Phase 3
Completed N=510
A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor
Source: ClinicalTrials.gov NCT02347657 ↗Enrolled (actual)
510
Serious AEs
15.3%
Results posted
Jun 2018
Primary outcomePrimary: Absolute Change From Baseline (Day 1) in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 24 — -0.6; 3.4 Percentage of predicted FEV1 — p=<0.0001
◆ Published Evidence
Highly cited
777citations · ~86 / year
Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del.
Summary
This is a Phase 3, randomized, double blind, placebo controlled, parallel group, multicenter study in people with cystic fibrosis (CF) who are homozygous for the F508del CF transmembrane conductance regulator (CFTR) gene mutation.
Linked Publications (2)
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Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del.
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Development of the Cystic Fibrosis Questionnaire-Revised-8 Dimensions: Estimating Utilities From the Cystic Fibrosis Questionnaire-Revised.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Absolute Change From Baseline (Day 1) in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 24 |
-0.6; 3.4 | <0.0001 sig |
| SECONDARY Relative Change From Baseline (Day 1) in ppFEV1 Through Week 24 |
-0.5; 6.3 | <0.0001 sig |
| SECONDARY Number of Pulmonary Exacerbations Per Year |
0.99; 0.64 | 0.0054 sig |
| SECONDARY Absolute Change From Baseline (Day 1) Body Mass Index (BMI) at Week 24 |
0.12; 0.18 | 0.4127 |
| SECONDARY Absolute Change From Baseline (Day 1) in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 24 |
-0.1; 5.0 | — |
| SECONDARY Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) |
245; 227; 47; 31 | — |
| SECONDARY Number of Participants With at Least One Pulmonary Exacerbation Pulmonary Exacerbation Through Week 24 |
88; 62 | — |
| SECONDARY Absolute Change From Baseline (Day 1) in Sweat Chloride Through Week 24 |
0.2; -9.9 | — |
| SECONDARY Absolute Change From Baseline (Day 1) in BMI Z-score at Week 24 in Participants Less Than (<) 20 Years Old at the Time of Screening) |
-0.02; -0.06 | — |
| SECONDARY Absolute Change From Baseline (Day 1) in Body Weight at Week 24 |
0.6; 0.7 | — |
| SECONDARY Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolites (M1 VX-661 and M2-VX-661), Ivacaftor (IVA) and IVA Metabolite (M1-IVA) |
1890; 4730; 4830; 815; 1590 | — |
Eligibility Criteria
Inclusion Criteria
- Homozygous for the F508del CFTR mutation, genotype to be confirmed at the Screening Visit
- Confirmed diagnosis of CF defined as a sweat chloride value ≥60 mmol/L by quantitative pilocarpine iontophoresis
- Forced expiratory volume at one second (FEV1) ≥40% and ≤90% of predicted normal for age, sex, and height during screening
- Stable CF disease as judged by the investigator
- Willing to remain on a stable CF medication regimen through Week 24 or, if applicable, the Safety Follow up Visit
Exclusion Criteria
- History of any comorbidity that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the participant.
- An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 28 days before Day 1 (first dose of study drug)
- Pregnant or nursing females (females of childbearing potential must have a negative pregnancy test at Screening and Day 1)
- Sexually active participants of reproductive potential who are not willing to follow the contraception requirements
Data sourced from ClinicalTrials.gov (NCT02347657) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.