NCT03258424

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

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

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 15 (actual)
• Sponsor: Proteostasis Therapeutics, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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 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.

Conditions

• Cystic Fibrosis

Interventions

Type	Name	Description
DRUG	PTI-428	PTI-428 or placebo will be given QD for 14 days.
DRUG	Placebo	PTI-428 or placebo will be given QD for 14 days.

Timeline

• Start date: 2017-07-28
• Primary completion: 2018-10-03
• Completion: 2018-10-03
• First posted: 2017-08-23
• Last updated: 2020-03-26

Locations

2 sites across 1 country: United Kingdom

Regulatory

• FDA-regulated drug study