NCT03806452已完成2 期
Multicentre Randomized Double-blind Placebo-controlled Study to Evaluate the Effect on Albuminuria of 6 Months Treatment With Hydroxycarbamide (Siklos®) or a Placebo in Adults With Sickle Cell Disease:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Theravia
- 入组人数
- 86
- 试验地点
- 21
- 主要终点
- Number of Patients Achieving at Least a 30% Decrease in ACR Baseline Value
研究概览
简要总结
The purpose of this phase IIb, international, multicentre, double-blind, randomised, placebo-controlled study is to determine the effect of hydroxycarbamide on albuminuria after 6 months of treatment in SCD adult patients.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed and dated Informed Consent Form (ICF) by a legally competent patient.
- •Patients above 18 years.
- •Patients with HbSS or HbSβ0 SCD.
- •Patients with a value of albuminuria, assessed by ACR, over 3 mg/mmol and inferior to 100 mg/mmol confirmed by 3 positive urine samples taken one day apart.
- •Female patients of childbearing potential or postmenopausal female with last period < 12 months before screening agreeing to use a highly effective form of contraception (oral, injected or implanted hormonal contraception, intrauterine device, diaphragm, condom) during the trial and for 3 months after hydroxycarbamide discontinuation.
- •Male patients with partners of childbearing potential agreeing to use a highly effective contraception during the trial and for 3 months after hydroxycarbamide discontinuation. Men with pregnant or lactating women should be advised to use a barrier method of contraception (condom) to prevent the foetus or breastfed infant from exposure to hydroxycarbamide.
- •Patients who are covered by insurance scheme according to local regulatory requierements.
排除标准
- •Patients who had severe VOC requiring hospitalisation or ACS within the last 4 weeks preceding screening visit.
- •Patients treated with hydroxycarbamide for any reason within the previous 6 months.
- •Patients who have had chronic blood transfusion or transfusion in the last 3 months.
- •Patients with a history of hypertension (systolic blood pressure ≥ 140 or diastolic blood pressure ≥ 90 mmHg) treated with antihypertensive agent belonging to pharmacological class of RAS inhibitor.
- •Patients who have symptoms suggestive of urinary tract infection or patients with gross haematuria.
- •Patients with a concomitant primary kidney disease.
- •Patients with any systemic condition that could result in a glomerulopathy not related to SCD (e.g. diabetes mellitus, active hepatitis B or C infections, HIV infection, systemic lupus erythematosus, inflammatory arthropathies).
- •Patient with a stage 3, 4 or 5 chronic kidney disease (eGFR < 60 mL/min per 1.73 m2).
- •Patients with eGFR ≥ 140 ml/min/1,73m² due to the lack of information regarding the magnitude, direction and significance of the trends in eGFR evolution that could be expected in this population
- •Patients requiring long-term treatment with drugs potentially nephrotoxic (see non-exhaustive list).
- •Patients requiring ACE inhibitors or ARBs within the 3 months before inclusion regardless of the indication.
- •Patients requiring long-term treatment with non-steroid anti-inflammatory drugs.
- •Patients who have a treatment which can modify the kidney function (see non-exhaustive list) in the last 3 months.
- •Patients known to be infected with HIV.
- •Female patients who are pregnant or lactating.
- •Unreliable patients including non-compliant patients, patients with known alcoholism or drug abuse or with a history of a serious psychiatric disorder as well as patients unwilling to give informed consent or to abide by the requirements of the protocol.
- •Simultaneous participation in other clinical trials on an investigational medicinal product or previous participation within 30 days before inclusion.
- •Persons in detention by judicial or administrative decision.
- •Patients with chronic conditions that upon investigator judgment may lead to a limited life expectancy
研究组 & 干预措施
Hydroxycarbamide
Experimental
Hydroxycarbamide will be supplied as 100 mg or 1000 mg film-coated tablets. The posology will be based on the patient's body weight (bw). Hydroxycarbamide will be prescribed at a dose of 15 mg/kg bw/day and will be adminitered for 6 months.
Hydroxycarbamide will be administered for 12 months for patients qualified as responders willing to continue to participate in the trial.
干预措施: Hydroxycarbamide (Drug)
Placebo
Placebo Comparator
Placebo will be supplied as 100 mg or 1000 mg film-coated tablets. The posology will be based on the patient's body weight (bw). Placebo will be prescribed at a dose of 15 mg/kg bw/day and will be adminitered for 6 months.
Hydroxycarbamide will be administered for 12 months for patients qualified as responders willing to continue to participate in the trial.
干预措施: Placebo Oral Tablet (Drug)
结局指标
主要结局
Number of Patients Achieving at Least a 30% Decrease in ACR Baseline Value
时间窗: 6 months
The primary endpoint of this study is the proportion of patients in the hydroxycarbamide and placebo groups achieving at least a 30% decrease in ACR baseline value at 6 months after treatment initiation. Patients who do not achieve at least a 30% decrease of the ACR baseline value at month 6 will be considered non-responders.
次要结局
- Absolute Mean Changes in eGFR Value(6 months)
- Absolute Mean Changes in ACR Value(6 months)
- Proportion of Patients With a Shift From Macroalbuminuria to Microalbuminuria(6 months)
- Proportion of Patients With a Shift From Microalbuminuria to Normoalbuminuria(6 months)
- Proportion of Patients With a Shift From Macroalbuminuria to Normoalbuminuria(6 months)
- Proportion of Patients With a Shift From Microalbuminuria to Macroalbuminuria(6 months)
- Absolute Mean Changes of Systolic Blood Pressure(6 months)
- Absolute Mean Changes of Body Weight(6 months)
- Absolute Mean Changes of Diastolic Blood Pressure(6 months)
- Absolute Mean Changes of Heart Rate Measure(6 months)
- Absolute Mean Changes in White Blood Cells Count(6 months)
- Absolute Mean Changes in Platelets Count(6 months)
- Absolute Mean Changes in Mean Corpuscular Volume(6 months)
- Absolute Mean Changes in Mean Corpuscular Haemoglobin Concentration(6 months)
- Absolute Mean Changes in Mean Corpuscular Haemoglobin(6 months)
- Absolute Mean Changes in Hemoglobin Count(6 months)
- Absolute Mean Changes in Foetal Hemoglobin Count(6 months)
- Absolute Mean Changes in Free Hemoglobin Count(6 months and 12 months for responder patients willing to continue the study after month 6.)
- Absolute Mean Changes in Dense Red Blood Cells Percentage(6 months and 12 months for responder patients willing to continue the study after month 6.)
- Absolute Mean Changes in Endogenous Erythropoietin Count(6 months)
- Absolute Mean Changes in Ferritin Count(6 months)
- Absolute Mean Changes in Lactate Dehydrogenase(6 months)
- Absolute Mean Changes in Aspartate Aminotransferase(6 months)
- Absolute Mean Changes in Alanine Amino Transferase(6 months)
- Absolute Mean Changes in Blood Urea Nitrogen(6 months)
- Absolute Mean Changes in Conjugated Bilirubin(6 months)
- Absolute Mean Changes in Total Bilirubin(6 months)
- Absolute Mean Changes in Reticulocytes(6 months)
研究者
研究点 (21)
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