Study Assessing PTI-428 Safety, Tolerability, Pharmacokinetics and Effect in Subjects With Cystic Fibrosis



Status:Recruiting
Conditions:Pulmonary
Therapuetic Areas:Pulmonary / Respiratory Diseases
Healthy:No
Age Range:18 - Any
Updated:11/17/2018
Start Date:August 21, 2018
End Date:February 2019
Contact:Proteostasis Clinical Trials
Email:pticlinicaltrials@proteostasis.com
Phone:1-866-223-3262

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A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Effect of PTI-428 in Subjects With Cystic Fibrosis

The study population is comprised of adult subjects with cystic fibrosis (CF) who are
homozygous for the F508del mutation and are currently receiving background treatment with
tezacaftor/ivacaftor for a minimum of 1 month prior to Day 1. The planned sample size is
approximately 40 subjects. 20 subjects will be assigned to PTI-428 dose level 1 or placebo
and 20 subjects will be assigned to PTI-428 dose level 2 or placebo. At each dose level,
subjects will be randomized at a 3:1 randomization ratio. Subjects will receive once daily
oral doses of PTI-428 or placebo for 28 days, while the subjects continue to receive
background treatment with tezacaftor/ivacaftor per product label. The study drug
administration period will be followed by a 14-day safety follow-up period.


Inclusion Criteria:

- Confirmed diagnosis of CF with the F508del/F508del genotype on record

- On tezacaftor/ivacaftor dosing for both label indication and per label dosing for a
minimum of 1 month on Day 1

- Forced expiratory volume in 1 second (FEV1) 40-90% predicted, inclusive

- Clinically stable with no significant changes in health status within 14 days of Day 1

- Non-smoker and non-tobacco user for a minimum of 28 days prior to screening and for
the duration of the study

Exclusion Criteria:

- Participation in another clinical trial or treatment with an investigational agent
within 28 days or 5 half-lives, whichever is longer, prior to Study Day 1

- History of cancer within the past 5 years (excluding cervical cancer in situ with
curative therapy for at least one year prior to screening and non-melanoma skin
cancer)

- History of organ transplantation

- Hospitalization, sinopulmonary infection, CF exacerbation, or other clinically
significant infection or illness (as determined by the investigator) requiring an
increase or addition of medication, such as antibiotics or corticosteroids, within 14
days of Day 1

- Initiation of any new chronic therapy (e.g., ibuprofen, hypertonic saline,
azithromycin, Pulmozyme®, Cayston®, TOBI®)) or any change in chronic therapy
(excluding pancreatic enzyme replacement therapy) within 28 days prior to Day 1

- History or current evidence of alcohol or drug abuse or dependence within 12 months of
screening as determined by the investigator

- Pregnant or nursing women
We found this trial at
26
sites
1 Perkins Square
Akron, Ohio 44308
(330) 543-1000
Akron Children's Hospital From humble beginnings as a day nursery in 1890, Akron Children
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1720 2nd Ave S
Birmingham, Alabama 35233
(205) 934-4011 
University of Alabama at Birmingham The University of Alabama at Birmingham (UAB) traces its roots...
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185 Cambridge Street
Boston, Massachusetts 02114
617-724-5200
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700 Childrens Drive
Columbus, Ohio 43205
(616) 722-2000
Phone: 614-722-4755
Nationwide Children's Hospital At Nationwide Children’s, we are creating the future of pediatric health care....
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2401 Gillham Rd
Kansas City, Missouri 64108
(816) 234-3000
Children's Mercy Hospital Children's Mercy Hospitals and Clinics continues redefining pediatric medicine throughout the Midwest...
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Minneapolis, Minnesota 55455
(612) 625-5000
Univ of Minnesota With a flagship campus in the heart of the Twin Cities, and...
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4401 Penn Avenue
Pittsburgh, Pennsylvania 15224
412-692-5325
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22 Bramhall St
Portland, Maine 04102
(207) 662-0111
Maine Medical Center One of the country's consistently highest rated hospitals is right in your...
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201 Presidents Circle
Salt Lake City, Utah 84108
801) 581-7200
University of Utah Research is a major component in the life of the U benefiting...
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40 Sunshine Cottage Road
Valhalla, New York 10595
(914) 594-4000
New York Medical College The College was founded in 1860 by a group of New...
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Altamonte Springs, Florida 32701
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1150 East Medical Center Drive
Ann Arbor, Michigan 48109
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300 Longwood Ave
Boston, Massachusetts 02115
(617) 355-6000
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
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303 East Superior Street
Chicago, Illinois 60611
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1400 Jackson St
Denver, Colorado 80206
(303) 388-4461
National Jewish Health National Jewish Health is known worldwide for treatment of patients with respiratory,...
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2301 Erwin Rd
Durham, North Carolina 27710
919-684-8111
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Manchester, New Hampshire 03104
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New York, New York 10003
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630 W 168th St
New York, New York
212-305-2862
Columbia University Medical Center Situated on a 20-acre campus in Northern Manhattan and accounting for...
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450 Serra Mall
Stanford, California 94305
(650) 723-2300
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Tucson, Arizona 85721
(520) 621-2211
University of Arizona The University of Arizona is a premier, public research university. Established in...
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