ACTRN12619001347156招募中1 期
Orkambi in Patients with Cystic Fibrosis and Severe Liver Disease
Dr Adeline Lim0 个研究点目标入组 30 人开始时间: 2019年10月1日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 30
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 分配方式
- Non-randomised trial
- 主要目的
- Treatment
- 盲法
- Open (masking not used)
入排标准
- 年龄范围
- 2 Years 至 18 Years（—）
- 性别
- All
入选标准
- •1. Children between 6 years old and 18 years of age homozygous for Phe508del-CFTR
- •2. Severe cystic fibrosis related liver disease
- •1. Children between 2 years old and 18 years of age homozygous for Phe508del-CFTR
- •2. Severe cystic fibrosis related liver disease
- •Patients without severe cystic fibrosis related liver disease
- •1. Children between 2 years old and 18 years of age homozygous for Phe508del-CFTR
- •2. On full dose Orkambi for at least two weeks prior to enrolment to the study
排除标准
- •Cystic fibrosis without severe liver disease
- •Exclusion criteria - presence of cirrhosis and portal hypertension
- •These patients must already be receiving full dose Orkambi for at least two weeks prior to enrolment to this group.
研究者
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