Glycerol Phenylbutyrate Corrector Therapy For CF (Cystic Fibrosis)
Stopped: funding ended
United States16 participantsStarted 2018-12-02
Plain-language summary
We propose to test the effectiveness of the combination of CF pancreatic enzyme replacement therapy (PERT) on absorption of Ravicti® and subsequent restoration of nasal epithelial cystic fibrosis transmembrane conductance regulator (CFTR)-mediated chloride transport during the nasal potential difference (NPD) test. Funding source FDA Office of Orphan Products Development.
Who can participate
Age range18 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. Male or female ≥ 18 years of age.
✓. Confirmed diagnosis of CF based on the following criteria:
✓. Taking pancreatic enzyme replacement therapy (PERT), or have documented pancreatic sufficiency.
✓. Ability to perform acceptable spirometry.
✓. Ability to understand and sign a written informed consent and comply with the requirements of the study.
✓. FEV1 ≥30% of predicted normal for age, gender, and height (Hankinson standards): pre or post-bronchodilator at Screening.
✓. Oxygen saturation by pulse oximetry ≥90% breathing either ambient air or regular oxygen regimen at screening and Day 1.
✓. Hematology and clinical chemistry of blood and urine results with no clinically significant abnormalities that would interfere with the study assessments (as judged by the principal investigator) at screening. If electrolyte abnormality at screening, values must be corrected prior to dosing.
Exclusion criteria
✕. Administration of any investigational drug or device within 30 days of Screening or within 6 half-lives of the investigational drug (whichever is longer).
✕. History of any illness or condition that in the opinion of the investigator could confound the results of the study or pose additional risk in administering study drug to subjects.
✕. Any change in chronic therapies for CF lung disease (e.g., Ibuprofen, Pulmozyme®, hypertonic saline, Azithromycin, TOBI®, Cayston®) within 4 weeks of Study Day 1.
What they're measuring
1
Change in Chloride and Sodium Transport in Nasal Epithelium