EUCTR2011-003821-93-DE进行中（未招募）1 期
A Phase 2, Multicenter, Double Blinded, Placebo Controlled Study to Evaluate Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX-661 Monotherapy and VX-661/Ivacaftor Cotherapy in Subjects with Cystic Fibrosis, Homozygous or Heterozygous for the F508del CFTR Mutation
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 220
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •-For Groups 1 through 8: Male or female with confirmed diagnosis of CF, defined as:
- •a sweat chloride value 60 mmol/L by quantitative pilocarpine iontophoresis
- •OR 2 CF-causing mutations (all as documented in the subject’s medical record)
- •chronic sinopulmonary disease
- •OR gastrointestinal/nutritional abnormalities.
- •-For Groups 1 through 6: homozygous subjects must have the F508del-CFTR mutation in both alleles.
- •- For Group 7: heterozygous subjects must have the F508del-CFTR mutation on 1 allele, and the second CFTR allele must encode a mutation predicted to result in the gating mutation G551D in the CFTR gene. The CFTR mutations for Group 7 must be confirmed before randomization after reviewing the report provided by the central laboratory.
- •-For group 8: heterozygous subjects must have the F508del-CFTR
- •mutation on 1 allele, and a copy of a CFTR mutation associated with
- •residual CFTR function or defective mRNA splicing on the other allele.
- •The CFTR mutations for Group 8 must be confirmed before
- •randomization after reviewing the report provided by the central
- •laboratory AND
- •Subjects must have clinical evidence of residual CFTR function based on
- •any 1 of the following:
- •- Fecal elastase-1 >200 µg/g stool at screening
- •- Sweat chloride =80 mmol/L at screening
- •- =12 years of age at the time of diagnosis
- •The following inclusion criteria are applicable to Groups 1 through 8.
- •-FEV1 40% to 90% (inclusive) of predicted normal for age, gender, and height (Knudson standards ) at screening.
- •-If sexually active, male subjects who can father a child and female subjects of child bearing potential must meet the contraception requirements.
- •- For Groups 1 through 6 and Group 8: Eighteen years of age or older and able to read, understand, sign, and date a written informed consent form (ICF) before study participation at screening
- •For Group 7: Twelve of age or older on the date of signed ICF, and where appropriate, date of assent.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 5
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 215
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •-History of any illness that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject.
- •-An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before Study Day 1.
- •-Pregnant, breast-feeding, or not willing to follow contraception requirements.
研究者
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