Glycerol Phenylbutyrate Corrector Therapy For CF (Cystic Fibrosis)
Status:
Recruiting
Trial end date:
2022-06-30
Target enrollment:
Participant gender:
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.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
Johns Hopkins University National Jewish Health
Collaborators:
Children's Hospital of Philadelphia Horizon Pharma Ireland, Ltd., Dublin Ireland Johns Hopkins University University of Alabama at Birmingham