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
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300 Longwood Ave
Boston, Massachusetts 02115
(617) 355-6000
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1 Perkins Square
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1720 2nd Ave S
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(205) 934-4011 
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185 Cambridge Street
Boston, Massachusetts 02114
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700 Childrens Drive
Columbus, Ohio 43205
(616) 722-2000
Phone: 614-722-4755
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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
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4401 Penn Avenue
Pittsburgh, Pennsylvania 15224
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Portland, Maine 04102
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201 Presidents Circle
Salt Lake City, Utah 84108
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40 Sunshine Cottage Road
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Ann Arbor, Michigan 48109
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171 Ashley Avenue
Charleston, South Carolina 29425
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303 East Superior Street
Chicago, Illinois 60611
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Denver, Colorado 80206
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New York, New York 10003
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630 W 168th St
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Tucson, Arizona 85721
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