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

Cystic Fibrosis Clinical Trial

Official title:

A Phase I, Randomized, Placebo-Controlled, Study Designed to Assess the Safety, Tolerability, and Pharmacokinetics of PTI-428 in Subjects With Cystic Fibrosis.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03258424
• Other study ID #: PTI-428-05
• Status: Completed
• Phase: Phase 1
• Start date: July 28, 2017
• Est. completion date: October 3, 2018

Study information

• Verified date: March 2020
• Source: Proteostasis Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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 either PTI-428 or placebo. Each dose will be administered once daily (QD) for a total of 14 Days. The subjects will continue treatment with KALYDECO® throughout the study.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 15
• Est. completion date: October 3, 2018
• Est. primary completion date: October 3, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• 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 Criteria:

• 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.

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• PTI-428
PTI-428 or placebo will be given QD for 14 days.
• Placebo
PTI-428 or placebo will be given QD for 14 days.

Locations

Country	Name	City	State
United Kingdom	Celerion	Belfast
United Kingdom	Medicines Evaluation Unit	Manchester

Sponsors (1)

Lead Sponsor	Collaborator
Proteostasis Therapeutics, Inc.

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Change in nasal epithelial mRNA and protein expression over time		Baseline through Day 21
Other	Change in sweat chloride over time		Baseline through Day 21
Other	Change in FEV1 over time		Baseline through Day 21
Other	Change in weight over time		Baseline through Day 21
Primary	Safety and tolerability as assessed by adverse events, safety labs, electrocardiograms (ECGs), physical examinations, and vital signs		Baseline through Day 21
Secondary	t1/2 of multiple oral doses		Baseline through Day 14
Secondary	Tmax of multiple oral doses		Baseline through Day 14
Secondary	Cmax of multiple oral doses		Baseline through Day 14
Secondary	AUC0-t of multiple oral doses		Baseline through Day 14

External Links

•   Information about the sponsor