EUCTR2017-000540-18-DE进行中（未招募）1 期
Phase 3b, Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Assess the Safety, Efficacy, and Tolerability of Tezacaftor/Ivacaftor (TEZ/IVA) in an Orkambi-experienced Population Who Are Homozygous for the F508del-CFTR Mutation
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 98
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
入选标准
- •1. Subject (or subject’s legally appointed and authorized representative) will sign and date an informed consent form (ICF) and, where appropriate, an assent form.
- •2. Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures.
- •3. Males or females, aged 12 years or older on the date of informed consent or, where appropriate, date of assent.
- •4. Prior discontinuation of Orkambi, with at least 1 respiratory sign or symptom considered related to therapy, including but not limited to the following:
- •Chest discomfort
- •Dyspnea (shortness of breath)
- •Respiration abnormal (chest tightness)
- •Bronchial hyperreactivity
- •Bronchospasm
- •Asymptomatic reduction in relative change in ppFEV1 of >12% within 2 weeks after Orkambi initiation
- •Discontinuation from Orkambi should primarily be due to a respiratory event. However, concomitant non-respiratory events will not exclude subjects from participation. Documentation of specific qualifying signs or symptoms is not required. Investigator attestation will be accepted if information is not present in source materials.
- •5. Resolution or stabilization of qualifying event(s) >28 days prior to Screening.
- •6. Discontinuation of Orkambi therapy must have occurred within approximately 12 weeks from the first dose of Orkambi. In the event that the subject reinitiated Orkambi, discontinuation of Orkambi therapy must have occurred within approximately 12 weeks from the time of the most recent initiation. Investigator attestation will be accepted if information is not present in source materials.
- •7. Homozygous for F508del as documented in the subject’s medical record. If genotype documentation is not available in the medical record, genotyping will be performed during screening. If the screening genotype result is not received by the end of the Screening Period and all other eligibility criteria have been met, the subject may be randomized. Note: Subjects who have been randomized and whose screening genotype does not confirm study eligibility must be discontinued from the study as described in Section 9.9.
- •8. FEV1 =25% and =90% of predicted normal for age, sex, and height (equations of Wang et al. or Hankinson et al. 12, 13) at Screening Visit (Section 11.3.1). Spirometry measurements must meet American Thoracic Society (ATS)/European Respiratory Society (ERS) criteria for acceptability and repeatability (Section 11.3.1).
- •9. Stable CF disease as judged by the investigator.
- •10. Willing to remain on a stable CF medication regimen from screening through the Safety Follow-up Contact.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 18
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 72
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Subjects who meet any of the following exclusion criteria will not be eligible for this study:
- •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
- •Examples of such comorbidities may include, but are not limited to:
- •Respiratory:
- •o Massive hemoptysis within the last 12 months
- •o Any of the following within the past 12 months and not associated with an acute, resolved event:
- •- Six-minute walk test distance <400 m
- •- Resting arterial blood gas on room air showing PaCO2 >50 mm Hg or PaO2 <55 mm Hg
- •- Systolic pulmonary arterial pressure (PAP) >35 mm Hg on echocardiography or a mean PAP >25 mm Hg measured by right heart catheterization, in the absence of a hypoxemic exacerbation or with an alternate etiology to explain the findings
- •Non-respiratory: history of cirrhosis with portal hypertension, history of and/or risk factors for ventricular arrhythmia (e.g., long QT syndrome, hypokalemia, heart failure, left ventricular hypertrophy, bradycardia, myocardial infarction, cardiomyopathy, morbid obesity, acute neurologic events [subarachnoid hemorrhage, intracranial hemorrhage, cerebrovascular accident, and intracranial trauma], autonomic neuropathy, and significant anemia)
- •2. Recent rapid or progressive deterioration in respiratory status
- •3. Receiving continuous oxygen at >2L/min or on face-mask ventilation
- •4. Any of the following abnormal laboratory values at Screening:
- •Abnormal liver function defined as any 2 or more of the following: =3 × upper limit of normal (ULN) aspartate transferase (AST), =3 × ULN alanine transferase (ALT), =3 × ULN gamma-glutamyl transpeptidase, =3 × ULN alkaline phosphatase (ALP), or =2 × ULN total bilirubin.
- •Abnormal liver function defined as any increase of =5 × ULN AST or ALT.
- •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) for subjects =18 years of age and =45 mL/min/1.73 m2 (calculated by the Counahan-Barratt equation) for subjects aged 12 to 17 years (inclusive).
- •5. Child-Pugh Class B or C hepatic impairment
- •6. An acute upper or lower respiratory infection, pulmonary exacerbation, or change in therapy (including antibiotics) for pulmonary disease within 28 days before Day 1 (first dose of study drug)
- •7. Documentation of colonization with organisms associated with a more rapid decline in pulmonary status (e.g. Burkholderia cenocepacia, Burkholderia dolosa, and Mycobacterium abscessus)
- •8. History of lung transplantation since most recent initiation of Orkambi
- •9. History of alcohol or drug abuse in the past year as deemed by the investigator, including but not limited to cannabis, cocaine, and opiates
- •10. Participation in an investigational drug study or use of a CFTR modulator (including Orkambi) within 28 days or 5 terminal half-lives before screening of the previous investigational study drug or CFTR modulator, whichever is longer
- •Ongoing participation in a noninterventional study (includi
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