A study in people with cystic fibrosis (a rare pulmonary disease) to assess the pharmacokinetics and safety of a combination of two experimental drugs

Phase 1

Conditions: Cystic Fibrosis
MedDRA version: 20.0Level: SOCClassification code 10010331Term: Congenital, familial and genetic disordersSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Registration Number: EUCTR2017-001078-41-Outside-EU/EEA

Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: A

Sex: All

Target Recruitment: 58

Inclusion Criteria

•Subjects (males and/or females), 6 through 11 years of age, inclusive, on the date of
informed consent (and assent, if applicable) (Part A and Part B).
•Confirmed diagnosis of CF defined as: with 2 CF-causing mutations, chronic sinopulmonary disease or gastrointestinal/nutritional abnormalities
•Subjects who weigh =15 kg without shoes at Screening Visit
•Subjects who are homozygous for the F508del-CFTR mutation
•Subjects with percent predicted forced expiratory volume in 1 second (FEV1) of 70% to 105% (inclusive) (Part A) or =40% (Part B) at Screening Visit
•Subjects with stable CF disease and who are willing to remain on stable CF medication regimen
•Able to swallow tablets

Are the trial subjects under 18? yes
Number of subjects for this age range: 56
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

•History of any illness or condition that might confound the results of the study or pose an additional risk in administering study drug to the subject
•Acute respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 28 days before Day 1 of the study
•Abnormal liver function as defined in the protocol at Screening Visit
•Abnormal renal function as defined in the protocol at Screening Visit
•History of solid organ or hematological transplantation
•Ongoing participation in an investigational drug study or prior participation in an investigational drug study within 30 days prior of Screening Visit
•History or evidence of lens opacity or cataract at Screening Visit
•Colonization with organisms associated with a more rapid decline in pulmonary status at Screening Visit (Part A only)
•A standard 12-lead ECG demonstrating QTcF >450 msec at Screening Visit