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

Completed · Phase 1 · Has a placebo group

Conditions studied: Cystic Fibrosis

In brief

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 near the expected therapeutic exposure level in preparation for Phase 2 studies of N91115 added to the CFTR modulator combination lumacaftor/ivacaftor when launched.

Key facts

Study ID

NCT02275936

Run by

Nivalis Therapeutics, Inc.

People needed

51

Starts

2015-02-01

Expected to finish

2015-07-01

Last updated by the study team

2016-11-07

Who can join

Age: 18 and older, up to 80. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• University of Alabama @ Birmingham — Birmingham, Alabama, United States
• Stanford University — Palo Alto, California, United States
• Children's CO — Aurora, Colorado, United States
• National Jewish Health — Denver, Colorado, United States
• Northwestern University — Chicago, Illinois, United States
• Indiana University — Indianapolis, Indiana, United States
• University of Iowa Children's Hospital — Iowa City, Iowa, United States
• Johns Hopkins Hospital — Baltimore, Maryland, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• University of Minnesota — Minneapolis, Minnesota, United States
• Washington University — St Louis, Missouri, United States
• Columbia University — New York, New York, United States
• The New York Presbyterian Hospital, Columbia University Medical Center — New York, New York, United States
• University of North Carolina — Chapel Hill, North Carolina, United States
• Cincinnati Children's Hospital — Cincinnati, Ohio, United States
• Rainbow Babies and Children's Hospital - Case Medical Center — Cleveland, Ohio, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Seattle Children's Hospital — Seattle, Washington, United States

Full record on ClinicalTrials.gov

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