Clinical Trial: Clearing Lungs With ENaC Inhibition in Primary Ciliary Dyskinesia

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Phase 2a, 2-part,Randomized, Double-blind, Placebo-controlled, Incomplete Block Crossover Study to Evaluate the Safety and Efficacy of VX-371 Solution for Inhalation With and Without Oral Ivacaftor

Brief Summary: To evaluate the safety and efficacy of treatment with VX-371 with and without ivacaftor, and the effect of VX-371 with and without ivacaftor on quality of life (QOL) in subjects with primary ciliary dyskinesia (PCD).

Detailed Summary:
Sponsor: Vertex Pharmaceuticals Incorporated

Current Primary Outcome:

  • Safety and Tolerability assessments as determined by number of subjects with adverse events (AEs) and serious adverse events (SAEs) in Part A and Part B [ Time Frame: From the first dose of study drug in each treatment period to the first day of the next treatment period and for 28 days after the last dose in the study ]
  • Absolute change in percent predicted FEV1 in Part A and Part B [ Time Frame: Baseline, after 28 days of treatment ]


Original Primary Outcome:

  • Safety and Tolerability assessments as determined by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: From the first dose of study drug to 28 days after the last dose in the same period ]
  • Absolute change in percent predicted FEV1 [ Time Frame: Baseline, after 28 days of treatment ]


Current Secondary Outcome: Change in Quality of Life (QOL), as determined by mean change in QOL-PCD and SGRQ (St. George's Respiratory Questionnaire) scores in Part A and Part B [ Time Frame: Baseline, after 28 days of treatment ]

Original Secondary Outcome: Change in Quality of Life (QOL), as determined by mean change in QOL-PCD and SGRQ (St. George's Respiratory Questionnaire) scores. [ Time Frame: Baseline, after 28 days of treatment ]

Information By: Vertex Pharmaceuticals Incorporated

Dates:
Date Received: August 12, 2016
Date Started: August 2016
Date Completion: April 2019
Last Updated: April 14, 2017
Last Verified: April 2017