Phase 3
Completed N=168
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
Source: ClinicalTrials.gov NCT02516410 ↗Enrolled (actual)
168
Serious AEs
14.9%
Results posted
Jun 2018
Primary outcomePrimary: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 12 — -0.1; 1 Percentage of predicted FEV1 — p== 0.1176
◆ Published Evidence
Established
35citations · ~6 / year
Tezacaftor/ivacaftor in people with cystic fibrosis heterozygous for minimal function CFTR mutations.
Summary
Study to evaluate the efficacy of VX-661 in combination with ivacaftor (IVA, VX-770) through Week 12 in participants with cystic fibrosis (CF) who are heterozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene and with a second CFTR mutation that is not likely to respond to VX-661 and/or IVA therapy (F508del/not responsive [NR]).
Linked Publications
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Tezacaftor/ivacaftor in people with cystic fibrosis heterozygous for minimal function CFTR mutations.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 12 |
-0.1; 1 | = 0.1176 |
| SECONDARY Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 12 |
3.8; 5.9 | — |
| SECONDARY Number of Pulmonary Exacerbation Events |
23; 22 | — |
| SECONDARY Number of Pulmonary Exacerbation Events Per Year |
0.98; 0.97 | — |
| SECONDARY Absolute Change From Baseline in Body Mass Index (BMI) at Week 12 |
0.22; 0.14 | — |
| SECONDARY Relative Change From Baseline in Percent Predicted FEV1 Through Week 12 |
0.1; 2.1 | — |
| SECONDARY Absolute Change From Baseline in Sweat Chloride Through Week 12 |
-1.2; -4.7 | — |
| SECONDARY Number of Participants With at Least One Pulmonary Exacerbation Through Week 12 |
21; 19 | — |
| SECONDARY Absolute Change From Baseline in BMI Z-score at Week 12 (in Participants Less Than [<] 20 Years Old at the Time of Screening) |
0.07; 0.02 | — |
| SECONDARY Absolute Change From Baseline in Body Weight at Week 12 |
0.7; 0.6 | — |
| SECONDARY Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) |
68; 64; 14; 11 | — |
| SECONDARY Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolite (M1 VX-661), Ivacaftor (IVA) and IVA Metabolite (M1-IVA) |
1880; 4600; 831; 1580; 1780; 4420 | — |
Eligibility Criteria
Inclusion Criteria
- Confirmed diagnosis of CF defined as a sweat chloride value greater than or equal to (>=)60 millimole per liter (mmol/L) by quantitative pilocarpine iontophoresis.
- Heterozygous for the F508del-CFTR mutation and with a second CFTR mutation that is not likely to respond to VX-661 and/or ivacaftor therapy, genotype to be confirmed via assessment at the Screening Visit.
- Forced Expiratory Volume in 1 Second (FEV1) >=40 percent (%) and less than or equal to (<=)90% of predicted normal for age, sex, and height at Screening 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).
- History of solid organ or hematological transplantation.
- Ongoing or prior participation in an investigational drug study or use of commercially available CFTR modulator within 30 days of screening.
- Pregnant or nursing females.
Data sourced from ClinicalTrials.gov (NCT02516410) 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.