Phase 1b Safety, Tolerability, and PK Study to Assess GS-5737 in Subjects With CF

A Phase 1b, Randomized, Double-Blind, Placebo-Controlled, Single Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of GS-5737 in Subjects With Cystic Fibrosis

This study will determine the safety, tolerability, and pharmacokinetics of a single dose of GS-5737 administered with a 2.8% saline solution vehicle in adult subjects with CF.

Study Overview

• Status: Completed
• Conditions: Cystic Fibrosis
• Intervention / Treatment: Drug: Placebo; Drug: GS-5737

• Study Type: Interventional
• Enrollment (Actual): 8
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Florida
    • Orlando, Florida, United States, 32806
      • Compass Research Phase 1, LLC

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Males or females, ≥ 18 years of age, at Screening
• Diagnosis of CF as determined by the 1997 CF Consensus Conference criteria, with at least 1 of the following: Documented sweat chloride ≥ 60 mEq/L by quantitative pilocarpine iontophoresis test OR Abnormal nasal transepithelial potential difference (NPD) test OR Two well-characterized, disease-causing genetic mutations in the CF transmembrane conductance regulator (CFTR) gene AND 1 or more accompanying clinical features consistent with CF
• FEV1 ≥ 40% and ≤ 90% predicted
• BMI ≥ 19 and ≤ 30 kg/m2
• Clinically stable with no evidence of significant new or acute respiratory symptoms
• Chest radiograph without significant acute findings; or chest radiograph, CT, or MRI obtained and interpreted within 90 days prior to enrollment, without acute findings and no significant intercurrent illness; chronic, stable findings are allowed

• History of lifetime smoking < 5 pack-years (ie, 1 pack per day x 1 year =

  1 pack-year) and non-smokers of at least 60 days duration prior to Screening

• Estimated creatinine clearance ≥ 80 mL/min at Screening
• Negative drug tests; including alcohol
• Hepatitis B, C, & HIV Negative
• Surgically sterile or ≥ 12 months post-menopausal
• Non-pregnant females

Exclusion Criteria:

• Experienced symptoms of recent acute upper or lower respiratory tract infection or acute pulmonary exacerbation requiring treatment within 2 weeks prior to Screening
• Plasma potassium ≥ 5 mEq/L
• Changes in chronic azithromycin use, bronchodilator (BD), dornase alfa, HS, physiotherapy technique or regimen, antibiotics or corticosteroid medications within 28 days prior to Screening
• History of sputum or throat swab culture yielding Burkholderia species within 2 years of Screening

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Single Group Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: GS-5737; The GS-5737 85 μg dose is contained in 4 mL of 10 mM citrate buffer, pH 5.0 in 2.8% (w/v) saline.	Drug: GS-5737
Placebo Comparator: Placebo; The vehicle (placebo) control contains 10 mM citrate buffer, pH 5.0 in 2.8% (w/v) saline in 4 mL.	Drug: Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Peak Plasma Concentration (Cmax) of GS-5737	1 day

Collaborators and Investigators

• Sponsor: Gilead Sciences

Study record dates

Study Major Dates

• Study Start: May 1, 2013
• Primary Completion (Actual): July 1, 2013
• Study Completion (Actual): August 1, 2013

Study Registration Dates

• First Submitted: April 15, 2013
• First Submitted That Met QC Criteria: April 23, 2013
• First Posted (Estimate): April 26, 2013

Study Record Updates

• Last Update Posted (Estimate): August 12, 2014
• Last Update Submitted That Met QC Criteria: August 8, 2014
• Last Verified: August 1, 2014

More Information

Terms related to this study

• Keywords: PK; Cystic Fibrosis; CF
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Pancreatic Diseases; Fibrosis; Cystic Fibrosis
• Other Study ID Numbers: GS-US-234-0117