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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 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 Jun 27, 2026, 2:57 AM 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 Jun 27, 2026, 2:57 AM 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 Jun 27, 2026, 2:57 AM 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 Jun 27, 2026, 2:57 AM EDT
Completed Phase 3 Interventional Results available

A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of VX-661/Ivacaftor in Pediatric Subjects With Cystic Fibrosis (CF)

NCT02953314
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
TEZ, TEZ/IVA, IVA
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
6 Years to 11 Years
Enrollment
83 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
30
States / cities
Birmingham, Alabama • Anchorage, Alaska • Little Rock, Arkansas + 27 more
Source: ClinicalTrials.gov public record
Updated Mar 3, 2020 · Synced Jun 27, 2026, 2:57 AM 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 Jun 27, 2026, 2:57 AM 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 Jun 27, 2026, 2:57 AM EDT
Completed Not applicable Interventional

Safety of Stopping Pancreatic Enzyme Replacement Therapy in Children With Improved Pancreatic Function After Highly Effective Modulator Therapy

NCT07632768
View directory record Official record
Conditions
Cystic Fibrosis (CF), Pancreatic Insufficiency, Pancreas Disease, Exocrine Pancreatic Insufficiency, Exocrine Pancreatic Insufficiency in Subjects With Cystic Fibrosis, CFTR Gene Mutation
Interventions
Discontinuation of Pancreatic Enzyme Replacement Therapy
Drug
Lead sponsor
Indiana University
Other
Eligibility
0 Years to 18 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Jun 7, 2026 · Synced Jun 27, 2026, 2:57 AM EDT
Completed Phase 3 Interventional Results available

Rollover Study of Ivacaftor in Subjects With Cystic Fibrosis and a Non G551D CFTR Mutation

NCT01707290
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Ivacaftor
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
6 Years and older
Enrollment
125 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
33
States / cities
Birmingham, Alabama • Palo Alto, California • Denver, Colorado + 30 more
Source: ClinicalTrials.gov public record
Updated May 11, 2017 · Synced Jun 27, 2026, 2:57 AM 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 Jun 27, 2026, 2:57 AM EDT
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 Jun 27, 2026, 2:57 AM EDT
Completed Phase 3 Interventional Results available

A Study to Evaluate the Safety and Efficacy of Long Term Treatment With VX-661 in Combination With Ivacaftor in Participants With Cystic Fibrosis Who Have an F508del-CFTR Mutation

NCT02565914
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
TEZ/IVA, IVA
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years and older
Enrollment
1,131 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2022
U.S. locations
69
States / cities
Birmingham, Alabama • Tucson, Arizona • Little Rock, Arkansas + 66 more
Source: ClinicalTrials.gov public record
Updated Sep 27, 2023 · Synced Jun 27, 2026, 2:57 AM 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 Jun 27, 2026, 2:57 AM 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 Jun 27, 2026, 2:57 AM EDT
Withdrawn Not applicable Interventional

The Relationship Between CFTR Gene Mutations and Exercise Capacity

NCT05157646
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Fitness tracker
Behavioral
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Sep 13, 2022 · Synced Jun 27, 2026, 2:57 AM 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 Jun 27, 2026, 2:57 AM 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 Jun 27, 2026, 2:57 AM 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 Jun 27, 2026, 2:57 AM 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 Jun 27, 2026, 2:57 AM EDT
Completed Phase 3 Interventional Results available

Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have a Non-G551D CF Transmembrane Conductance Regulator (CFTR) Gating Mutation

NCT01614470
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Ivacaftor, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
6 Years and older
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
8
States / cities
Tampa, Florida • Atlanta, Georgia • Chicago, Illinois + 5 more
Source: ClinicalTrials.gov public record
Updated Oct 28, 2014 · Synced Jun 27, 2026, 2:57 AM EDT
Recruiting No phase listed Observational

Rare CFTR Mutation Cell Collection Protocol (RARE)

NCT03161808
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Not listed
Lead sponsor
George Solomon
Other
Eligibility
17 Years and older
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2028
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated May 28, 2026 · Synced Jun 27, 2026, 2:57 AM 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 Jun 27, 2026, 2:57 AM EDT
Completed Phase 3 Interventional Results available

Roll-Over Study of Ivacaftor in Cystic Fibrosis Pediatric Subjects With a CF Transmembrane Conductance Regulator Gene (CFTR) Gating Mutation

NCT01946412
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Ivacaftor
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
2 Years and older
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
12
States / cities
Birmingham, Alabama • Denver, Colorado • Atlanta, Georgia + 9 more
Source: ClinicalTrials.gov public record
Updated Jan 31, 2017 · Synced Jun 27, 2026, 2:57 AM EDT
Completed Phase 2 Interventional Results available

Study of VX-661 Alone and in Combination With Ivacaftor in Subjects Homozygous or Heterozygous to the F508del-Cystic Fibrosis Transmembrane Conductance Regulator(CFTR) Mutation

NCT01531673
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
12 Years and older
Enrollment
194 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
19
States / cities
Birmingham, Alabama • Oakland, California • Boise, Idaho + 16 more
Source: ClinicalTrials.gov public record
Updated Apr 12, 2018 · Synced Jun 27, 2026, 2:57 AM EDT