Phase 2/3 Adaptive Study of VX-147 in Adults and Pediatric Subjects With APOL1-mediated Proteinuric Kidney Disease

You likely can't join if

• Part A: 1. History of any illness or any clinical condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug(s) to the subject. 2. Evidence of FSGS with a known cause other than due to APOL1 mutations. 3. History of diabetes mellitus. A diagnosis of prediabetes is permitted. 4. Known underlying cause of kidney disease 5. Abnormal laboratory values at screening that present a risk to subject safety in the opinion of the investigator, or any of the following abnormal laboratory values at screening: • Serum albumin <1 g/dL • Total bilirubin ≥1.5 × upper limit of normal (ULN) • Aspartate transaminase (AST) or alanine transaminase (ALT) ≥2 × ULN • Hemoglobin <9 g/dL. 6. Risk factors for Torsade de Pointes (e.g., familial long QT syndrome, chronic hypokalemia, heart failure) or concomitant medications that prolong the QT/QTc interval or any history of cardiac disorders 7. Any clinically significant ECG abnormality (as determined by the investigator) or median QTcF of triplicate standard 12-lead ECGs >450 msec at screening. 8. Positive for hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) RNA, or positive HIV test 9. Screening blood pressure, based on the average of 3 measurements, of ≥180 mm Hg (systolic) or ≥100 mm Hg (diastolic). 10. Pregnant or nursing female subjects. Females of childbearing potential must have a negative pregnancy test at screening (serum test) and Day 1 (urine test). 11. Known hypersensitivity to investigational medicinal product or to any of its excipients.
• Part B: 1. ESKD as defined in Section 9.10.1, in Part A. 2. History, new onset, or worsening 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. 3.Pregnant or breastfeeding females. Female subjects of childbearing potential must have a negative urine pregnancy test on Day 1 of Part B before receiving the first dose of study drug. 4.Any clinically significant laboratory abnormalities before Day 1 of Part B that would interfere with the study assessments or pose an undue safety risk for the subject, as deemed by the site investigator. 5.Ongoing or planned participation in another interventional, therapeutic clinical trial other than Part A of the study. Participation in a noninterventional study, including observational and registry studies, is permitted. 6.Any condition or circumstances, which in the opinion of the site investigator or Vertex, may make a subject unlikely or unable to complete the study or comply with study procedures and requirements of Part B, including study restrictions as defined in Section 9.5. 7.Subject, or close relative or a caregiver of the subject, is the investigator or a subinvestigator, research assistant, pharmacist, study coordinator, or other staff directly involved with the conduct of the study at that site. An adult (aged 18 years or older) who is a relative of a study staff member may be enrolled in the study provided the following: • The adult lives independently of and does not reside with the study staff member; and • The adult participates in the study at a site other than the site at which the family member is employed.