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Trial record 1 of 1 for:    PS-G202
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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 April 2017 by Vertex Pharmaceuticals Incorporated
Sponsor:
Collaborator:
Parion Sciences
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT02871778
First received: August 12, 2016
Last updated: May 30, 2017
Last verified: April 2017
  Purpose
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).

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

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) 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 ]

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 in Part A and Part B [ Time Frame: Baseline, after 28 days of treatment ]

Estimated Enrollment: 150
Study Start Date: August 2016
Estimated Study Completion Date: April 2019
Estimated Primary Completion Date: March 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: VX-371 in Hypertonic Saline (HS), then HS, then HS + Ivacaftor

Part A/ Treatment Period 1: VX-371 in Hypertonic Saline

Part A/ Treatment Period 2: Hypertonic Saline

Part B/ Treatment Period 3: Hypertonic Saline + Ivacaftor

Drug: VX-371 Drug: Hypertonic Saline Drug: Ivacaftor
Experimental: HS, then VX-371 in HS, then VX-371 in HS + Ivacaftor

Part A/ Treatment Period 1: Hypertonic Saline

Part A/ Treatment Period 2: VX-371 in Hypertonic Saline

Part B/ Treatment Period 3: VX-371 in Hypertonic Saline + Ivacaftor

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

Part A/ Treatment Period 1: VX-371

Part A/ Treatment Period 2: Placebo

Part B/ Treatment Period 3: Placebo + Ivacaftor

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

Part A/ Treatment Period 1: Placebo

Part A/ Treatment Period 2: VX-371

Part B/ Treatment Period 3: VX-371 + Ivacaftor

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

  Eligibility

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

Inclusion Criteria:

  • The subject must have evidence supportive of a PCD diagnosis.
  • 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 potential difference (NPD) tests or presence of 2 CF-causing mutations in CFTR gene.
  • History of any organ transplantation or lung resection or chest wall surgery.
  • 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 and long-acting bronchodilator use the night before the first and last clinic visit of each treatment period.
  • 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 28 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

Additional Exclusion Criteria for Part B:

  • In addition to the exclusion criteria above, subjects who participate in Part B and meet any of the following exclusion criteria will not be eligible to continue into Part B
  • Unable to swallow tablets.
  • Concomitant use of strong or moderate inhibitors or inducers of cytochrome P450 (CYP) 3A, including consumption of certain herbal medications (e.g., St. John's Wort), and grapefruit/grapefruit juice.
  • Known hypersensitivity to ivacaftor.
  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 ClinicalTrials.gov identifier: NCT02871778

Contacts
Contact: Ashley Simmons 919-313-1210 asimmons@parion.com
Contact: Medical Information 617-341-6777 medicalinfo@vrtx.com

  Show 28 Study Locations
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
Parion Sciences
  More Information

Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT02871778     History of Changes
Other Study ID Numbers: PS-G202
2015-004917-26 ( EudraCT Number )
Study First Received: August 12, 2016
Last Updated: May 30, 2017

Additional relevant MeSH terms:
Dyskinesias
Ciliary Motility Disorders
Kartagener Syndrome
Movement Disorders
Central Nervous System Diseases
Nervous System Diseases
Neurologic Manifestations
Signs and Symptoms
Respiratory Tract Diseases
Otorhinolaryngologic Diseases
Bronchiectasis
Bronchial Diseases
Respiratory System Abnormalities
Dextrocardia
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Heart Diseases
Congenital Abnormalities
Situs Inversus
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on June 26, 2017