NCT03500263

Safety, Tolerability, and Pharmacokinetics of PTI-808, PTI-801, and PTI-428 Combination Therapy in Subjects With Cystic Fibrosis

A Phase 1 / 2, Randomized, Double-Blind, Placebo-Controlled Study Designed to Evaluate the Safety, Tolerability, and Pharmacokinetics of PTI-808, PTI-801, and PTI-428 Combination Therapy in Subjects With Cystic Fibrosis

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: Proteostasis Therapeutics, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The study is a randomized, double-blind, placebo-controlled, study that will be conducted at multiple centers in subjects with Cystic Fibrosis (CF) who are either homozygous for the F508del mutation or heterozygous with at least copy of the F508del mutation.

Detailed description

Study PTI-808-02 will enroll up to approximately 32 subjects. Subjects in the first cohort will receive PTI-808 and PTI-801. Following completion of Cohort 1, initiation of enrollment into subsequent cohorts will be based upon review and approval by the Safety Review Committee (SRC).

Conditions

• Cystic Fibrosis

Interventions

Type	Name	Description
DRUG	PTI-808	Active
DRUG	Placebo	Placebo
DRUG	PTI-801	Active
DRUG	PTI-428	Active
DRUG	Placebo	Placebo

Timeline

• Start date: 2018-01-30
• Primary completion: 2019-03-13
• Completion: 2019-03-13
• First posted: 2018-04-18
• Last updated: 2020-04-06

Locations

4 sites across 1 country: United Kingdom

Regulatory

• FDA-regulated drug study