Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Parion Sciences
With
Vertex Pharmaceuticals Incorporated
Age
12–any
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
The subject must have evidence supportive of a PCD diagnosis.
Subjects with percent predicted FEV1 of ≥40 to \<90 percentage points
Non-smoker for at least 90 days prior to the Screening Visit and less than a 5 pack-year lifetime history of smoking
Stable regimen of medications and chest physiotherapy for the 28 days prior to Day 1
If currently using daily inhaled HS, must be able to discontinue its use for the duration of the study.
If taking daily chronic or chronic cycling antibiotics, has been on a consistent regimen for at least 4 months prior to the Screening Visit.
Clinically stable (as deemed by the investigator) for at least 14 days prior to the Screening Visit
Female subjects of childbearing potential must have a negative serum pregnancy test at the Screening Visit. Subjects of childbearing potential and who are sexually active must meet the contraception requirements.
You may not be if
Diagnosis of CF based on results of sweat chloride or nasal potential difference (NPD) tests or presence of 2 CF-causing mutations in CFTR gene.
History of any organ transplantation or lung resection or chest wall surgery.
Significant congenital heart defects, other than a laterality defect, at the discretion of the investigator
Diagnosis of Cri du chat syndrome (chromosome 5p deletion syndrome).
Inability to withhold short-acting bronchodilator use for 4 hours prior to clinic visit and long-acting bronchodilator use the night before the first and last clinic visit of each treatment period.
Use of diuretics (including amiloride) or renin-angiotensin antihypertensive drugs
Symptoms of acute upper or lower respiratory tract infection, acute pulmonary exacerbation, or treatment or was treated with systemic antibiotics for ear or sinus disease within 28 days before Day 1 (topical otic antibiotics allowed).
History of significant intolerance to inhaled HS
Pregnant and/or nursing females
Any clinically significant laboratory abnormalities
History of chronic B. cepacia complex or M. abscessus or M. avium
Surgery that required general anesthesia and hospitalization within 3 months of Day 1
Unable to swallow tablets.
Concomitant use of strong or moderate inhibitors or inducers of cytochrome P450 (CYP) 3A, including consumption of certain herbal medications (e.g., St. John's Wort), and grapefruit/grapefruit juice.
Known hypersensitivity to ivacaftor.
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