Status:
COMPLETED
Study Designed to Assess the Safety, Tolerability and PK of PTI-808 in Healthy Volunteers and in Adults With Cystic Fibrosis
Lead Sponsor:
Proteostasis Therapeutics, Inc.
Conditions:
Healthy Volunteer - Complete
Cystic Fibrosis - Complete
Eligibility:
All Genders
18-99 years
Phase:
PHASE1
PHASE2
Brief Summary
Part 1 of this trial will enroll healthy volunteers into a single ascending dose (SAD), multiple ascending dose (MAD), and Food Effect (FE) treatment groups. The SAD treatment group is comprised of a...
Detailed Description
Part 1 of this trial will enroll healthy volunteers into a single ascending dose (SAD), multiple ascending dose (MAD), and Food Effect (FE) treatment groups. The SAD treatment group is comprised of a...
Eligibility Criteria
Inclusion
- Part 1 and Part 2
- Adults aged 18 to 55 years old, inclusive, at the time of informed consent
- Body mass index ≥18 and \<30 kg/m2
- Subject must be a non-smoker and non-tobacco user for a minimum of 30 days prior to screening and for the duration of the study.
- Subject understands the full nature and purpose of the study, including possible risks and side effects, and is willing and able to comply with all compulsory study procedures and provides informed consent/permission prior to any study procedures being performed.
- Females of childbearing potential and males capable of fathering a child must meet the contraception requirements
- Part 1 \& Part 2
Exclusion
- History or current evidence of any clinically significant cardiac, endocrinologic, hematologic, hepatobiliary, immunologic, metabolic, urologic, pulmonary, neurologic, dermatologic, psychiatric, renal, or other major disease, as determined by the investigator
- Prolonged QT interval with Fridericia's correction \>450 msec at screening
- Abnormal liver function as defined by aspartate transaminase (AST), alanine transaminase (ALT), or bilirubin \>1.5× the upper limit of the normal range
- Abnormal renal function at screening defined as creatinine clearance \<90 mL/min using the Cockroft-Gault equation
- Clinically significant screening results that would exclude subject from the study (e.g., medical histories, PE, ECGs, vital signs, and laboratory profiles) as deemed by the investigator
- Participation in another clinical study or treatment with an investigational agent within 30 days or five half-lives, whichever is longer, prior to Study Day 1
- History of cancer within the past 5 years (excluding non-melanoma skin cancer)
- History or current evidence of alcohol or drug abuse or dependence within 12 months of screening as determined by the investigator
- Positive urine screen for prohibited drugs (cocaine, cannabinoids, nicotine \[urine cotinine is the detection mechanism for nicotine\], opiates, barbiturates, amphetamines, and benzodiazepines) or positive alcohol test at screening
- Positive blood screen for human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg), or hepatitis C virus antibody (HCVAb)
- Clinically significant infection within 3 months of screening as determined by the investigator
- Known or suspected hypersensitivity or idiosyncratic reaction to study medication or any components thereof
- Has donated blood within 3 months of screening or plans to donate blood within 3 months of study completion
- Pregnant or nursing women
- Any conditions that, in the opinion of the investigator, would make the subject unsuitable for enrollment or could interfere with the subject's participation in or completion of the study
- Use of prohibited medications within 14 days prior to dosing of study drug
- Part 3 CF Inclusion Criteria:
- Confirmed diagnosis of CF with the F508del/F508del genotype
- Forced expiratory volume in 1 second (FEV1) 40-90% predicted, inclusive
- Non-smoker and non-tobacco user for a minimum of 30 days prior to screening
- Part 3 CF
Key Trial Info
Start Date :
July 17 2017
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 23 2019
Estimated Enrollment :
179 Patients enrolled
Trial Details
Trial ID
NCT03251092
Start Date
July 17 2017
End Date
December 23 2019
Last Update
April 22 2020
Active Locations (48)
Enter a location and click search to find clinical trials sorted by distance.
1
Banner University of Arizona Medical Center
Tucson, Arizona, United States, 85724
2
Stanford University Medical Center
Stanford, California, United States, 94305
3
National Jewish Health
Denver, Colorado, United States, 80206
4
Central Florida Pulmonary Group
Altamonte Springs, Florida, United States, 32803