CompletedNCT03224351Updated Apr 22, 2021Results posted

A Study Evaluating the Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis

A Phase 2 interventional study of VX-659 and TEZ/IVA in Cystic Fibrosis, sponsored by Vertex Pharmaceuticals Incorporated. Completed at 47 sites in 4 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2021-04-22.

Sponsored by Vertex Pharmaceuticals Incorporated · Phase 2, Interventional, and Treatment

Phase: Phase 2
Study type: Interventional
Enrollment: 124
Allocation: Randomized
Ages: 18 Years and older
Sex: All

Study summary

This is a Phase 2, randomized, double-blind, placebo- and tezacaftor/ivacaftor (TEZ/IVA)-controlled, parallel-group, 3-part, multicenter study designed to evaluate the safety and efficacy of VX-659 in triple combination (TC) with TEZ and IVA in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation of the CF transmembrane conductance regulator (CFTR) gene (F/F genotype), or who are heterozygous for the F508del mutation and a minimal function (MF) CFTR mutation not likely to respond to TEZ, IVA, or TEZ/IVA (F/MF genotypes).

Conditions studied

Cystic Fibrosis

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In context

Cystic Fibrosis

1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.

This study's enrollment of 124 is above the median of 36 across 1,034 interventional studies indexed under Cystic Fibrosis.

Browse Cystic Fibrosis studies →

Lead sponsor

Vertex Pharmaceuticals Incorporated is the lead sponsor of 243 studies on the registry; 19 are open to participants now.

Of its 102 completed or terminated interventional studies of FDA-regulated products, 49 (48%) have results posted.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 18 Years and older
Sexes eligible: All
Accepts healthy volunteers: No

Eligibility criteria

Key Inclusion Criteria:

Body weight ≥35 kg.
Subjects must have an eligibleCFTR genotype.
- Part 1 and Part 3: Heterozygous for F508del and an MF mutation (F/MF)
- Part 2: Homozygous for F508del (F/F)
FEV1 value ≥40% and ≤90% of predicted mean for age, sex, and height

Key Exclusion Criteria:

History of clinically significant cirrhosis with or without portal hypertension.
Glucose-6-phosphate dehydrogenase (G6PD) deficiency
Lung infection with organisms associated with a more rapid decline in pulmonary status.
History of solid organ or hematological transplantation.

Other protocol defined Inclusion/Exclusion criteria may apply.

Study design

Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel assignment
Masking: Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment: 124 participants (actual)

Study arms

Placebo comparator
Part 1: Placebo
Participants received placebo matched to VX-659/TEZ/IVA in TC treatment period for 4 weeks and placebo matched TEZ/IVA in washout period for 4 days.
Drug: Placebo (matched to VX-659/TEZ/IVA)
Experimental
Part 1: VX-659/TEZ/IVA TC - Low Dose
Participants received VX-659 80 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in TC treatment period for 4 weeks and TEZ 100 mg qd/IVA 150 mg q12h in washout period for 4 days.
Drug: VX-659 · Drug: TEZ/IVA · Drug: IVA
Experimental
Part 1: VX-659/TEZ/IVA TC - Medium Dose
Participants received VX-659 240 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in TC treatment period for 4 weeks and TEZ 100 mg qd/IVA 150 mg q12h in washout period for 4 days.
Drug: VX-659 · Drug: TEZ/IVA · Drug: IVA
Experimental
Part 1: VX-659/TEZ/IVA TC - High Dose
Participants received VX-659 400 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in TC treatment period for 4 weeks and TEZ 100 mg qd/IVA 150 mg q12h in washout period for 4 days.
Drug: VX-659 · Drug: TEZ/IVA · Drug: IVA
Active comparator
Part 2: TEZ/IVA
Following run-in period with TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks, participants received TEZ 100 mg qd/IVA 150 mg q12h in TC treatment period for 4 weeks and TEZ 100 mg qd/IVA 150 mg q12h in washout period for 4 weeks.
Drug: TEZ/IVA · Drug: IVA
Experimental
Part 2: VX-659/TEZ/IVA TC
Following run-in period with TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks, participants received VX-659 400 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in TC treatment period for 4 weeks and TEZ 100 mg qd/IVA 150 mg q12h in washout period for 4 weeks.
Drug: VX-659 · Drug: TEZ/IVA · Drug: IVA
Placebo comparator
Part 3: Placebo
Participants received placebo matched to VX-659/TEZ/VX-561 in TC treatment period for 4 weeks.
Drug: Placebo (matched to VX-659/TEZ/VX-561)
Experimental
Part 3: VX-659/TEZ/VX-561 TC
Participants received VX-659 400 mg qd/TEZ 100 mg qd/VX-561 200 mg qd in TC treatment period for 4 weeks.
Drug: VX-659 · Drug: TEZ · Drug: VX-561

Interventions

DrugVX-659
Tablet for oral administration.
DrugTEZ/IVA
TEZ/IVA fixed-dose combination tablet for oral administration.
Also known as: VX-661/VX-770, Tezacaftor/Ivacaftor
DrugIVA
Tablet for oral administration.
Also known as: VX-770, Ivacaftor
DrugPlacebo (matched to VX-659/TEZ/IVA)
Placebo matched to VX-659 and TEZ/IVA.
DrugTEZ
Tablet for oral administration.
Also known as: VX-661, Tezacaftor
DrugVX-561
Tablet for oral administration.
Also known as: CTP-656
DrugPlacebo (matched to VX-659/TEZ/VX-561)
Placebo matched to VX-659, TEZ and VX-561.

What researchers measure

Primary outcomes

Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time frame: Day 1 Through Safety Follow-up (up to Day 61 for Part 1, Day 85 for Part 2 and Day 57 for Part 3)
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Time frame: From Baseline Through Day 29

Secondary outcomes

Absolute Change in Sweat Chloride Concentrations
Sweat samples were collected using an approved collection device.
Time frame: From Baseline Through Day 29
Relative Change in ppFEV1
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Time frame: From Baseline Through Day 29
Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score
The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
Time frame: From Baseline at Day 29
Observed Pre-dose Concentration (Ctrough) of VX-659, TEZ, M1-TEZ, IVA, M1-IVA, and VX-561
Time frame: Pre-dose at Day 15 and Day 29

Results

Posted Apr 22, 2021

Participant flow

Total of 124 participants were enrolled in this study (63 in Part 1, 36 in Part 2 and 25 in Part C). Out of 36 participants enrolled in Part 2, 7 participants discontinued treatment in run-in period and were not randomized in triple combination (TC) treatment period. Therefore, below results are presented for 117 participants.

Participant flow — Overall Study
Milestone	Part 1: Placebo	Part 1: VX-659/TEZ/IVA TC - Low Dose	Part 1: VX-659/TEZ/IVA TC - Medium Dose	Part 1: VX-659/TEZ/IVA TC - High Dose	Part 2: TEZ/IVA	Part 2: VX-659/TEZ/IVA TC	Part 3: Placebo	Part 3: VX-659/TEZ/VX-561 TC
Started	10	11	20	22	11	18	6	19
Completed	10	11	20	22	11	18	6	19
Not completed	0	0	0	0	0	0	0	0

Outcome measures

PrimarySafety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Time frame:: Day 1 Through Safety Follow-up (up to Day 61 for Part 1, Day 85 for Part 2 and Day 57 for Part 3)
Reported as:: Number · participants

Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
participants	Part 1: Placebo	Part 1: VX-659/TEZ/IVA TC - Low Dose	Part 1: VX-659/TEZ/IVA TC - Medium Dose	Part 1: VX-659/TEZ/IVA TC - High Dose	Part 2: TEZ/IVA	Part 2: VX-659/TEZ/IVA TC	Part 3: Placebo	Part 3: VX-659/TEZ/VX-561 TC
Participants with TEAEs	9	10	15	17	9	15	6	18
Participants with SAEs	3	1	4	1	2	1	3	2

PrimaryAbsolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Time frame:: From Baseline Through Day 29
Reported as:: Least squares mean · percentage points

Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
percentage points	Part 1: Placebo	Part 1: VX-659/TEZ/IVA TC - Low Dose	Part 1: VX-659/TEZ/IVA TC - Medium Dose	Part 1: VX-659/TEZ/IVA TC - High Dose	Part 2: TEZ/IVA	Part 2: VX-659/TEZ/IVA TC	Part 3: Placebo	Part 3: VX-659/TEZ/VX-561 TC
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)	0.4 (-5.3 to 6.1)	10.2 (4.8 to 15.5)	12.0 (8.0 to 16.0)	13.3 (9.5 to 17.1)	0.0 (-3.9 to 3.9)	9.7 (6.6 to 12.7)	-5.0 (-12.2 to 2.1)	12.2 (8.3 to 16.2)

SecondaryAbsolute Change in Sweat Chloride Concentrations

Sweat samples were collected using an approved collection device.

Time frame:: From Baseline Through Day 29
Reported as:: Least squares mean · millimole per liter (mmol/L)

Absolute Change in Sweat Chloride Concentrations
millimole per liter (mmol/L)	Part 1: Placebo	Part 1: VX-659/TEZ/IVA TC - Low Dose	Part 1: VX-659/TEZ/IVA TC - Medium Dose	Part 1: VX-659/TEZ/IVA TC - High Dose	Part 2: TEZ/IVA	Part 2: VX-659/TEZ/IVA TC	Part 3: Placebo	Part 3: VX-659/TEZ/VX-561 TC
Absolute Change in Sweat Chloride Concentrations	2.9 (-6.3 to 12.2)	-45.7 (-54.4 to -37.0)	-43.8 (-50.7 to -37.0)	-51.4 (-57.8 to -44.9)	3.0 (-2.8 to 8.9)	-42.2 (-46.8 to -37.7)	-1.3 (-12.4 to 9.8)	-38.1 (-44.4 to -31.8)

SecondaryRelative Change in ppFEV1

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Time frame:: From Baseline Through Day 29
Reported as:: Least squares mean · percent change

Relative Change in ppFEV1
percent change	Part 1: Placebo	Part 1: VX-659/TEZ/IVA TC - Low Dose	Part 1: VX-659/TEZ/IVA TC - Medium Dose	Part 1: VX-659/TEZ/IVA TC - High Dose	Part 2: TEZ/IVA	Part 2: VX-659/TEZ/IVA TC	Part 3: Placebo	Part 3: VX-659/TEZ/VX-561 TC
Relative Change in ppFEV1	0.0 (-10.5 to 10.4)	18.8 (8.9 to 28.7)	21.1 (13.8 to 28.5)	24.6 (17.6 to 31.6)	0.1 (-7.1 to 7.3)	17.3 (11.7 to 23.0)	-11.3 (-23.7 to 1.1)	21.5 (14.6 to 28.4)

SecondaryAbsolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score

The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.

Time frame:: From Baseline at Day 29
Reported as:: Least squares mean · units on a scale

Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score
units on a scale	Part 1: Placebo	Part 1: VX-659/TEZ/IVA TC - Low Dose	Part 1: VX-659/TEZ/IVA TC - Medium Dose	Part 1: VX-659/TEZ/IVA TC - High Dose	Part 2: TEZ/IVA	Part 2: VX-659/TEZ/IVA TC	Part 3: Placebo	Part 3: VX-659/TEZ/VX-561 TC
Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score	4.7 (-7.5 to 16.8)	24.6 (13.0 to 36.2)	19.8 (11.0 to 28.6)	21.8 (13.6 to 30.0)	2.9 (-5.2 to 11.1)	19.5 (13.1 to 25.9)	-4.1 (-17.8 to 9.6)	14.7 (7.1 to 22.4)

SecondaryObserved Pre-dose Concentration (Ctrough) of VX-659, TEZ, M1-TEZ, IVA, M1-IVA, and VX-561

Time frame:: Pre-dose at Day 15 and Day 29
Reported as:: Mean · nanogram per milliliter (ng/mL)

Observed Pre-dose Concentration (Ctrough) of VX-659, TEZ, M1-TEZ, IVA, M1-IVA, and VX-561
nanogram per milliliter (ng/mL)	Part 1: VX-659/TEZ/IVA TC - Low Dose	Part 1: VX-659/TEZ/IVA TC - Medium Dose	Part 1: VX-659/TEZ/IVA TC - High Dose	Part 2: TEZ/IVA	Part 2: VX-659/TEZ/IVA TC	Part 3: VX-659/TEZ/VX-561 TC
VX-659: Day 15	393 ± 604	622 ± 429	1100 ± 731	—	835 ± 474	1140 ± 646
VX-659: Day 29	566 ± 861	699 ± 489	1080 ± 582	—	1070 ± 914	923 ± 582
TEZ: Day 15	1910 ± 1360	1250 ± 907	1110 ± 455	1050 ± 473	1350 ± 1440	1000 ± 305
TEZ: Day 29	1910 ± 1230	1050 ± 553	1010 ± 479	1150 ± 480	955 ± 422	776 ± 321
M1-TEZ: Day 15	4390 ± 949	3710 ± 1230	4280 ± 1190	4160 ± 1260	4010 ± 1210	4060 ± 660
M1-TEZ: Day 29	4300 ± 774	3650 ± 1280	3870 ± 1020	3790 ± 1080	3810 ± 1120	3660 ± 1190
IVA: Day 15	824 ± 781	522 ± 567	423 ± 261	458 ± 239	313 ± 158	—
IVA: Day 29	719 ± 604	443 ± 398	371 ± 220	490 ± 179	296 ± 175	—
M1-IVA: Day 15	1200 ± 711	1050 ± 852	1170 ± 674	1310 ± 684	844 ± 622	—
M1-IVA Day 29	1130 ± 682	1140 ± 950	1030 ± 460	1240 ± 321	866 ± 691	—
VX-561: Day 15	—	—	—	—	—	380 ± 240
VX-561: Day 29	—	—	—	—	—	288 ± 165

Adverse events

Collected over Day 1 Through Safety Follow-up (up to Day 61 for Part 1, Day 85 for Part 2 and Day 57 for Part 3). Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
Group	Deaths	Serious	Other
Part 1: Placebo	0/10 (0%)	3/10 (30%)	8/10 (80%)
Part 1: VX-659/TEZ/IVA TC - Low Dose	0/11 (0%)	1/11 (9.1%)	10/11 (90.9%)
Part 1: VX-659/TEZ/IVA TC - Medium Dose	0/20 (0%)	4/20 (20%)	15/20 (75%)
Part 1: VX-659/TEZ/IVA TC - High Dose	0/22 (0%)	1/22 (4.5%)	17/22 (77.3%)
Part 2: TEZ/IVA	0/11 (0%)	2/11 (18.2%)	8/11 (72.7%)
Part 2: VX-659/TEZ/IVA TC	0/18 (0%)	1/18 (5.6%)	15/18 (83.3%)
Part 3: Placebo	0/6 (0%)	3/6 (50%)	5/6 (83.3%)
Part 3: VX-659/TEZ/VX-561 TC	0/19 (0%)	2/19 (10.5%)	18/19 (94.7%)

Most frequent serious events

Most frequent serious events
Event	Part 1: Placebo	Part 1: VX-659/TEZ/IVA TC - Low Dose	Part 1: VX-659/TEZ/IVA TC - Medium Dose	Part 1: VX-659/TEZ/IVA TC - High Dose	Part 2: TEZ/IVA	Part 2: VX-659/TEZ/IVA TC	Part 3: Placebo	Part 3: VX-659/TEZ/VX-561 TC
Infective pulmonary exacerbation of cystic fibrosisInfections and infestations	2/10	1/11	2/20	1/22	2/11	1/18	3/6	1/19
Pulmonary function test decreasedInvestigations	1/10	0/11	0/20	0/22	0/11	0/18	0/6	0/19
DyspnoeaRespiratory, thoracic and mediastinal disorders	0/10	0/11	0/20	0/22	0/11	0/18	0/6	1/19
Pleuritic painRespiratory, thoracic and mediastinal disorders	0/10	0/11	0/20	0/22	0/11	0/18	0/6	1/19
PyrexiaGeneral disorders	0/10	0/11	0/20	0/22	0/11	0/18	0/6	1/19
PneumoniaInfections and infestations	0/10	0/11	0/20	0/22	0/11	0/18	0/6	1/19
InfluenzaInfections and infestations	0/10	0/11	1/20	0/22	0/11	0/18	0/6	0/19
Respiratory tract infection viralInfections and infestations	0/10	0/11	1/20	0/22	0/11	0/18	0/6	0/19

Most frequent other events

Showing 10 of 121

Most frequent other events
Event	Part 1: Placebo	Part 1: VX-659/TEZ/IVA TC - Low Dose	Part 1: VX-659/TEZ/IVA TC - Medium Dose	Part 1: VX-659/TEZ/IVA TC - High Dose	Part 2: TEZ/IVA	Part 2: VX-659/TEZ/IVA TC	Part 3: Placebo	Part 3: VX-659/TEZ/VX-561 TC
CoughRespiratory, thoracic and mediastinal disorders	1/10	3/11	6/20	4/22	2/11	4/18	2/6	4/19
RalesRespiratory, thoracic and mediastinal disorders	0/10	0/11	0/20	0/22	0/11	0/18	2/6	0/19
Nasal congestionRespiratory, thoracic and mediastinal disorders	0/10	0/11	1/20	0/22	0/11	4/18	0/6	0/19
Infective pulmonary exacerbation of cystic fibrosisInfections and infestations	1/10	2/11	1/20	3/22	1/11	4/18	0/6	2/19
HeadacheNervous system disorders	0/10	1/11	4/20	4/22	0/11	3/18	1/6	1/19
FatigueGeneral disorders	0/10	2/11	2/20	0/22	1/11	0/18	1/6	0/19
Blood creatine phosphokinase increasedInvestigations	0/10	1/11	1/20	3/22	2/11	1/18	0/6	0/19
Sputum increasedRespiratory, thoracic and mediastinal disorders	0/10	2/11	1/20	3/22	1/11	3/18	0/6	1/19
Oropharyngeal painRespiratory, thoracic and mediastinal disorders	0/10	0/11	3/20	4/22	0/11	2/18	0/6	2/19
HaemoptysisRespiratory, thoracic and mediastinal disorders	0/10	2/11	1/20	0/22	1/11	1/18	1/6	0/19

Baseline characteristics

Age, Customized
Age, Customized(Participants)	Part 1: Placebo	Part 1: VX-659/TEZ/IVA TC - Low Dose	Part 1: VX-659/TEZ/IVA TC - Medium Dose	Part 1: VX-659/TEZ/IVA TC - High Dose	Part 2: TEZ/IVA	Part 2: VX-659/TEZ/IVA TC	Part 3: Placebo	Part 3: VX-659/TEZ/VX-561 TC	Total
<18 years	0	0	0	0	0	0	0	0	0
>=18 and <65 years	10	11	20	22	11	18	6	19	117
>=65 years	0	0	0	0	0	0	0	0	0

Sex: Female, Male
Sex: Female, Male(Participants)	Part 1: Placebo	Part 1: VX-659/TEZ/IVA TC - Low Dose	Part 1: VX-659/TEZ/IVA TC - Medium Dose	Part 1: VX-659/TEZ/IVA TC - High Dose	Part 2: TEZ/IVA	Part 2: VX-659/TEZ/IVA TC	Part 3: Placebo	Part 3: VX-659/TEZ/VX-561 TC	Total
Female	4	7	7	12	4	6	3	11	54
Male	6	4	13	10	7	12	3	8	63

Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)	Part 1: Placebo	Part 1: VX-659/TEZ/IVA TC - Low Dose	Part 1: VX-659/TEZ/IVA TC - Medium Dose	Part 1: VX-659/TEZ/IVA TC - High Dose	Part 2: TEZ/IVA	Part 2: VX-659/TEZ/IVA TC	Part 3: Placebo	Part 3: VX-659/TEZ/VX-561 TC	Total
Hispanic or Latino	0	0	2	0	1	1	1	1	6
Not Hispanic or Latino	10	11	18	22	10	17	5	18	111
Unknown or Not Reported	0	0	0	0	0	0	0	0	0

Race (NIH/OMB)
Race (NIH/OMB)(Participants)	Part 1: Placebo	Part 1: VX-659/TEZ/IVA TC - Low Dose	Part 1: VX-659/TEZ/IVA TC - Medium Dose	Part 1: VX-659/TEZ/IVA TC - High Dose	Part 2: TEZ/IVA	Part 2: VX-659/TEZ/IVA TC	Part 3: Placebo	Part 3: VX-659/TEZ/VX-561 TC	Total
American Indian or Alaska Native	0	0	0	0	0	0	0	0	0
Asian	0	0	0	0	0	1	0	0	1
Native Hawaiian or Other Pacific Islander	0	0	0	0	0	0	0	0	0
Black or African American	0	0	0	0	0	0	0	2	2
White	10	11	20	22	11	16	6	17	113
More than one race	0	0	0	0	0	0	0	0	0
Unknown or Not Reported	0	0	0	0	0	1	0	0	1

Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)(Participants)	Part 1: Placebo	Part 1: VX-659/TEZ/IVA TC - Low Dose	Part 1: VX-659/TEZ/IVA TC - Medium Dose	Part 1: VX-659/TEZ/IVA TC - High Dose	Part 2: TEZ/IVA	Part 2: VX-659/TEZ/IVA TC	Part 3: Placebo	Part 3: VX-659/TEZ/VX-561 TC	Total
<40 percent	2	0	1	2	0	1	0	2	8
≥40 to <70 percent	6	9	13	13	8	12	5	13	79
≥70 to ≤90 percent	2	2	6	7	3	5	1	4	30
>90 percent	0	0	0	0	0	0	0	0	0

Study locations

47 sites

Yale New Haven Hospital
New Haven, Connecticut 06520, United States
University of Miami/Miller School of Medicine
Miami, Florida 33136, United States
Advocate Children's Hospital - Park Ridge / North Suburban Pulmonary and Critical Care Consultants
Morton Grove, Illinois 60053, United States
Indiana University Health
Indianapolis, Indiana 46202, United States
The University of Iowa Hospitals and Clinics
Iowa City, Iowa 52242, United States
The Johns Hopkins Hospital/ Johns Hopkins Hospital, David Rubenstein Child Health Building
Baltimore, Maryland 21287, United States
Boston Children's Hospital
Boston, Massachusetts 02155, United States
University of Massachusetts Memorial Medical Center
Worcester, Massachusetts 01655, United States
University of Michigan Health System
Ann Arbor, Michigan 48109, United States
Helen DeVos Children's Hospital CF Center
Grand Rapids, Michigan 49503, United States
Children's Mercy Hospital
Kansas City, Missouri 64108, United States
Rutgers-Robert Wood Johnson Medical School/ Rutgers-Robert Wood Johnson Medical School, Clinical Research Center
New Brunswick, New Jersey 08902, United States
Albany Medical College
Albany, New York 12208, United States
Northwell Health, Long Island Jewish Medical Center
New Hyde Park, New York 11040, United States
Columbia University Medical Center
New York, New York 10032, United States
SUNY Upstate Medical University
Syracuse, New York 13210, United States
Respiratory Diseases of Children & Adolescents
Oklahoma City, Oklahoma 73112, United States
Children's Hospital of Pittsburgh of University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania 15224, United States
Sanford Research / USD
Sioux Falls, South Dakota 57104, United States
University of Tennessee Medical Center-Adult Cystic Fibrosis Clinic
Knoxville, Tennessee 37920, United States
Children's Foundation Research Center / Le Bonheur Children's Hospital
Memphis, Tennessee 38103, United States
Vanderbilt University Medical Center
Nashville, Tennessee 37232, United States
University of Utah / Primary Children's Medical Center
Salt Lake City, Utah 84014, United States
Seattle Children's Hospital
Seattle, Washington 98105, United States
Providence Pediatric Pulmonary & Allergy/Immunology Clinic
Spokane, Washington 99204, United States
Cork University Hospital
Cork, Ireland
St. Vincent's University Hosptial
Dublin, Ireland
Galway University Hospitals
Galway, Ireland
University Hospital Limerick
Limerick, Ireland
Carmel Medical Center
Haifa, Israel
Ruth Children's Hospital Rambam Health Care Campus
Haifa, Israel
Hadassah Medical Organization
Jerusalem, Israel
The Chaim Sheba medical center
Ramat Gan, Israel
Schneider Children's Medical Center
Tikvah, Israel
Birmingham Heartlands Hospital
Birmingham, B95SS, United Kingdom
Papworth Hospital NHS Foundation Trust, Papworth Everard
Cambridge, United Kingdom
University Hospital Llandough in Cardiff
Cardiff, United Kingdom
Royal Devon and Exeter NHS Foundation Trust, Royal Devon and Exeter Hospital
Devon, United Kingdom
The Newcastle upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary
Fulham, United Kingdom
Greater Glasgow and Clyde NHS Board, Glasgow Clinical Research Facility
Glasgow, United Kingdom
Southampton University Hospitals NHS Foundation Trust
Hampshire, United Kingdom
Regional Respiratory Centre Belfast City Hospital
London, United Kingdom
Royal Brompton & Harefied NHS Foundation Trust
London, United Kingdom
University Hospital of South Manchester NHS Trust, North West Lung Centre
Manchester, United Kingdom
Liverpool Heart and Chest Hospital
Merseyside, United Kingdom
Nottingham University Hospitals NHS Trust
Nottingham, United Kingdom
Ruth Children's Hospital Rambam Health Care Campus
Nottingham, United Kingdom

References and documents

Publications

Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3. PubMed 33331662 ↗
Davies JC, Moskowitz SM, Brown C, Horsley A, Mall MA, McKone EF, Plant BJ, Prais D, Ramsey BW, Taylor-Cousar JL, Tullis E, Uluer A, McKee CM, Robertson S, Shilling RA, Simard C, Van Goor F, Waltz D, Xuan F, Young T, Rowe SM; VX16-659-101 Study Group. VX-659-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles. N Engl J Med. 2018 Oct 25;379(17):1599-1611. doi: 10.1056/NEJMoa1807119. Epub 2018 Oct 18. PubMed 30334693 ↗

Study documents

Study protocol · Sep 1, 2017
Statistical analysis plan · Sep 19, 2017

Documents are hosted by the registry — open the source record to download them.

Updates

Tracking since Sep 25, 2026

No changes since tracking began. The registry record was last updated on Apr 22, 2021, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT03224351
Lead sponsor: Vertex Pharmaceuticals Incorporated
Responsible party: Sponsor
First posted: Jul 21, 2017
Start date: Aug 8, 2017
Primary completion: Feb 28, 2018
Completion: Feb 28, 2018
Results posted: Apr 22, 2021
Last update: Apr 22, 2021

Oversight

Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No

View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Feb 2021. You cannot join it, but the record below documents what was studied.

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Discussion

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