Status:
COMPLETED
Study Assessing PTI-428 Safety, Tolerability, and Pharmacokinetics in Subjects With Cystic Fibrosis on KALYDECO® as Background Therapy
Lead Sponsor:
Proteostasis Therapeutics, Inc.
Conditions:
Cystic Fibrosis
Eligibility:
All Genders
18+ years
Phase:
PHASE1
Brief Summary
The trial will consist of a single treatment group enrolling adult subjects with CF on background therapy with KALYDECO®. Approximately 16 subjects will be enrolled.
Detailed Description
The single treatment group will enroll adult subjects with CF currently on stable KALYDECO® background therapy for a minimum of 3 months at the time of randomization. Subjects will be randomized to ei...
Eligibility Criteria
Inclusion
- Confirmed diagnosis of CF.
- Stable on Kalydeco dosing for both label indication and per label dosing for a minimum of 3 months at the time of randomization
- Forced expiratory volume in 1 second (FEV1) 40-90% predicted.
- Non-smoker and non-tobacco user for a minimum of 30 days prior to screening and for the duration of the study.
Exclusion
- Participation in another clinical trial or treatment with an investigational agent within 30 days or 5 half-lives, whichever is longer, prior to Study Day 1.
- History of cancer within the past five years (excluding cervical CIS with curative therapy for at least one year prior to screening and non-melanoma skin cancer).
- History of organ transplantation.
- Any sinopulmonary infection or CF exacerbation requiring a change or addition of medication (including antibiotics) within 1 month of Study Day 1 or any other clinically significant infection as determined by the investigator within 1 month of Day 1.
- History of alcohol or drug abuse or dependence within 12 months of screening as determined by the Investigator.
- Male and female of child-bearing potential, unless they are using highly effective methods of contraception during participation in the clinical study and for 4 weeks after termination from study.
- Pregnant or nursing women.
Key Trial Info
Start Date :
July 28 2017
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 3 2018
Estimated Enrollment :
15 Patients enrolled
Trial Details
Trial ID
NCT03258424
Start Date
July 28 2017
End Date
October 3 2018
Last Update
March 26 2020
Active Locations (2)
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1
Celerion
Belfast, United Kingdom, BT9 6AD
2
Medicines Evaluation Unit
Manchester, United Kingdom