Study Assessing PTI-428 Safety, Tolerability, and Pharmacokinetics in Subjects With Cystic Fibrosis on KALYDECO® as Background Therapy

NCT03258424 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: PTI-428-05
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 2

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.

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

• Safety and tolerability as assessed by adverse events, safety labs, electrocardiograms (ECGs), physical examinations, and vital signs

  Baseline through Day 21

Secondary Outcomes (4)

• t1/2 of multiple oral doses

  Baseline through Day 14

• Tmax of multiple oral doses

  Baseline through Day 14

• Cmax of multiple oral doses

  Baseline through Day 14

• AUC0-t of multiple oral doses

  Baseline through Day 14

Other Outcomes (4)

• Change in nasal epithelial mRNA and protein expression over time

  Baseline through Day 21

• Change in sweat chloride over time

  Baseline through Day 21

• Change in FEV1 over time

  Baseline through Day 21

Study Arms (2)

PTI-428

ACTIVE COMPARATOR

Subjects will receive once daily dosing of PTI-428 or placebo for 14 days.

Drug: PTI-428

Placebo

PLACEBO COMPARATOR

Subjects will receive once daily dosing of PTI-428 or placebo for 14 days.

Drug: Placebo

Interventions

PTI-428 DRUG

PTI-428 or placebo will be given QD for 14 days.

PTI-428

Placebo DRUG

PTI-428 or placebo will be given QD for 14 days.

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Proteostasis Therapeutics, Inc.: lead

Study Sites (2)

Celerion

Belfast, BT9 6AD, United Kingdom

Location

Medicines Evaluation Unit

Manchester, United Kingdom

Location

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 18, 2017
• First Posted: August 23, 2017
• Study Start: July 28, 2017
• Primary Completion: October 3, 2018
• Study Completion: October 3, 2018
• Last Updated: March 26, 2020

Record last verified: 2020-03

Data Sharing

• IPD Sharing: Will share

Locations

GB (2)