ESCORT-HU Extension: European Sickle Cell Disease Cohort - Hydroxyurea - Extension
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- Theravia
- 入组人数
- 2,093
- 试验地点
- 87
- 主要终点
- Occurrence and incidence of the number of malignancies, leg ulcers, male fertility impairment and serious unexpected AEs causally related to Siklos®
研究概览
简要总结
As safety information pertaining to the long-term use of HU remains incomplete in spite of the first safety study (ESCORT-HU), an extension of the latter is proposed. ESCORT-HU Extension study aims at evaluating the long-term safety of Siklos® focusing on some questions regarding its safety when used in current practice in adults and paediatric patients treated with Siklos® and followed for up to 5 years. The study will focus on the following concerns : occurrence and incidence of malignancies, leg ulcers, male fertility impairment and serious unexpected AEs causally related to Siklos®.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female patients with symptomatic SCD,
- •≥ 2 years old,
- •Treated with Siklos®,
- •Having been informed of the study by the investigator and consenting to participate, or whose parents or legal guardians are consenting for their child to participate.
- •To allow risk evaluation, participants must belong at least to one of the subpopulations defined below:
- •Participants previously enrolled in ESCORT-HU who agree to participate in ESCORT-HU Extension study,
- •New participants with any of the following criteria:
- •history of HU treatment for more than 5 years or
- •prepubescent over 10 years of age for girls and 13 years of age for boys at enrolment, or
- •with history of leg ulcer, or
- •pregnant women without interruption of Siklos® 3 months before the beginning of the pregnancy or,
- •males treated with Siklos® whose partner is pregnant and without discontinuation of Siklos® 3 months before the beginning of the pregnancy.
排除标准
- 未提供
研究组 & 干预措施
Sickle cell disease patients treated with Siklos
干预措施: Hydroxycarbamide (Drug)
结局指标
主要结局
Occurrence and incidence of the number of malignancies, leg ulcers, male fertility impairment and serious unexpected AEs causally related to Siklos®
时间窗: 5 years
Occurrence and incidence of the number of leg ulcers
时间窗: 5 years
Occurrence and incidence of male fertility impairment
时间窗: 5 years
Occurrence and incidence of serious unexpected AEs causally related to Siklos®
时间窗: 5 years
次要结局
- Mean age of puberty(5 years)
- Mean number of patients with the use of a contraception, incidence of the use of semen cryopreservation and of semen analysis performed(5 years)
- Frequency and reason for temporary or permanent discontinuations of Siklos®(5 years)
- Number of switch and combination with alternative treatment(5 years)
- Measure of quality of life with SF12 questionnaire(5 years)
- Incidence of the use of semen cryopreservation and of semen analysis performed(5 years)
- Incidence of semen analysis performed(5 years)
- Management of Siklos® during pregnancy and outcome of pregnancy following exposure at the time of conception or at any time during the pregnancy(5 years)