Study of N91115 in Patients With Cystic Fibrosis Homozygous F508del-CFTR Mutation

• Register: ClinicalTrials.gov
• Last refreshed on: 14 November 2016
• Main ID: NCT02275936
• Date of registration: 14/10/2014
• Prospective Registration: Yes
• Primary sponsor: Nivalis Therapeutics, Inc.
• Public title: Study of N91115 in Patients With Cystic Fibrosis Homozygous F508del-CFTR Mutation SNO4
• Scientific title: A Phase 1b, Randomized, Double-Blind, Placebo-Controlled, Parallel, Group Study of N91115 to Evaluate Safety and Pharmacokinetics in Patients With Cystic Fibrosis Homozygous for the F508del-CFTR Mutation
• Date of first enrolment: February 2015
• Target sample size: 51
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02275936
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Scott Donaldson, MD
• Affiliation: University of North Carolina

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Male or female, age = 18 years with confirmed diagnosis of CF, homozygous for the
F508del-CFTR mutation based on historical results generated by Ambry Genetics within
the past two years or if unavailable, confirmed by testing done within the past 28
days

2. Sweat chloride = 60 (milliequivalents) mEq/L, by quantitative pilocarpine
iontophoresis test (QPIT) at screening

3. Weight = 40 kg at screening

4. Forced expiratory volume (FEV1) = 40% of predicted normal for age, gender, and height
(Hankinson standards) pre- or post-bronchodilator value, at screening

5. Oxygen saturation by pulse oximetry = 90% breathing ambient air, at screening

6. Hematology, clinical chemistry and urinalysis results with no clinically significant
abnormalities that would interfere with the study assessments at screening

Exclusion Criteria:

1. Any acute infection, including acute upper or lower respiratory infections and
pulmonary exacerbations that require treatment or hospitalization within 2 weeks of
Study Day 1

2. Any change in chronic therapies for CF lung disease (e.g., Ibuprofen, Pulmozyme®,
hypertonic saline, Azithromycin, Tobi®, Cayston®) within 4 weeks of Study Day 1

3. Blood hemoglobin < 10 g/dL at screening

4. Serum albumin < 2.5 g/dL at screening

5. Abnormal liver function defined as = 3 x upper limit of normal (ULN) in 3 or more of
the following: aspartate aminotransferase (AST), alanine aminotransferase (ALT),
g-glutamyl transferase (GGT), alkaline phosphatase (ALP), or total bilirubin at
screening

6. History of abnormal renal function (creatinine clearance < 50 mL/min using
Cockcroft-Gault equation) within a year of screening

7. History, including the screening assessment, of ventricular tachycardia or other
ventricular arrhythmias

8. History, including the screening assessment, of prolonged cardiac QT interval and/or
QTcF (QT with Fridericia's correction) interval (> 450 msec)

9. History of solid organ or hematological transplantation

10. History of alcohol abuse or drug abuse (including cannabis, cocaine, and opioids) in
the year prior to screening

11. Use of continuous (24 hr/day) or nocturnal supplemental oxygen



Age minimum: 18 Years
Age maximum: 80 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: N91115

Primary Outcome(s)

Safety assessments based on clinical evaluations, laboratory assessments, and adverse events. [Time Frame: 28 Days]

Secondary Outcome(s)

Pharmacokinetic (PK) parameters of N91115 and its glucuronide metabolite in plasma [Time Frame: 28 Days]

Secondary ID(s)

N91115-1CF-03

Source(s) of Monetary Support

Please refer to primary and secondary sponsors