Please select the eligibility criteria that correspond to the clinical trial below.
Title: A Phase IIa, Randomized, Double-blind, Placebo-controlled Study to Evaluate Multiple Doses of GLPG2222 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del Mutation
Brief Summary: This is a Phase IIa, multi-center, randomized, double-blind, placebo-controlled, parallel-group study to evaluate 4 different doses of GLPG2222 administered for 4 weeks to adult subjects with a confirmed diagnosis of CF and homozygous for the F508del Cystic Fibrosis Transmembrane conductance Regulator (CFTR) mutation.


Options:
A: Key Inclusion Criteria:

* Homozygous for the F508del mutation (F/F)
* Forced expiratory volume in 1 second (FEV1) value ≥40% and ≤90% of predicted mean for age, sex, and height

Key Exclusion Criteria:

* Clinically significant cirrhosis with or without portal hypertension
* Lung infection with organisms associated with a more rapid decline in pulmonary status
* Solid organ or hematological transplantation

Other protocol defined Inclusion/Exclusion criteria may apply
B: Key Inclusion Criteria:

* Ability to provide written informed consent (parental/guardian consent and participant assent if less than \[\<\] 18 years of age).
* Evidence of completed participation in the double-blind study, PTC124-GD-009-CF (Study 009).
* Body weight greater than or equal to (≥) 16 kilograms (kg).
* Performance of a valid, reproducible spirometry test using the study-specific spirometer during the screening period.
* Confirmed laboratory values within the central laboratory ranges at screening.
* In male and female participants who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during the study drug administration and 60-day follow-up period.
* Willingness and ability to comply with all study procedures and assessments, including scheduled visits, drug administration plan, laboratory tests, and study restrictions.

Key Exclusion Criteria:

* Chronic use of systemic tobramycin within 4 weeks prior to screening.
* Evidence of pulmonary exacerbation or acute upper or lower respiratory tract infection (including viral illnesses) within 3 weeks prior to screening or between screening and randomization.
* Any change (initiation, change in type of drug, dose modification, schedule modification, interruption, discontinuation, or re-initiation) in a chronic treatment/prophylaxis regimen for CF or for CF-related conditions within 4 weeks prior to screening and randomization.
* Known hypersensitivity to any of the ingredients or excipients of the study drug.
* Exposure to another investigational drug within 4 weeks prior to screening.
* Treatment with intravenous antibiotics within 3 weeks prior to screening.
* History of solid organ or hematological transplantation.
* Ongoing immunosuppressive therapy (other than corticosteroids).
* Positive hepatitis B surface antigen, hepatitis C antibody test or human immunodeficiency virus (HIV) test.
* Known portal hypertension.
* Pregnancy or breast-feeding.
C: Inclusion Criteria:

* Subject voluntarily agrees to participate in this study and signs an Institutional Review Board (IRB)-approved informed consent prior to performing any of the screening procedures and in the opinion of the PI complies with all the requirements of the study.
* Subject is healthy, determined at the screening medical evaluation (including but not limited to medical history, physical examination and clinical laboratory evaluations).
* Subject is Caucasian.
* Female subject must be of non-childbearing potential (surgically sterile \[hysterectomy or bilateral tubal ligation\] or post-menopausal ≥ 1 year with follicle stimulating hormone \[FSH\] \> 40 U/L). Women receiving hormone replacement therapy (HRT) are eligible to enroll.
* Male subject must agree to use condoms and refrain from sperm donation from Day 1 until 30 days post last dose or have documentation of vasectomy.
* Subject has a body weight \> 50 kg and body mass index (BMI) between 19.5 and 32 kg/m2, inclusive, at screening.
* Subject has no clinically significant abnormal findings related to their systolic or diastolic blood pressure (BP), per the investigator's judgment, at screening or Day 1.
* Subject has no clinically significant abnormal findings in 12 lead ECG, per the investigator's judgment, at screening.

Exclusion Criteria:

* Subject has clinically significant history or evidence of cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrine, neurological, immunological, or psychiatric disorder(s) as determined by the investigator or designee.
* Subject has clinically significant abnormalities on a 12 lead ECG done at screening
* Subject has clinically significant abnormalities on a 48-hour ambulatory ECG done at screening
* Subject has any disorder that would interfere with the absorption, distribution, metabolism, or excretion of drugs.
* Subject has any concurrent disease or condition that, in the opinion of the investigator, would make the subject unsuitable for participation in the clinical study.
* Subject is unlikely to comply with the protocol requirements, instructions, and study related restrictions; e.g., uncooperative attitude, unavailable for follow-up call, and/or improbability of completing the clinical study.
D: Inclusion Criteria:

1. Male or female subject ≥ 18 years of age, on the day of signing the Informed Consent Form (ICF).
2. A confirmed clinical diagnosis of CF and homozygous for the F508del CFTR mutation
3. Weight ≥ 40 kg.
4. Stable concomitant treatment for at least 4 weeks (28 days) prior to baseline
5. Forced expiratory volume in 1 second (FEV1) ≥ 40% of predicted normal for age, gender and height at screening

Exclusion Criteria:

1. History of clinically meaningful unstable or uncontrolled chronic disease that makes the subject unsuitable for inclusion in the study in the opinion of the investigator.
2. Unstable pulmonary status or respiratory tract infection requiring a change in therapy within 4 weeks of baseline.
3. Need for supplemental oxygen during the day, and \>2 liters per minute (LPM) while sleeping.
4. Use of CFTR modulator therapy (e.g. lumacaftor or ivacaftor) within 4 weeks prior to the first study drug administration.
5. History of hepatic cirrhosis with portal hypertension.
6. Abnormal liver function test at screening; defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) and/ or alkaline phosphatase and/or gamma-glutamyl transferase (GGT) ≥ 3x the upper limit of normal (ULN); and/or total bilirubin (\>1.5 times ULN)
7. Estimated creatinine clearance \< 60 mL/min using the Cockcroft-Gault formula at screening.

Answer: D