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Report for Clinical Trial NCT02323100

Developed by Shray Alag, 2020.
SNP Clinical Trial Gene

A Double Blind, Placebo Controlled, Dose Escalation Trial of Glycerol Phenylbutyrate Corrector Therapy for Cystic Fibrosis

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.

NCT02323100 Cystic Fibrosis
MeSH:Cystic Fibrosis Fibrosis

3 Interventions

Name: Ravicti low dose

Description: 8 am, 4pm and midnight
Type: Drug
Group Labels: 1

Ravicti low dose

Name: Ravicti high dose

Description: 8 am, 4pm and midnight
Type: Drug
Group Labels: 1

Ravicti high dose

Name: Placebo

Description: 8 am, 4pm and midnight
Type: Drug
Group Labels: 1


Primary Outcomes

Description: chloride and sodium transport in nasal epithelium

Measure: The primary biological endpoint will be the change in average measurement of nasal potential difference between day 7 and baseline.

Time: 7 days

Secondary Outcomes

Description: change between date and baseline in sodium and chloride transport

Measure: 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.

Time: 14 days

Description: change between study time point and baseline in sweat chloride

Measure: Change in average sweat chloride measurement between days 4, 7, 14 and baseline.

Time: 14 days

Description: standard safety and tolerability lab values

Measure: Safety and tolerability.

Time: 14 days

Description: quantification of exogenous pancreatic enzyme effects on release of active drug from the pro-drug triglyceride form

Measure: Efficacy of PERT on absorption of Ravicti®.

Time: 14 days

Other Outcomes

Description: blood pharmacokinetics

Measure: Plasma will be sampled for pharmacokinetics (PK) studies

Time: 14 days

Description: blood counts, metabolic measures, CRP

Measure: Safety labs: hematology, complete metabolic panel (CMP), C-reactive protein (CRP), erythrocyte sedimentation rate (ESR), uric acid. Sputum microbiology and spirometry will be performed.

Time: 14 days

Purpose: Treatment

Allocation: Randomized

Parallel Assignment

There is one SNP


1 G551D

In the ensuing years, Vertex Pharmaceuticals, Inc. has had success with the development of ivacaftor3;3;4 (VX-770) as a potentiator of G551D CFTR and has studied the drug alone and in combination with their corrector lumacaftor5 (VX-809) and VX-661. --- G551D ---

HPO Nodes