A Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation
NCT ID: NCT02709109
Last Updated: 2021-07-29
Study Results
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
COMPLETED
PHASE2
147 participants
INTERVENTIONAL
2016-02-29
2017-09-30
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Study to Evaluate Safety and Efficacy of VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation With an Open-Label Expansion
NCT02070744
Study of VX-809 in Cystic Fibrosis Subjects With the ∆F508-CFTR Gene Mutation
NCT00865904
A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)
NCT05668741
Study of VX-121 in Healthy Subjects and in Subjects With Cystic Fibrosis
NCT03768089
A Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic Fibrosis
NCT03912233
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
CROSSOVER
TREATMENT
TRIPLE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Sequence 1: VX-371 + Hypertonic Saline (HS), then HS
Participants received 85 microgram (mcg) VX-371 diluted in 3 milliliter (mL) 4.2 percent (%) HS through oral nebulized inhalation twice daily for 28 days (Day 1 to Day 28) in treatment period 1 followed by a 28 days washout period (Day 29 to Day 56) and then received 3 mL 4.2% HS through oral nebulized inhalation twice daily for 28 days (Day 57 to Day 84) in treatment period 2. Participants also received a stable dose of 2 tablets of Orkambi (lumacaftor 400 milligram (mg)/ivacaftor 250 mg) orally every 12 hours with fat-containing food throughout the study as part of their cystic fibrosis (CF) standard of care.
VX-371 + HS
Hypertonic saline
Orkambi
Sequence 2: HS, then VX-371 + HS
Participants received 3 mL 4.2% HS through oral nebulized inhalation twice daily for 28 days (Day 1 to Day 28) in treatment period 1 followed by a 28 days washout period (Day 29 to Day 56) and then received 85 mcg VX-371 diluted in 3 mL 4.2% HS through oral nebulized inhalation twice daily for 28 days (Day 57 to Day 84) in treatment period 2. Participants also received a stable dose of 2 tablets of Orkambi (lumacaftor 400 mg/ivacaftor 250 mg) orally every 12 hours with fat-containing food throughout the study as part of their CF standard of care.
VX-371 + HS
Hypertonic saline
Orkambi
Sequence 3: VX-371 + Placebo, then Placebo
Participants received 85 mcg VX-371 diluted in 3 mL 0.17% Saline (placebo) through oral nebulized inhalation twice daily for 28 days (Day 1 to Day 28) in treatment period 1 followed by a 28 days washout period (Day 29 to Day 56) and then received 3 mL 0.17% saline (placebo) through oral nebulized inhalation twice daily for 28 days (Day 57 to Day 84) in treatment period 2. Participants also received a stable dose of 2 tablets of Orkambi (lumacaftor 400 mg/ivacaftor 250 mg) orally every 12 hours with fat-containing food throughout the study as part of their CF standard of care.
Placebo
Orkambi
VX-371
Sequence 4: Placebo, then VX-371 + Placebo
Participants received 3 mL 0.17% saline (placebo) through oral nebulized inhalation twice daily for 28 days (Day 1 to Day 28) in treatment period 1 followed by a 28 days washout period (Day 29 to Day 56) and then received 85 mcg VX-371 diluted in 3 mL 0.17% saline (placebo) through oral nebulized inhalation twice daily for 28 days (Day 57 to Day 84) in treatment period 2. Participants also received a stable dose of 2 tablets of Orkambi (lumacaftor 400 mg/ivacaftor 250 mg) orally every 12 hours with fat-containing food throughout the study as part of their CF standard of care.
Placebo
Orkambi
VX-371
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
VX-371 + HS
Hypertonic saline
Placebo
Orkambi
VX-371
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* Confirmed diagnosis of CF, defined as a sweat chloride value ≥60 mmol/L by quantitative pilocarpine iontophoresis.
* Homozygous for the F508del CFTR mutation. If the CFTR screening genotype result is not received before randomization, a previous CFTR genotype lab report may be used to establish eligibility.
* Percent predicted forced expiratory volume at one second (FEV1) of ≥40 to \<90 percentage points adjusted for age, sex, and height according to the Global Lung Initiative (GLI) at the Screening Visit.
* Willing to discontinue physician-prescribed saline use.
* Female subjects of childbearing potential with a negative serum pregnancy test at the Screening Visit.
Exclusion Criteria
* Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject.
* An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 28 days before Day 1 (first dose of study drug).
* A 12 lead ECG demonstrating QTcF \>450 msec at the Screening Visit.
* History of solid organ or hematological transplantation.
* Used diuretics or renin-angiotensin aldosterone system antihypertensive drugs in the 28 days prior to Screening or an anticipated need for any of these medications during the study.
* Ongoing or prior participation in an investigational drug study within 30 days of the Screening Visit.
* Inability to withhold short-acting, long-acting, or once-daily, long-acting bronchodilator use for 4, 12, or 24 hours prior to clinic visit, respectively.
* History of significant intolerance to inhaled saline, or intolerance to the single dose of saline at Screening
* Known hypersensitivity or history of intolerance to Orkambi.
* Pregnant and nursing females.
* Subjects who have participated in Parion Sciences Study NCT02343445.
12 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Vertex Pharmaceuticals Incorporated
INDUSTRY
Parion Sciences
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Alison Church, MD
Role: STUDY_CHAIR
Parion Sciences
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Little Rock, Arkansas, United States
Oakland, California, United States
Gainesville, Florida, United States
Miami, Florida, United States
Orlando, Florida, United States
Chicago, Illinois, United States
Glenview, Illinois, United States
Kansas City, Kansas, United States
New Orleans, Louisiana, United States
Worcester, Massachusetts, United States
Detroit, Michigan, United States
Grand Rapids, Michigan, United States
Minneapolis, Minnesota, United States
Jackson, Mississippi, United States
Lebanon, New Hampshire, United States
Albuquerque, New Mexico, United States
Albany, New York, United States
Hawthorne, New York, United States
Syracuse, New York, United States
Charlotte, North Carolina, United States
Cleveland, Ohio, United States
Pittsburgh, Pennsylvania, United States
Austin, Texas, United States
Dallas, Texas, United States
Fort Worth, Texas, United States
Tyler, Texas, United States
Charlottesville, Virginia, United States
Richmond, Virginia, United States
Madison, Wisconsin, United States
Milwaukee, Wisconsin, United States
Bron, , France
Pierre-Bénite, , France
Roscoff, , France
Strasbourg, , France
Dublin, , Ireland
Birmingham, , United Kingdom
Bristol, , United Kingdom
London, , United Kingdom
Manchester, , United Kingdom
Southampton, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
Provided Documents
Download supplemental materials such as informed consent forms, study protocols, or participant manuals.
Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
2015-004841-13
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
VX15-371-101
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.