Cystic Fibrosis MedDRA version: 20.0 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Sponsors
Vertex Pharmaceuticals Incorporated
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Subject (or the subject's legally appointed and authorized representative) will sign and date an informed consent form (ICF) and, when appropriate, an assent form. 2. Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines (as applicable), and other study procedures. . For subjects =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. History of any illness or any clinical condition that might confound the results of the study or pose an additional risk in administering study drug(s) to the subject. This may include, but is not limited to: • History of allergy, intolerance, or hypersensitivity to any component of the investigational drug product (ELX/TEZ/IVA tablets and IVA tablets) or placebo, including excipients • Clinically significant liver cirrhosis with or without portal hypertension • Solid organ or hematological transplantation • Alcohol or drug abuse in the past year, including, but not limited to, cannabis, cocaine, and opiates, as deemed by the investigator Cancer, except for squamous cell skin cancer, basal cell skin cancer, and Stage 0 cervical carcinoma in situ (all 3 with no recurrence for the last 5 years). 2. Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject (as deemed by the investigator). 3. Any of the following abnormal laboratory values at screening: . Hemoglobin <10 g/dL . Total bilirubin =2 x upper limit of normal (ULN) . AST, ALT, gamma-glutamyl transferase (GGT), or alkaline phosphatase (ALP) = 3 x 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) for subjects =18 years of age and =45 mL/min/1.73 m2 (calculated by the Counahan-Barratt equation) 15 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 Day 1 (first dose of study drug). 5. Lung infection with organisms associated with a more rapid decline in pulmonary status (including, but not limited to, Burkholderia cenocepacia, Burkholderia dolosa, and Mycobaterium 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, and . 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 one 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 (Day 1). 7. Ongoing or prior participation in an investigational drug study within 28 days of the Screening Visit. . A washout period of 5 terminal half-lives of the previous investigational study drug, or 28 days, whichever is longer, must elapse before the Screening Visit. . The duration of the elapsed time may be longer if required by local regulations. 8. Pregnant and breast-feeding females. Female subjects of childbearing potential (Section 11.5.6.1) must have a negative pregnancy test at the Screening Visit and the Day 1 Visit. 9. Use of restricted medication within specified duration before the first dose of study drug as defined in Table 9-3. 10. Subject, or close relative of the subject, is the investigator or a subinvestigator, research assistant, pharmacist, study coordinator, or other staff directly involved with
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the efficacy and pharmacodynamics (PD) of ELX/TEZ/IVA;Secondary Objective: To evaluate safety and tolerability of ELX/TEZ/IVA;Primary end point(s): Absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1) through Week 24;Timepoint(s) of evaluation of this end point: From signing of ICF until safety Follow up visit. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): •Absolute change from baseline in SwCl through Week 24 • Absolute change from baseline in Cystic Fibrosis Questionnaire- Revised respiratory domain (CFQ-R RD) score through Week 24 • Absolute change from baseline in body mass index (BMI) at Week 24 • Absolute change from baseline in weight at Week 24 • Number of pulmonary exacerbations (PEx) through Week 24 • Safety and tolerability assessments based on AE, clinical laboratory values, ECGs, vital signs, and pulse oximetry;Timepoint(s) of evaluation of this end point: From signing of ICF until safety Follow up visit. | — |
Countries
Austria, Belgium, Canada, Czechia, Czech Republic, France, Germany, Hungary, Italy, Netherlands, Norway, Poland, Portugal, Spain, Sweden, Switzerland
Contacts
Public ContactClinical Trials and Medical Info
Vertex Pharmaceuticals Incorporated
Outcome results
None listed