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Glycerol Phenylbutyrate Corrector Therapy for Cystic Fibrosis
NCT02323100Phase 2
Key facts
- Status
- Terminated
- Start
- Dec 2018
- Primary completion
- Mar 2022
- Countries
- 1
- Sponsor
- National Jewish Health
- Modality
- Small Molecules
- Conditions
- cystic fibrosis (CF)
01
Overview
Phase 2, randomised, double-blind trial in cystic fibrosis (CF) comparing Ravicti low dose with Placebo (2 arms). Primary endpoint: Change in Chloride and Sodium Transport in Nasal Epithelium at 7 days. Started Dec 2018; status: terminated.
02
Design
2 arms- Study design
- RandomizedDouble-blind
Ravicti low dose
Low dose Ravicti oral liquid at 6ml (6.6 gm) by mouth or gastrostomy tube at 8 am, 5.5 ml (6.05gm) at 4pm and midnight for 7 days.
Placebo
Matching placebo taken at 8am, 4pm and midnight for 7 days.
03
Who it enrols
Adults with Cystic Fibrosis (any CFTR genotype except two stop codons) taking pancreatic enzyme replacement therapy, with FEV1 >= 30%.
04
Endpoints
Primary
Change in Chloride and Sodium Transport in Nasal Epithelium
Secondary
Change in Average Sweat Chloride
Change in Baseline Nasal Potential Difference
Change in FEV1
Safety and Tolerability
05
Results
Not all primary endpoints met
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