NCT02871778: CLEAN-PCD

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 in Subjects With Primary Ciliary Dyskinesia

Completed Phase 2 Results posted Last updated 18 November 2021

What this trial tests

Phase 2 trial testing VX-371 in Primary Ciliary Dyskinesia in 123 participants. Completed in 20 November 2018.

Timeline

1 August 2016

Primary endpoint
20 November 2018

20 November 2018

Quick facts

Lead sponsor	Parion Sciences
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	crossover
Masking	triple
Primary purpose	treatment
Enrollment	123
Start date	1 August 2016
Primary completion	20 November 2018
Estimated completion	20 November 2018
Sites	34 locations across United States, Canada, Denmark, Germany, Italy, Netherlands, Poland, United Kingdom

Drugs / interventions tested

• VX-371 — full drug profile →
• Hypertonic Saline — full drug profile →
• Placebo (0.17% saline) — full drug profile →
• VX-371 + HS — full drug profile →
• Ivacaftor (IVACAFTOR) — full drug profile →

Conditions studied

• Primary Ciliary Dyskinesia — all drugs for Primary Ciliary Dyskinesia →

Sponsor

Parion Sciences — full company profile →

Who can join

12 and older, any sex, with Primary Ciliary Dyskinesia. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Part A: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs
  Time frame: Part A: From first dose of study drug up 84 days
  An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. AEs included abnormal clinically significant findings for spirometry, clinical laboratory parameters, standard 12-lead electrocardiograms (ECGs), vital signs and pulse oximetry examinations. Serious adverse event (SAE) was an AE resulting in any of the following
• Part B: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs
  Time frame: Part B: Day 85 up to 28 days after last dose of study drug (56 days)
  An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. AEs included abnormal clinically significant findings for spirometry, clinical laboratory parameters, standard 12-lead electrocardiograms (ECGs), vital signs and pulse oximetry examinations. SAE was an AE resulting in any of the following outcomes or deemed signi
• Part A: Absolute Change From Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 29
  Time frame: Part A: Study Baseline, Day 29 of each treatment period
  FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. The study baseline is defined as the most recent non-missing measurement (scheduled or unscheduled) collected before the first dose of study drug in the study.
• Part B: Absolute Change From Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 29
  Time frame: Study Baseline, Day 29 of Part B
  FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. The study baseline is defined as the most recent non-missing measurement (scheduled or unscheduled) collected before the first dose of study drug in the study.
• Part B: Absolute Change From Part B Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 29
  Time frame: Part B Baseline, Day 29 of Part B
  FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Part B baseline was defined as the most recent non-missing measurement (scheduled or unscheduled) collected before the first dose of ivacaftor in Part B and after the last dose in Period 2.

Sponsor's own description

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).

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Diagnosis of Primary Ciliary Dyskinesia. An Official American Thoracic Society Clinical Practice Guideline.
   Shapiro AJ, Davis SD, Polineni D, Manion M, et al · · 2018 · cited 329× · PMID 29905515 · DOI 10.1164/rccm.201805-0819st
2. Current and Future Treatments in Primary Ciliary Dyskinesia.
   Paff T, Omran H, Nielsen KG, Haarman EG. · · 2021 · cited 77× · PMID 34575997 · DOI 10.3390/ijms22189834
3. ENaC inhibition in cystic fibrosis: potential role in the new era of CFTR modulator therapies.
   Mall MA. · · 2020 · cited 56× · PMID 32732328 · DOI 10.1183/13993003.00946-2020
4. Clinical care for primary ciliary dyskinesia: current challenges and future directions.
   Rubbo B, Lucas JS. · · 2017 · cited 39× · PMID 28877972 · DOI 10.1183/16000617.0023-2017
5. The expanding phenotype of OFD1-related disorders: Hemizygous loss-of-function variants in three patients with primary ciliary dyskinesia.
   Hannah WB, DeBrosse S, Kinghorn B, Strausbaugh S, et al · · 2019 · cited 34× · PMID 31373179 · DOI 10.1002/mgg3.911
6. Why, when and how to investigate primary ciliary dyskinesia in adult patients with bronchiectasis.
   Contarini M, Shoemark A, Rademacher J, Finch S, et al · · 2018 · cited 24× · PMID 30151188 · DOI 10.1186/s40248-018-0143-6
7. Validation of pediatric health-related quality of life instruments for primary ciliary dyskinesia (QOL-PCD).
   Behan L, Leigh MW, Dell SD, Quittner AL, et al · · 2019 · cited 23× · PMID 31475479 · DOI 10.1002/ppul.24507
8. The Epithelial Sodium Channel-An Underestimated Drug Target.
   Lemmens-Gruber R, Tzotzos S. · · 2023 · cited 19× · PMID 37175488 · DOI 10.3390/ijms24097775