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Showing 1–24 of 44 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: CFTR Gene Mutation Clear all
Completed Phase 3 Interventional Results available

Study of Ivacaftor in Cystic Fibrosis Subjects 2 Through 5 Years of Age With a CFTR Gating Mutation

NCT01705145
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Ivacaftor
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
2 Years to 5 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
16
States / cities
Birmingham, Alabama • Aurora, Colorado • Atlanta, Georgia + 13 more
Source: ClinicalTrials.gov public record
Updated Apr 4, 2016 · Synced Sep 7, 2026, 10:16 PM EDT
Completed Phase 2 Interventional Results available

A Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation

NCT02709109
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
VX-371 + HS, Hypertonic saline, Placebo, Orkambi, VX-371
Drug
Lead sponsor
Parion Sciences
Industry
Eligibility
12 Years and older
Enrollment
147 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
30
States / cities
Little Rock, Arkansas • Oakland, California • Gainesville, Florida + 27 more
Source: ClinicalTrials.gov public record
Updated Jul 28, 2021 · Synced Sep 7, 2026, 10:16 PM EDT
Recruiting Phase 2 Interventional

Safety, Tolerability and Efficacy Study of ARCT-032 in People With Cystic Fibrosis

NCT06747858
View directory record Official record
Conditions
Cystic Fibrosis, CFTR Gene Mutation
Interventions
ARCT-032
Biological
Lead sponsor
Arcturus Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
13
States / cities
Tucson, Arizona • Little Rock, Arkansas • Los Angeles, California + 10 more
Source: ClinicalTrials.gov public record
Updated May 7, 2026 · Synced Sep 7, 2026, 10:16 PM EDT
Completed Phase 2 Interventional Results available

A Phase 2 Study to Evaluate Effects of VX-661/Ivacaftor on Lung and Extrapulmonary Systems in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

NCT02508207
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Tezacaftor/Ivacaftor, Ivacaftor, Tezacaftor/Ivacaftor matching placebo, Ivacaftor matching placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
7
States / cities
Birmingham, Alabama • Indianapolis, Indiana • Baltimore, Maryland + 4 more
Source: ClinicalTrials.gov public record
Updated Jul 18, 2021 · Synced Sep 7, 2026, 10:16 PM EDT
Not listed Phase 2 Interventional

Study of Cavosonstat (N91115) in CF Patients Who Are Heterozygous for F508del-CFTR and a Gating Mutation and Being Treated With Ivacaftor

NCT02724527
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Cavosonstat, Placebo
Drug
Lead sponsor
Nivalis Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
12
States / cities
Denver, Colorado • Baltimore, Maryland • Boston, Massachusetts + 9 more
Source: ClinicalTrials.gov public record
Updated Nov 20, 2016 · Synced Sep 7, 2026, 10:16 PM EDT
Completed Phase 1 Interventional

Study of N91115 in Patients With Cystic Fibrosis Homozygous F508del-CFTR Mutation

NCT02275936
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
N91115
Drug
Lead sponsor
Nivalis Therapeutics, Inc.
Industry
Eligibility
18 Years to 80 Years
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015
U.S. locations
19
States / cities
Birmingham, Alabama • Palo Alto, California • Aurora, Colorado + 15 more
Source: ClinicalTrials.gov public record
Updated Nov 6, 2016 · Synced Sep 7, 2026, 10:16 PM EDT
Completed Phase 3 Interventional Results available

A Phase 3 Rollover Study of Lumacaftor in Combination With Ivacaftor in Subjects 12 Years and Older With Cystic Fibrosis

NCT01931839
View directory record Official record
Conditions
Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation
Interventions
Lumacaftor Plus Ivacaftor Combination, Ivacaftor
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years and older
Enrollment
1,164 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
91
States / cities
Birmingham, Alabama • Anchorage, Alaska • Tucson, Arizona + 88 more
Source: ClinicalTrials.gov public record
Updated May 11, 2017 · Synced Sep 7, 2026, 10:16 PM EDT
Completed Phase 3 Interventional Results available

A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

NCT03125395
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
LUM/IVA
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
2 Years and older
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
17
States / cities
Palo Alto, California • Aurora, Colorado • Chicago, Illinois + 14 more
Source: ClinicalTrials.gov public record
Updated Aug 6, 2020 · Synced Sep 7, 2026, 10:16 PM EDT
Completed Phase 3 Interventional Results available

A Phase 3 Study to Evaluate the Efficacy and Safety of Ivacaftor and VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Mutation

NCT02392234
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
VX-661/Ivacaftor, Ivacaftor, Placebo matched to VX-661/ ivacaftor, Placebo matched to Ivacaftor
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years and older
Enrollment
248 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
47
States / cities
Birmingham, Alabama • Phoenix, Arizona • Tucson, Arizona + 44 more
Source: ClinicalTrials.gov public record
Updated Jun 11, 2018 · Synced Sep 7, 2026, 10:16 PM EDT
Completed Phase 3 Interventional Results available

Study of Lumacaftor in Combination With Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

NCT01897233
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Lumacaftor, Ivacaftor
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
6 Years to 11 Years
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
19
States / cities
Birmingham, Alabama • Tucson, Arizona • Long Beach, California + 16 more
Source: ClinicalTrials.gov public record
Updated Jun 19, 2017 · Synced Sep 7, 2026, 10:16 PM EDT
Completed Phase 1 Interventional

Safety Study of Orally Administered Curcuminoids in Adult Subjects With Cystic Fibrosis

NCT00219882
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
standardized turmeric root extract
Drug
Lead sponsor
Ramsey, Bonnie, MD
Individual
Eligibility
18 Years to 40 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
2
States / cities
Baltimore, Maryland • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Dec 24, 2007 · Synced Sep 7, 2026, 10:16 PM EDT
Active, not recruiting No phase listed Observational

Ivacaftor Impact in an Adolescent Without a Cystic Fibrosis-Associated CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) Mutation for Chronic Pancreatitis: Single-Patient Study Tracking Flare Frequency and Health Data Compared to Historical Records.

NCT07022327
View directory record Official record
Conditions
Chronic Pancreatitis
Interventions
Ivacaftor 150 MG (milligrams)
Drug
Lead sponsor
Mission: Cure
Other
Eligibility
Not listed
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Jun 14, 2025 · Synced Sep 7, 2026, 10:16 PM EDT
Completed Phase 3 Interventional Results available

A Phase 3 Study of Tezacaftor (VX-661) in Combination With Ivacaftor (VX-770) in Subjects Aged 12 Years and Older With Cystic Fibrosis (CF), Who Have One F508del-CFTR Mutation and a Second Mutation That Has Been Demonstrated to be Clinically Responsive to Ivacaftor

NCT02412111
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Ivacaftor, Tezacaftor/Ivacaftor
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years and older
Enrollment
156 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
33
States / cities
Birmingham, Alabama • Oakland, California • San Diego, California + 30 more
Source: ClinicalTrials.gov public record
Updated Jan 7, 2019 · Synced Sep 7, 2026, 10:16 PM EDT
Completed Phase 1 Interventional Results available

Exploratory Study to Evaluate QR-010 in Subjects With Cystic Fibrosis ΔF508 CFTR Mutation

NCT02564354
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
QR-010
Drug
Lead sponsor
ProQR Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
3
States / cities
Birmingham, Alabama • Denver, Colorado • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 23, 2020 · Synced Sep 7, 2026, 10:16 PM EDT
Completed Phase 3 Interventional Results available

A Study to Evaluate Safety, Efficacy, and Tolerability of TEZ/IVA in Orkambi® (Lumacaftor/Ivacaftor) -Experienced Subjects With Cystic Fibrosis (CF)

NCT03150719
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Tezacaftor/Ivacaftor, Ivacaftor, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years and older
Enrollment
98 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
41
States / cities
Birmingham, Alabama • Phoenix, Arizona • Fresno, California + 34 more
Source: ClinicalTrials.gov public record
Updated Sep 11, 2019 · Synced Sep 7, 2026, 10:16 PM EDT
Completed Phase 1 Interventional Results available

Safety and Pharmacokinetic Study of N6022 in Subjects With Cystic Fibrosis Homozygous for the F508del-CFTR Mutation

NCT01746784
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
N6022, Normal saline
Drug
Lead sponsor
Nivalis Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014
U.S. locations
17
States / cities
Birmingham, Alabama • Anchorage, Alaska • Palo Alto, California + 14 more
Source: ClinicalTrials.gov public record
Updated Nov 23, 2014 · Synced Sep 7, 2026, 10:16 PM EDT
Completed Phase 3 Interventional Results available

A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation

NCT01807923
View directory record Official record
Conditions
Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
Interventions
Lumacaftor Plus Ivacaftor Combination, Ivacaftor, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years and older
Enrollment
559 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
48
States / cities
Birmingham, Alabama • Anchorage, Alaska • Tucson, Arizona + 45 more
Source: ClinicalTrials.gov public record
Updated Aug 30, 2015 · Synced Sep 7, 2026, 10:16 PM EDT
Completed Phase 2 Interventional Results available

Study of VX-809 in Cystic Fibrosis Subjects With the ∆F508-CFTR Gene Mutation

NCT00865904
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
VX-809, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years and older
Enrollment
93 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009
U.S. locations
18
States / cities
Birmingham, Alabama • Palo Alto, California • San Diego, California + 15 more
Source: ClinicalTrials.gov public record
Updated Aug 27, 2015 · Synced Sep 7, 2026, 10:16 PM EDT
Completed Phase 2 Interventional Results available

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
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
VX-661, Ivacaftor, Placebo matched to VX-661, Placebo matched to Ivacaftor
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
23
States / cities
Birmingham, Alabama • Palo Alto, California • Stanford, California + 20 more
Source: ClinicalTrials.gov public record
Updated Sep 23, 2025 · Synced Sep 7, 2026, 10:16 PM EDT
Completed Phase 3 Interventional Results available

A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation

NCT01807949
View directory record Official record
Conditions
Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
Interventions
Placebo, Lumacaftor Plus Ivacaftor Combination, Ivacaftor
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years to 65 Years
Enrollment
563 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
46
States / cities
Oakland, California • Sacramento, California • Aurora, Colorado + 43 more
Source: ClinicalTrials.gov public record
Updated Sep 26, 2016 · Synced Sep 7, 2026, 10:16 PM EDT
Completed Phase 2 Interventional Results available

Safety Study of Ivacaftor in Subjects With Cystic Fibrosis

NCT00457821
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Ivacaftor 25 mg/75 mg, Ivacaftor 75 mg/150 mg, Ivacaftor 150 mg or 250 mg, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years and older
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
13
States / cities
Birmingham, Alabama • Palo Alto, California • Aurora, Colorado + 9 more
Source: ClinicalTrials.gov public record
Updated Oct 4, 2012 · Synced Sep 7, 2026, 10:16 PM EDT
Completed Phase 3 Interventional Results available

A Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation

NCT02516410
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
VX-661 plus ivacaftor combination, Ivacaftor, Placebo (matched to VX-661 plus ivacaftor combination), Placebo (matched to ivacaftor)
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years and older
Enrollment
168 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
22
States / cities
Birmingham, Alabama • La Jolla, California • Los Angeles, California + 19 more
Source: ClinicalTrials.gov public record
Updated Jun 11, 2018 · Synced Sep 7, 2026, 10:16 PM EDT
Completed Phase 3 Interventional Results available

Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor

NCT02544451
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
LUM/IVA
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
6 Years and older
Enrollment
246 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
37
States / cities
Birmingham, Alabama • Tucson, Arizona • Long Beach, California + 34 more
Source: ClinicalTrials.gov public record
Updated May 23, 2021 · Synced Sep 7, 2026, 10:16 PM EDT
Completed Phase 3 Interventional Results available

A Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects With CF, Homozygous for the F508del-CFTR Mutation

NCT02514473
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
VX-809, Placebo, VX-770
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
6 Years to 11 Years
Enrollment
206 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
29
States / cities
Birmingham, Alabama • Los Angeles, California • Palo Alto, California + 26 more
Source: ClinicalTrials.gov public record
Updated Oct 22, 2017 · Synced Sep 7, 2026, 10:16 PM EDT