NL-OMON47983撤回2 期
A Phase 2, Randomized, Double-blind Study to Evaluate the Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis - A Study to Evaluate Efficacy and Safety of VX-561
适应症
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 入组人数
- 2
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 至 99（—）
入选标准
- •1. Subject will sign and date an informed consent form (ICF)., 2. Willing and
- •able to comply with scheduled visits, treatment plan, study restrictions,
- •laboratory tests, contraceptive guidelines, and other study procedures., 3.
- •Subjects (males and females) aged 18 years or older on the date of informed
- •consent., 4. Receiving IVA treatment with no interruptions for at least 28 days
- •before screening., 5. Tolerating IVA therapy as judged by the investigator., 6.
- •Female subjects must have a negative pregnancy test at Screening., 7. Body
- •weight *35 kg., 8. Subjects must be able to produce a valid
- •(quantity-sufficient) sweat sample at screening. If the initial screening
- •collection results in insufficient sweat volume, then the sweat chloride
- •collection may be repeated., 9. Confirmed diagnosis of CF as determined by the
- •investigator., 10. Has 1 of the following mutations in their CF gene: G551D,
- •G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R. If the screening
- •CFTR genotype result is not received before randomization, a previous CFTR
- •genotype laboratory report may be used to establish eligibility. Subjects who
- •have been enrolled and whose screening genotype does not confirm study
- •eligibility must be discontinued from the study., 11. Subjects must have a
- •forced expiratory volume in 1 second (FEV1) *40% and *100% of predicted normal
- •for age, sex, and height (equations of the Global Lung Function Initiative
- •[GLI])8 at the Screening Visit. Spirometry measurements must meet American
- •Thoracic Society/European Respiratory Society criteria9 for acceptability and
- •repeatability., 12. Stable CF disease as judged by the investigator., 13.
- •Willing to remain on a stable CF treatment regimen (other than
- •protocol-specified changes in CFTR modulator regimen) through the Safety
- •Follow-up Visit.
排除标准
- •1. History of any comorbidity 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., 2. History of clinically significant cirrhosis with
- •or without portal hypertension., 3. Any of the following abnormal laboratory
- •values at screening:, * Hemoglobin <10 g/dL, * Total bilirubin *2 × upper
- •limit of normal (ULN), * Aspartate transaminase (AST), alanine transaminase
- •(ALT), gamma-glutamyl transferase (GGT), or alkaline phosphatase (ALP) *3 ×
- •ULN, * Abnormal renal function defined as glomerular filtration rate *50
- •mL/min/1.73 m2 (calculated by the Modification of Diet in Renal Disease Study
- •Equation)10,11 for subjects *18 years of age, 4. An acute upper or lower
- •respiratory infection, pulmonary exacerbation, or changes in therapy (including
- •antibiotics) for sinopulmonary disease within 28 days before the first dose of
- •study drug., 5. Lung infection with organisms associated with a more rapid
- •decline in pulmonary status (e.g., Burkholderia cenocepacia, Burkholderia
- •dolosa, and Mycobacterium abscessus). For subjects who have had a history of a
- •positive culture, the investigator will apply the following criteria to
- •establish whether the subject is free of infection with such organisms:, * The
- •subject has not had a respiratory tract culture positive for these organisms
- •within the 12 months before the date of informed consent., * The subject has
- •had at least 2 respiratory tract cultures negative for such organisms within
- •the 12 months before the date of informed consent, with the first and last of
- •these separated by at least 3 months, and the most recent 1 within the 6 months
- •before the date of informed consent., 6. An acute illness not related to CF
- •(e.g., gastroenteritis) within 14 days before the first dose of study drug., 7.
- •Standard 12-lead ECG demonstrating QTcF >450 msec at screening. If QTcF
- •exceeds 450 msec, the ECG will be repeated 2 more times, and the average of the
- •3 QTcF values will be used to determine the subject*s eligibility., 8. History
- •of solid organ or hematological transplantation., 9. History of alcohol or drug
- •abuse in the past year, including, but not limited to, cannabis, cocaine, and
- •opiates, as deemed by the investigator., 10. Ongoing or prior participation in
- •a study of an investigational treatment with the exception of the following:, *
- •Ongoing or prior participation in an investigational study of a Vertex CFTR
- •modulator. A washout period of 28 days must elapse before Day 1., * For
- •prospective subjects with ongoing or prior participation in all other
- •interventional studies, a washout period of 28 days or 5 terminal half-lives
- •(whichever is longer) must elapse before screening. The duration of the elapsed
- •time may be longer if required by local regulations., * Ongoing participation
- •in a noninterventional study (including observational studies and studies
- •requiring assessments without administration of study drug or assignment to
- •other interventions) is permitted., 11. Use of prohibited medications as
- •defined in the protocol, within the specified window before the first dose of
- •study drug., 12. Pregnant or nursing female subjects., 13. Subject, or close
- •relative of the subject, is the investigator or a subinvestigator, research
- •assistant, pharmac
研究者
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