Study of N91115 in CF Patients Who Are Heterozygous for F508del-CFTR and a Gating Mutation and Being Treated With Ivacaftor



Status:Recruiting
Conditions:Pulmonary
Therapuetic Areas:Pulmonary / Respiratory Diseases
Healthy:No
Age Range:18 - Any
Updated:7/23/2016
Start Date:April 2016
End Date:April 2017
Contact:Steven Shoemaker, MD
Email:steven.shoemaker@nivalis.com
Phone:720-945-7719

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A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study of N91115 for Efficacy and Safety in Patients With CF Heterozygous for F508del-CFTR + Gating Mutation Being Treated With Ivacaftor

N91115 is being studied as a potential novel therapy for cystic fibrosis (CF), and this
study assesses a target population of patients who are heterozygous for F508del-CFTR and a
gating mutation that is approved for treatment with ivacaftor (G551D, G1244E, G1349D, G178R,
G551S, S1251N, S1255P, S549N, or S549R).

Assess the effect of N91115 on lung function when added to preexisting treatment with
ivacaftor in adult patients with CF who are heterozygous for F508del-CFTR and a gating
mutation that is approved for treatment with ivacaftor (G551D, G1244E, G1349D, G178R, G551S,
S1251N, S1255P, S549N, or S549R).

Inclusion Criteria:

- Confirmed diagnosis of CF, heterozygous for F508del-CFTR and a gating mutation that
is approved for treatment with ivacaftor (G551D, G1244E, G1349D, G178R, G551S,
S1251N, S1255P, S549N, or S549R)

- Have been treated with chronic ivacaftor twice daily for at least 6 months prior to
Screening (date of consent) and are currently being treated with commercially
available Ivacaftor

- Negative serum pregnancy test

- Weight ≥ 40 kg at screening

- Oxygen saturation by pulse oximetry ≥ 90% breathing ambient air, at screening

Exclusion Criteria:

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

- Recent infection (per investigator discretion) with organisms associated with more
rapid decline in pulmonary status, for example: Burkholderia cenocepacia,
Burkholderia dolosa, and Mycobacterium abscessus

- Any change in the regimen for chronic therapies for CF lung disease (e.g.,
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 or renal function

- History of ventricular tachycardia or other clinically significant ventricular
arrhythmias

- History, including the screening assessment, of prolonged QT and/or QTcF
(Fridericia's correction) interval (> 450 msec for men; > 470 msec for women)

- 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
We found this trial at
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Cincinnati, Ohio 45229
Principal Investigator: John Clancy, MD
Phone: 513-803-7429
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700 Childrens Drive
Columbus, Ohio 43205
(616) 722-2000
Principal Investigator: Karen McCoy, MD
Phone: 614-722-4766
Nationwide Children's Hospital At Nationwide Children’s, we are creating the future of pediatric health care....
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116th St and Broadway
New York, New York 10027
(212) 854-1754
Phone: 212-305-0290
Columbia University In 1897, the university moved from Forty-ninth Street and Madison Avenue, where it...
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3181 Southwest Sam Jackson Park Road
Portland, Oregon 97239
503 494-8311
Principal Investigator: Gopal Aliada, MD
Phone: 503-494-6052
Oregon Health and Science University In 1887, the inaugural class of the University of Oregon...
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201 Presidents Circle
Salt Lake City, Utah 84108
801) 581-7200
Principal Investigator: Theodore Liou, MD
Phone: 801-581-3961
University of Utah Research is a major component in the life of the U benefiting...
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1 Brookings Drive
St. Louis, Missouri 63110
 (314) 935-5000
Principal Investigator: Daniel Rosenbluth, MD
Phone: 314-454-5175
Washington University Washington University creates an environment to encourage and support an ethos of wide-ranging...
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1800 Orleans St.
Baltimore, Maryland 21287
410-955-5000
Principal Investigator: Pamela Zeitlin, MD, PhD
Phone: 410-955-1167
Johns Hopkins Hospital Patients are the focus of everything we do at The Johns Hopkins...
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300 Longwood Ave
Boston, Massachusetts 02115
(617) 355-6000
Principal Investigator: Alicia Casey, MD
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Cleveland, Ohio 44106
Principal Investigator: James Chmiel, MD
Phone: 216-844-5733
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1400 Jackson St
Denver, Colorado 80206
(303) 388-4461
Principal Investigator: Jennifer Taylor-Cousar, MD
Phone: 303-398-1265
National Jewish Health National Jewish Health is known worldwide for treatment of patients with respiratory,...
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Madison, Wisconsin 53792
Principal Investigator: Trina Hollatz, MD
Phone: 608-263-4464
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3414 Fifth Avenue
Pittsburgh, Pennsylvania 15213
Principal Investigator: Joseph Pilewski, MD
Phone: 412-692-5872
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