CFDB - Cystic Fibrosis DataBase

ongoing trials trial from www.clinicaltrials.gov

Glycerol Phenylbutyrate Corrector Therapy For CF (Cystic Fibrosis) - Phase 1|Phase 2 - Recruiting

Study design (if review, criteria of inclusion for studies)

Interventional - Allocation: Randomized|Intervention Model: Parallel Assignment|Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|Primary Purpose: Treatment

Participants

Cystic Fibrosis - 18 Years and older   (Adult, Older Adult)

Interventions

Drug: Ravicti low dose|Drug: Ravicti high dose|Drug: Placebo

Outcome measures

The primary biological endpoint will be the change in average measurement of nasal potential difference between day 7 and baseline.|Change in other NPD measures from baseline and Days 4, 7, and 14 to include baseline PD, change in amiloride, low chloride, and low chloride plus isoproterenol.|Change in average sweat chloride measurement between days 4, 7, 14 and baseline.|Safety and tolerability.|Efficacy of PERT on absorption of Ravicti®.

Keywords: Ravicti; glycerol-phenylbutyrate; CFTR Modulators; pharmacological_intervention;