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A Study Evaluating the Safety and Efficacy of VX-440 Combination Therapy in Subjects With Cystic Fibrosis
Bu çalışma, kistik fibroz (cystic fibrosis) hastalarında VX-440 ilacının TEZ ve IVA ile kombinasyon tedavisinin güvenliğini ve etkililiğini plaseboyla karşılaştırarak incelemiştir. Çalışmaya, uygun CFTR genotipine sahip (F508del için homozigot ya da F508del ve minimal fonksiyon mutasyonu için heterozigot) hastalar alınmıştır. Çalışma Faz 2 aşamasında yürütülmüş ve tamamlanmıştır.
Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.
Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.
Inclusion Criteria: * Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures. * To prevent pregnancy, female participants of childbearing potential and their male partners will be required to use pre-specified, highly effective methods of non-hormonal contraception. Male participants with female partners of childbearing potential will be required to use a condom. * Body weight ≥35 kg. * Sweat chloride value ≥60 mmol/L from test results obtained during screening. * Subjects must have an eligible CFTR genotype: * Heterozygous for F508del and a minimal function (MF) mutation known or predicted not to be responsive to TEZ and/or IVA. * Homozygous for F508del * Subjects must have an FEV1 ≥40% and ≤90% of predicted normal for age, sex, and height at the Screening Visit * Stable CF disease as judged by the investigator. * Willing to remain on a stable CF medication regimen through the planned end of treatment or, if applicable, the Safety Follow up Visit. Exclusion Criteria: * 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. * History of cirrhosis with portal hypertension. * Risk factors for Torsade de Pointes * History of hemolysis. * Glucose-6-phosphate dehydrogenase (G6PD) deficiency assessed at Screening. * Clinically significant abnormal laboratory values at screening * An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 28 days before the first dose of study drug. * Lung infection with organisms associated with a more rapid decline in pulmonary status * An acute illness not related to CF within 14 days before the first dose of study drug * A standard digital ECG demonstrating QTc \>450 msec at screening. * History of solid organ or hematological transplantation. * History or evidence of cataract or lens opacity determined to be clinically significant by the ophthalmologist or optometrist based on the ophthalmologic examination during the Screening Period. * History of alcohol or drug abuse in the past year, including but not limited to, cannabis, cocaine, and opiates, as deemed by the investigator. * Ongoing or prior participation in an investigational drug study, with certain exceptions. (e.g., ongoing participation in NCT02565914) * Use of commercially available CFTR modulator (e.g., Kalydeco, Orkambi) within 14 days before screening (applies only to the Heterozygous F508del/MF cohorts; does not apply to the Homozygous F508del/F508del Cohort). * Pregnant or nursing females: Females of childbearing potential must have a negative pregnancy test at screening and Day 1.