Study of N91115 in Patients With CF Homozygous for the F508del-CFTR Mutation



Status:Active, not recruiting
Conditions:Pulmonary
Therapuetic Areas:Pulmonary / Respiratory Diseases
Healthy:No
Age Range:18 - Any
Updated:7/29/2016
Start Date:November 2015
End Date:December 2016

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A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study of N91115 to Evaluate Efficacy and Safety in Patients With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation Treated With Lumacaftor/Ivacaftor

This will be a double-blind, randomized, placebo-controlled, parallel group study. The
purpose of this study is to investigate the efficacy and safety of N91115 in adult patients
with CF who are homozygous for the F508del-CFTR mutation and being treated with
lumacaftor/ivacaftor (Orkambi™).

Primary Objective:

- Assess the efficacy of N91115 at 12 weeks when added to preexisting treatment with
lumacaftor/ivacaftor in adult patients with CF who are homozygous for the F508del-CFTR
mutation

Secondary Objectives:

- Assess the effect of N91115 added to lumacaftor/ivacaftor on safety

- Assess the effect of lumacaftor/ivacaftor added to N91115 on the pharmacokinetics of
N91115, lumacaftor, and ivacaftor

Inclusion Criteria:

- Patients must have been treated with lumacaftor/ivacaftor for at least 8 weeks prior
to Day 1 (start of dosing)

- A history of Sweat Chloride (SC) ≥ 60 mEq/L by quantitative pilocarpine iontophoresis
test (QPIT) (either before or after starting lumacaftor/ivacaftor treatment)

- Body weight ≥ 40 kg

- ppFEV1 40 - 85 % predicted (inclusive) at screening

- Oxygen saturation ≥ 90% breathing ambient air at screening

Exclusion Criteria:

- Any acute infection that requires treatment or hospitalization within 2 weeks of
Study Day 1

- Colonization with organisms associated with more rapid decline in pulmonary status,
such as 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

- Are pregnant, planning a pregnancy, or breast-feeding at screening

- 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)

- History of abnormal renal function within 3 months of screening

- 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

- History of solid organ or hematological transplantation

- History of alcohol abuse or drug abuse

- Ongoing participation in another therapeutic clinical trial

- Use of continuous (24 hr/day) or nocturnal supplemental oxygen
We found this trial at
46
sites
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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Chapel Hill, North Carolina 27599
(919) 962-2211
Principal Investigator: Scott Donaldson, MD
Phone: 984-974-2961
University of North Carolina at Chapel Hill Carolina’s vibrant people and programs attest to the...
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425 University Blvd.
Indianapolis, Indiana 46202
(317) 274-4591
Principal Investigator: Cynthia Brown, MD
Phone: 317-274-1441
Indiana University INDIANA UNIVERSITY is a major multi-campus public research institution, grounded in the liberal...
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4650 Sunset Blvd
Los Angeles, California 90027
 (323) 660-2450
Principal Investigator: Thomas Keens
Phone: 323-361-3255
Childrens Hospital Los Angeles Children's Hospital Los Angeles is a 501(c)(3) nonprofit hospital for pediatric...
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Minneapolis, Minnesota 55455
(612) 625-5000
Principal Investigator: Joanne Billings, MD
Phone: 612-626-7425
Univ of Minnesota With a flagship campus in the heart of the Twin Cities, and...
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116th St and Broadway
New York, New York 10027
(212) 854-1754
Principal Investigator: Emily DiMango, MD
Phone: 212-305-0251
Columbia University In 1897, the university moved from Forty-ninth Street and Madison Avenue, where it...
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South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Principal Investigator: Ronald Rubenstein, MD
Phone: 267-426-9626
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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4401 Penn Avenue
Pittsburgh, Pennsylvania 15224
412-692-5325
Principal Investigator: Joseph Pilewski, MD
Phone: 412-692-7042
Children's Hospital of Pittsburgh of UPMC UPMC is one of the leading nonprofit health systems...
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22 Bramhall St
Portland, Maine 04102
(207) 662-0111
Principal Investigator: Edmund Sears, MD
Phone: 207-662-5917
Maine Medical Center One of the country's consistently highest rated hospitals is right in your...
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3181 Southwest Sam Jackson Park Road
Portland, Oregon 97239
503 494-8311
Principal Investigator: Michael Powers, MD
Phone: 503-494-6052
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Sacramento, California 95814
Principal Investigator: Brian Morrissey, MD
Phone: 916-734-8686
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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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1 Tampa General Cir
Tampa, Florida 33606
(813) 844-7000
Principal Investigator: Mark Rolfe, MD
Phone: 813-844-7179
Tampa General Hospital In a diverse city known for its rich culture and beautiful beaches,...
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1100 N. St. Francis
Wichita, Kansas 67214
316-291-4774
Principal Investigator: Douglas Lewis, MD
Phone: 316-268-6028
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Akron, Ohio 44308
Principal Investigator: Greg Omlor, MD
Phone: 330-543-3089
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13123 E 16th Ave
Aurora, Colorado 80045
(720) 777-1234
Principal Investigator: Edith Zemanick, MD
Phone: 720-777-6162
Children's Hospital Colorado At Children's Hospital Colorado, we see more, treat more and heal more...
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Austin, Texas 78723
Principal Investigator: Bennie McWilliams, MD
Phone: 512-380-9200
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1800 Orleans St.
Baltimore, Maryland 21287
410-955-5000
Principal Investigator: Pamela Zeitlin, MD, PhD
Phone: 410-955-9782
Johns Hopkins Hospital Patients are the focus of everything we do at The Johns Hopkins...
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Birmingham, Alabama 35294
Principal Investigator: George Solomon, MD
Phone: 205-638-9410
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Boise, Idaho 83712
Principal Investigator: Karen Miller, MD
Phone: 208-381-7311
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300 Longwood Ave
Boston, Massachusetts 02115
(617) 355-6000
Principal Investigator: Alicia Casey, MD
Phone: 617-355-2446
Boston Children's Hospital Boston Children's Hospital is a 395-bed comprehensive center for pediatric health care....
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171 Ashley Avenue
Charleston, South Carolina 29425
843-792-1414
Principal Investigator: Patrick Flume, MD
Phone: 843-792-3162
Medical University of South Carolina The Medical University of South Carolina (MUSC) has grown from...
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303 East Superior Street
Chicago, Illinois 60611
Principal Investigator: Manu Jain, MD
Phone: 312-695-0415
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Cincinnati, Ohio 45229
Principal Investigator: John P Clancy, MD
Phone: 513-636-9780
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Cleveland, Ohio 44106
Principal Investigator: James Chmiel, MD
Phone: 216-844-8211
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1801 Inwood Rd
Dallas, Texas 75390
(214) 645-3300
Principal Investigator: Raksha Jain, MD
Phone: 214-648-2817
University of Texas Southwestern Medical Center UT Southwestern is an academic medical center, world-renowned for...
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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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Detroit, Michigan 48202
Principal Investigator: Dana Kissner, MD
Phone: 313-745-2221
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801 7th Avenue
Fort Worth, Texas 76104
(682) 885-4000
Principal Investigator: Karen Schultz, MD
Phone: 682-885-1244
Cook Children's Medical Center Cook Children's Health Care System is a not-for-profit, nationally recognized pediatric...
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Grand Rapids, Michigan 44506
Principal Investigator: Stephen Fitch, MD
Phone: 616-391-9366
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6621 Fannin St
Houston, Texas 77030
(832) 824-1000
Principal Investigator: Tara Barto, MD
Phone: 832-822-4772
Texas Children's Hospital Texas Children's Hospital, located in Houston, Texas, is a not-for-profit organization whose...
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Iowa City, Iowa 52242
Principal Investigator: Richard Ahrens, MD
Phone: 319-335-7555
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Lexington, Kentucky
859) 257-9000
Principal Investigator: Michael Anstead, MD
Phone: 859-218-4811
University of Kentucky The University of Kentucky is a public, land grant university dedicated to...
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Madison, Wisconsin 53792
Principal Investigator: Keith Meyer, MD
Phone: 608-263-4464
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Morristown, New Jersey 07962
Principal Investigator: Stanley Fiel, MD
Phone: 973-971-5138
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New Haven, Connecticut 6520
(203) 432-4771
Principal Investigator: Marie Egan, MD
Phone: 203-785-4971
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Emile St
Omaha, Nebraska 68198
(402) 559-4000
Principal Investigator: Peter Murphy, MD
Phone: 402-559-8296
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Palo Alto, California 94304
Principal Investigator: Richard Moss, MD
Phone: 650-736-0388
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Peoria, Illinois 61637
Principal Investigator: Subramanyam Chittivelu, MD
Phone: 309-624-2409
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4800 Sand Point Way NE
Seattle, Washington 98105
(206) 987-2000
Principal Investigator: Ronald Gibson, MD
Phone: 206-987-3921
Seattle Children's Hospital Seattle Children’s Hospital specializes in meeting the unique physical, emotional and developmental...
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3635 Vista at Grand Ave.
St. Louis, Missouri 63220
Principal Investigator: Ravi Nayak, MD
Phone: 314-678-5457
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Toledo, Ohio 43606
Principal Investigator: Bruce Barnett, MD
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Tucson, Arizona 85724
Phone: 520-626-0462
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Winston-Salem, North Carolina 26157
Principal Investigator: Victor Ortega, MD
Phone: 336-713-9190
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55 N Lake Ave
Worcester, Massachusetts 01655
(508) 856-8989
Principal Investigator: Evan Bailey, MD
Phone: 508-856-6970
Univ of Massachusetts Med School As the commonwealth's only public medical school, we take seriously...
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