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ClinicalTrials.gov public records Last synced Oct 27, 2026, 2:52 PM EDT

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Showing 1–24 of 44 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: CFTR Gene Mutation Clear all
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 24, 2021 · Synced Oct 27, 2026, 2:52 PM EDT
Completed Phase 3 Interventional Results available

A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have an Ivacaftor-Responsive CFTR Mutation

NCT02725567
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
IVA
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
1 Month to 24 Months
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
13
States / cities
Birmingham, Alabama • Palo Alto, California • Atlanta, Georgia + 10 more
Source: ClinicalTrials.gov public record
Updated Sep 7, 2023 · Synced Oct 27, 2026, 2:52 PM 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Withdrawn Not applicable Interventional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 14, 2022 · Synced Oct 27, 2026, 2:52 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 31, 2015 · Synced Oct 27, 2026, 2:52 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 24, 2014 · Synced Oct 27, 2026, 2:52 PM EDT
Completed Phase 3 Interventional Results available

A Study to Evaluate the Safety and Efficacy of Long-term Treatment With TEZ/IVA in CF Participants With an F508del CFTR Mutation

NCT03537651
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
TEZ/IVA, IVA
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
6 Years and older
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
28
States / cities
Birmingham, Alabama • Anchorage, Alaska • Little Rock, Arkansas + 25 more
Source: ClinicalTrials.gov public record
Updated Apr 19, 2024 · Synced Oct 27, 2026, 2:52 PM EDT
Completed Phase 2 Interventional Results available

Study of VX-809 Alone and in Combination With VX-770 in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation

NCT01225211
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Lumacaftor, Ivacaftor, Lumacaftor Placebo, Ivacaftor Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years and older
Enrollment
312 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
38
States / cities
Birmingham, Alabama • Anchorage, Alaska • La Jolla, California + 35 more
Source: ClinicalTrials.gov public record
Updated Oct 5, 2015 · Synced Oct 27, 2026, 2:52 PM EDT
Terminated Phase 2 Interventional Results available

Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Homozygous for the F508del-CFTR Mutation

NCT00953706
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Ivacaftor, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years and older
Enrollment
140 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
34
States / cities
Birmingham, Alabama • Anchorage, Alaska • Oakland, California + 30 more
Source: ClinicalTrials.gov public record
Updated Sep 11, 2015 · Synced Oct 27, 2026, 2:52 PM EDT
Completed Phase 3 Interventional Results available

A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor

NCT02347657
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
VX-661 Plus Ivacaftor Combination, Ivacaftor, VX-661 Plus Ivacaftor Combination Placebo, Ivacaftor placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years and older
Enrollment
510 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
27
States / cities
Little Rock, Arkansas • Long Beach, California • Denver, Colorado + 24 more
Source: ClinicalTrials.gov public record
Updated Jun 12, 2018 · Synced Oct 27, 2026, 2:52 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 29, 2021 · Synced Oct 27, 2026, 2:52 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 12, 2018 · Synced Oct 27, 2026, 2:52 PM EDT
Terminated Phase 4 Interventional Results available

A Pilot Study to Evaluate the Use of Smart Adherence Technology to Measure Lumacaftor/Ivacaftor Adherence in CF Subjects Homozygous for the F508del CFTR Mutation

NCT02823470
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
LUM/IVA, activated smart device, de-activated smart device
Drug · Device
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
16 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
7
States / cities
Hartford, Connecticut • Washington D.C., District of Columbia • Orlando, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 27, 2018 · Synced Oct 27, 2026, 2:52 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 8, 2026 · Synced Oct 27, 2026, 2:52 PM EDT
Completed Phase 3 Interventional Results available

Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With Advanced Lung Disease

NCT02390219
View directory record Official record
Conditions
Cystic Fibrosis, Advanced Lung Disease
Interventions
Lumacaftor, Ivacaftor
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years and older
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
6
States / cities
Denver, Colorado • Tampa, Florida • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 6, 2017 · Synced Oct 27, 2026, 2:52 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 12, 2017 · Synced Oct 27, 2026, 2:52 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 7, 2016 · Synced Oct 27, 2026, 2:52 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 23, 2017 · Synced Oct 27, 2026, 2:52 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 7, 2020 · Synced Oct 27, 2026, 2:52 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 12, 2018 · Synced Oct 27, 2026, 2:52 PM 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 4, 2020 · Synced Oct 27, 2026, 2:52 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 24, 2025 · Synced Oct 27, 2026, 2:52 PM 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 5, 2016 · Synced Oct 27, 2026, 2:52 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 15, 2025 · Synced Oct 27, 2026, 2:52 PM EDT
Completed Phase 3 Interventional Results available

A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation

NCT05076149
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
VX-121/TEZ/D-IVA, ELX/TEZ/IVA, IVA, Placebo (matched to VX-121/TEZ/D-IVA), Placebo (matched to ELX/TEZ/IVA), Placebo (matched to IVA)
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years and older
Enrollment
597 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
62
States / cities
Birmingham, Alabama • Tucson, Arizona • Little Rock, Arkansas + 55 more
Source: ClinicalTrials.gov public record
Updated Jun 13, 2024 · Synced Oct 27, 2026, 2:52 PM EDT