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ClinicalTrials.gov public records Last synced Jun 27, 2026, 4:33 AM EDT

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Showing 25–44 of 20 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

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 Jun 27, 2026, 4:33 AM 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 5, 2017 · Synced Jun 27, 2026, 4:33 AM 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 10, 2015 · Synced Jun 27, 2026, 4:33 AM 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 12, 2024 · Synced Jun 27, 2026, 4:33 AM 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 Jun 27, 2026, 4:33 AM 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 Jun 27, 2026, 4:33 AM 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 Jun 27, 2026, 4:33 AM EDT
Completed Phase 3 Interventional Results available

Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have the R117H-CF Transmembrane Conductance Regulator (CFTR) Mutation (KONDUCT)

NCT01614457
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Ivacaftor, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
6 Years and older
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
29
States / cities
Birmingham, Alabama • Palo Alto, California • Hartford, Connecticut + 26 more
Source: ClinicalTrials.gov public record
Updated Feb 11, 2015 · Synced Jun 27, 2026, 4:33 AM 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 Jun 27, 2026, 4:33 AM EDT
Completed Phase 2 Interventional

Study of Cavosonstat (N91115) in Patients With CF Homozygous for the F508del-CFTR Mutation

NCT02589236
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Cavosonstat, Placebo
Drug
Lead sponsor
Nivalis Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
138 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
46
States / cities
Birmingham, Alabama • Tucson, Arizona • Los Angeles, California + 42 more
Source: ClinicalTrials.gov public record
Updated Jan 9, 2017 · Synced Jun 27, 2026, 4:33 AM 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 Jun 27, 2026, 4:33 AM 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 Jun 27, 2026, 4:33 AM 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 Jun 27, 2026, 4:33 AM EDT
Completed Phase 3 Interventional Results available

Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del

NCT02797132
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
LUM/IVA
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
2 Years to 5 Years
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
17
States / cities
Palo Alto, California • Aurora, Colorado • Chicago, Illinois + 14 more
Source: ClinicalTrials.gov public record
Updated Oct 29, 2018 · Synced Jun 27, 2026, 4:33 AM 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 18, 2024 · Synced Jun 27, 2026, 4:33 AM 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 Jun 27, 2026, 4:33 AM 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 11, 2018 · Synced Jun 27, 2026, 4:33 AM 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 26, 2018 · Synced Jun 27, 2026, 4:33 AM 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 4, 2015 · Synced Jun 27, 2026, 4:33 AM 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 6, 2023 · Synced Jun 27, 2026, 4:33 AM EDT