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Clearing Lungs With ENaC Inhibition in Primary Ciliary Dyskinesia (CLEAN-PCD)

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2017 by Vertex Pharmaceuticals Incorporated
Parion Sciences
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated Identifier:
First received: August 12, 2016
Last updated: February 14, 2017
Last verified: February 2017
To evaluate the safety and efficacy of treatment with VX-371, and the effect of VX-371 on quality of life (QOL) in subjects with primary ciliary dyskinesia (PCD).

Condition Intervention Phase
Primary Ciliary Dyskinesia
Drug: VX-371
Drug: Hypertonic Saline
Drug: Placebo (0.17% saline)
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Participant, Investigator, Outcomes Assessor
Primary Purpose: Treatment
Official Title: A Phase 2a, Randomized, Double-blind, Placebo-controlled, Incomplete Block Crossover Study to Evaluate the Safety and Efficacy of VX-371 Solution for Inhalation in Subjects With Primary Ciliary Dyskinesia

Resource links provided by NLM:

Further study details as provided by Vertex Pharmaceuticals Incorporated:

Primary Outcome Measures:
  • 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 ]

Secondary Outcome Measures:
  • 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 ]

Estimated Enrollment: 150
Study Start Date: August 2016
Estimated Study Completion Date: October 2018
Estimated Primary Completion Date: September 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: VX-371 in Hypertonic Saline, then Hypertonic Saline

Treatment Period 1: VX-371 in Hypertonic Saline

Treatment Period 2: Hypertonic Saline

Drug: VX-371 Drug: Hypertonic Saline
Experimental: Hypertonic Saline, then VX-371 in Hypertonic Saline

Treatment Period 1: Hypertonic Saline

Treatment Period 2: VX-371 in Hypertonic Saline

Drug: VX-371 Drug: Hypertonic Saline
Experimental: VX-371, then Placebo

Treatment Period 1: VX-371

Treatment Period 2: Placebo

Drug: VX-371 Drug: Placebo (0.17% saline)
Experimental: Placebo, then VX-371

Treatment Period 1: Placebo

Treatment Period 2: VX-371

Drug: VX-371 Drug: Placebo (0.17% saline)


Ages Eligible for Study:   12 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Confirmed diagnosis of PCD
  • Subjects with percent predicted FEV1 of ≥40 to <90 percentage points
  • Non-smoker for at least 90 days prior to the Screening Visit and less than a 5 pack-year lifetime history of smoking
  • Stable regimen of medications and chest physiotherapy for the 28 days prior to Day 1
  • If currently using daily inhaled HS, must be able to discontinue its use for the duration of the study.
  • If taking daily chronic or chronic cycling antibiotics, has been on a consistent regimen for at least 4 months prior to the Screening Visit.
  • Clinically stable (as deemed by the investigator) for at least 14 days prior to the Screening Visit
  • Female subjects of childbearing potential must have a negative serum pregnancy test at the Screening Visit. Subjects of childbearing potential and who are sexually active must meet the contraception requirements.

Exclusion Criteria:

  • Diagnosis of CF based on results of sweat chloride or nasal transepithelial potential difference (NPD) tests or presence of 2 CF-causing genetic mutations in CFTR gene.
  • History of any organ transplantation or lung resection or chest wall surgery of such severity that it has an impact on pulmonary function.
  • Significant congenital heart defects, other than a laterality defect, at the discretion of the investigator
  • Diagnosis of Cri du chat syndrome (chromosome 5p deletion syndrome).
  • Inability to withhold short-acting bronchodilator use for 4 hours prior to clinic visit.
  • Use of diuretics (including amiloride) or renin-angiotensin antihypertensive drugs
  • Symptoms of acute upper or lower respiratory tract infection, acute pulmonary exacerbation, or treatment or was treated with systemic antibiotics for ear or sinus disease within 14 days before Day 1 (topical otic antibiotics allowed).
  • History of significant intolerance to inhaled HS
  • Pregnant and/or nursing females
  • Any clinically significant laboratory abnormalities
  • History of chronic B. cepacia complex or M. abscessus or M. avium
  • Surgery that required general anesthesia and hospitalization within 3 months of Day 1
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT02871778

Contact: Ashley Simmons 919-313-1210
Contact: Medical Information 617-341-6777

  Show 24 Study Locations
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
Parion Sciences
  More Information

Responsible Party: Vertex Pharmaceuticals Incorporated Identifier: NCT02871778     History of Changes
Other Study ID Numbers: PS-G202
2015-004917-26 ( EudraCT Number )
Study First Received: August 12, 2016
Last Updated: February 14, 2017

Additional relevant MeSH terms:
Ciliary Motility Disorders
Kartagener Syndrome
Movement Disorders
Central Nervous System Diseases
Nervous System Diseases
Neurologic Manifestations
Signs and Symptoms
Respiratory Tract Diseases
Otorhinolaryngologic Diseases
Bronchial Diseases
Respiratory System Abnormalities
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Heart Diseases
Congenital Abnormalities
Situs Inversus
Genetic Diseases, Inborn processed this record on March 24, 2017