 6, 9 and 12 months after treatment initiation
次要结局
- Absolute mean change from baseline in HbF levels(Baseline, 3, 6, 9 and 12 months after treatment initiation)
- Daily AUC (AUC0-24h) at maintenance dose derived from the final PPK model(Baseline, 3, 6, 9 and 12 months after treatment initiation)
- Absolute mean change from baseline in haematological parameters(Baseline,1, 3, 6, 9 and 12 months after treatment initiation)
- Distribution of the scores related to the ease of using the administration kit for treatment administration to the child based on a 5-point Likert scale evaluated by the parent(s) or legally acceptable representative(s)(3 months after treatment initiation)
- Proportion of patients with a relative difference in Cmax ≥ 30% from BID maintenance dose relative to the one simulated on a once daily regimen giving an equivalent AUC0-24h.(Baseline, 3, 6, 9 and 12 months after treatment initiation)
- Number of SCD events occurring during the study(Baseline, 1, 3, 6, 9, 12 months after treatment initiation)
- HC plasma concentrations and HbF levels(Baseline, 3, 6, 9 and 12 months after treatment initiation)
- Acceptability score based on a 5-point Likert scale evaluated by the parent(s) or legally acceptable representative(s)(3 months after treatment initiation)
- Compliance with Hydroxycarbamide Paediatric dispersible tablets administered BID by treatment unit accountability calculated by the pharmacy (patient will bring the kits with used and unused bottles to the pharmacy at each visit)(Baseline, 1, 3, 6, 9, 12 months after treatment initiation)
- Number of adverse events (AEs) and percentage of patients reporting at least one AE(Baseline, 1, 3, 6, 9, 12 months after treatment initiation)
- Acceptability score based on a hedonic face scale evaluated by the child from 3 years old(3 months after treatment initiation)
研究者
研究点 (6)
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