Cystic Fibrosis Trial, Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• People who have signed a consent form (and an assent form where required for younger participants)
• People who are willing and able to attend study visits and follow the study requirements
• People who are male or female, aged 6 years or older, and within the age range approved by the FDA for the specific CFTR modulator drug being studied (Ivacaftor: 4 months+; Lumacaftor/Ivacaftor: 2 years+; Tezacaftor/Ivacaftor: 12 years+; Elexacaftor/Tezacaftor/Ivacaftor: 12 years+)
• People who have at least one rare cystic fibrosis (CF) gene variant — meaning one that appears in fewer than 5% of people with CF
• People who have a confirmed CF diagnosis, supported by clinical symptoms plus at least one of: a high sweat chloride test result (≥60 mmol/L), two mutations found in the CF gene, or an abnormal nasal test result that supports a CF diagnosis
• People whose lung function test (FEV1) shows a result greater than 50% of what would be expected for their age
• People whose regular CF treatments have not changed in the past 28 days or more (rotating inhaled antibiotics such as tobramycin are an exception)
• People who have been prescribed a CFTR modulator medication by a licensed doctor
• People who have no medical reason preventing them from taking the study drug at the time treatment begins

Who may not be able to join:

• People who have a condition or health issue that, in the investigator's judgement, could put them at risk or affect the reliability of study data
• Women who are pregnant, who test positive for pregnancy at the first visit, or who are currently breastfeeding
• Women of childbearing age who are unwilling to use an acceptable form of contraception (such as abstinence, hormonal birth control, an intrauterine device, or a barrier method with spermicide) unless they have been surgically sterilised or have gone through menopause
• People who are underweight — below the 10th percentile for body weight relative to age (for those under 18), or with a BMI below 20 kg/m² (for those 18 and older)
• People whose lung function test (FEV1) result is 50% or less of what would be expected for their age
• People who have had certain CF-related bacterial infections (such as nontuberculous mycobacteria or Burkholderia species) found in sputum tests within the six months before enrolment, if those infections are linked to unstable disease
• People currently taking certain medications that interact with a liver enzyme called CYP3A — for example, voriconazole, fluconazole, or rifampin — or who are taking more than 20 mg of prednisone daily
• People with poorly controlled diabetes (confirm with trial site for specific lab thresholds)
• People with advanced CF-related liver disease involving serious complications such as cirrhosis, portal hypertension, or fluid build-up in the abdomen, or with abnormal liver test results (confirm with trial site for specific thresholds)
• People with end-stage kidney disease
• People who have previously had an organ transplant
• People with other medical conditions that, in the investigator's judgement, could put them at risk or affect their ability to complete the trial — such as uncontrolled depression, an anxiety disorder, poor adherence to CF treatments, or active allergic bronchopulmonary aspergillosis (ABPA)
• People whose blood test results at the screening visit fall outside acceptable ranges, including abnormal white blood cell counts, low haemoglobin, low platelet counts, reduced kidney function, significant sugar in the urine, or abnormal liver or clotting results (confirm with trial site for specific values)
• People who test positive for pregnancy at the screening visit

Important: Always verify eligibility with the trial site directly before applying.