A Research Study to Advance the CF Therapeutics Pipeline for People Without Modulators

Inclusion Criteria

• •Consent
• •A. Written informed consent (and assent when applicable) obtained from participant or participant's legal guardian
• •B. Is willing and able to adhere to the study visit schedule and other protocol requirements
• •Demographics
• •A. ≥ 12 years of age at Visit 1
• •Medical History
• •A. For persons of child-bearing potential: must not be pregnant at Visit 1 or plan to get pregnant during the 12-month study period
• •Disease History
• •A. Documentation of a CF diagnosis as evidenced by one or more clinical features consistent with the CF phenotype and one or more of the following criteria:
• •Sweat chloride ≥ 60 mEq/liter by quantitative pilocarpine iontophoresis test (QPIT)
• •Two well-characterized disease-causing pathogenic variants in the CFTR gene
• •or
• •One well-characterized disease-causing mutation and a second CFTR variant (with variable or uncharacterized disease-causing potential) and sweat ≥ 30 mmol/liter with permission of the study sponsor-investigators
• •B. Clinically stable with no significant changes in health status within the 28 days prior to and including Visit 1
• •C. Does not have a history of lung transplantation
• •Concomitant Medications
• •A. Not genetically eligible for a CFTR modulator according to product label indications and/or No use of CFTR modulator for 28 days prior to Visit 1 with no intent to start or restart during the study period
• •B. No use of an investigational drug within 90 days prior to and including Visit 1
• •C. Not currently participating in an interventional drug or device trial. Participation in long-term safety follow-up studies (without redosing) and/or behavioral intervention trials is allowed.
• •D. No initiation of new chronic therapy (e.g., ibuprofen, azithromycin, inhaled tobramycin, Cayston®) within 28 days prior to and including Visit 1
• •E. No acute use of antibiotics (oral, inhaled or IV) or acute use of systemic corticosteroids for respiratory tract symptoms within 28 days prior to and including Visit 1