drugset / Trial / NCT02323100
Glycerol Phenylbutyrate Corrector Therapy For CF (Cystic Fibrosis)
Phase 1/2
Terminated
16 enrolled
National Jewish Health
Children's Hospital of Philadelphia · collabHorizon Pharma Ireland, Ltd., Dublin Ireland · collabJohns Hopkins University · collabUniversity of Alabama at Birmingham · collab
RandomizedParallel-groupQuadruple-blindTreatment
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.
Timeline
- Start
- 2018-12-02
- Primary completion
- 2022-03-01
- Completion
- 2022-03-01
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Comparator | Glycerol phenylbutyrate | Other / unclassified | 6.05 g | Oral |