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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT01840735
• Date of registration: 15/04/2013
• Prospective Registration: Yes
• Primary sponsor: Gilead Sciences
• Public title: Phase 1b Safety, Tolerability, and PK Study to Assess GS-5737 in Subjects With CF
• Scientific title: 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
• Date of first enrolment: May 2013
• Target sample size: 8
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT01840735
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
• Phase: Phase 1

Countries of recruitment

United States

Key inclusion & exclusion criteria

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



Age minimum: 18 Years
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: GS-5737

Drug: Placebo

Primary Outcome(s)

Peak Plasma Concentration (Cmax) of GS-5737 [Time Frame: 1 day]

Secondary ID(s)

GS-US-234-0117

Source(s) of Monetary Support

Please refer to primary and secondary sponsors