Status:
COMPLETED
Study of N91115 in Patients With Cystic Fibrosis Homozygous F508del-CFTR Mutation
Lead Sponsor:
Nivalis Therapeutics, Inc.
Conditions:
Cystic Fibrosis
Eligibility:
All Genders
18-80 years
Phase:
PHASE1
Brief Summary
This Phase 1b study in F508del-CFTR homozygous CF patients is being conducted to assess the safety of N91115 as the sole cystic fibrosis transmembrane conductance regulator (CFTR) modulator at doses n...
Detailed Description
Study procedures, frequency and timing are provided in the attached study schema. Adverse events and concomitant medication will be monitored throughout the study from informed consent signing until e...
Eligibility Criteria
Inclusion
- 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
- Sweat chloride ≥ 60 (milliequivalents) mEq/L, by quantitative pilocarpine iontophoresis test (QPIT) at screening
- Weight ≥ 40 kg at screening
- Forced expiratory volume (FEV1) ≥ 40% of predicted normal for age, gender, and height (Hankinson standards) pre- or post-bronchodilator value, at screening
- Oxygen saturation by pulse oximetry ≥ 90% breathing ambient air, at screening
- Hematology, clinical chemistry and urinalysis results with no clinically significant abnormalities that would interfere with the study assessments at screening
Exclusion
- 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
- 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
- Blood hemoglobin \< 10 g/dL at screening
- Serum albumin \< 2.5 g/dL at screening
- 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
- History of abnormal renal function (creatinine clearance \< 50 mL/min using Cockcroft-Gault equation) within a year of screening
- History, including the screening assessment, of ventricular tachycardia or other ventricular arrhythmias
- History, including the screening assessment, of prolonged cardiac QT interval and/or QTcF (QT with Fridericia's correction) interval (\> 450 msec)
- History of solid organ or hematological transplantation
- History of alcohol abuse or drug abuse (including cannabis, cocaine, and opioids) in the year prior to screening
- Use of continuous (24 hr/day) or nocturnal supplemental oxygen
Key Trial Info
Start Date :
February 1 2015
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 1 2015
Estimated Enrollment :
51 Patients enrolled
Trial Details
Trial ID
NCT02275936
Start Date
February 1 2015
End Date
July 1 2015
Last Update
November 7 2016
Active Locations (19)
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1
University of Alabama @ Birmingham
Birmingham, Alabama, United States, 35294
2
Stanford University
Palo Alto, California, United States, 94304
3
Children's CO
Aurora, Colorado, United States, 80045
4
National Jewish Health
Denver, Colorado, United States, 80206