Phase 3
Completed N=248
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
Source: ClinicalTrials.gov NCT02392234 ↗Enrolled (actual)
248
Serious AEs
6.7%
Results posted
Jun 2018
Primary outcomePrimary: Absolute Change From Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Average of Week 4 and Week 8 — -0.3; 4.4; 6.5 percentage of predicted FEV1 — p=< 0.0001
◆ Published Evidence
Highly cited
511citations · ~57 / year
Tezacaftor-Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis.
Summary
The purpose of this study is to evaluate the efficacy and safety of VX-661 in combination with ivacaftor (IVA, VX-770) and IVA monotherapy in participants with Cystic Fibrosis (CF) who are heterozygous for F508del-CFTR allele and a second allele with a CFTR mutation predicted to have residual function.
Linked Publications (4)
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Tezacaftor-Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis.
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Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).
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Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).
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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 Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Average of Week 4 and Week 8 |
-0.3; 4.4; 6.5 | < 0.0001 sig |
| SECONDARY Absolute Change From Study Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Average of Week 4 and Week 8 |
-1.0; 8.7; 10.1 | <0.0001 sig |
| SECONDARY Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) |
126; 114; 117; 14; 10; 8 | — |
| SECONDARY Relative Change From Study Baseline in ppFEV1 at Average of Week 4 and Week 8 |
-0.2; 7.9; 11.2 | <0.0001 sig |
| SECONDARY Absolute Change From Study Baseline in Sweat Chloride at Average of Week 4 and Week 8 |
-0.4; -4.9; -9.9 | <0.0001 sig |
| SECONDARY Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolites (M1 VX-661), IVA and IVA Metabolite (M1 IVA) After Administration of VX-661/IVA Combination Therapy |
2370; 5230; 909; 2010 | — |
| SECONDARY Ctrough of IVA and IVA Metabolite (M1 IVA) After Administration of IVA Monotherapy |
696; 1550 | — |
Eligibility Criteria
Inclusion Criteria
- Heterozygous for F508del-CFTR and a second allele with a CFTR mutation predicted to have residual function
- Forced Expiratory Volume in 1 Second (FEV1) greater than or equal to (≥) 40 percent (%) and less than or equal to (≤) 90% of predicted normal for age, sex, and height during screening
- Sweat chloride value ≥60 millimole per liter (mmol/L) during screening OR as documented in the participant's medical record
- Stable CF disease as judged by the investigator
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
- History of solid organ or hematological transplantation
- Ongoing or prior participation in an investigational drug study (including studies investigating VX-661, lumacaftor [VX-809], and/or ivacaftor) within 30 days of screening
- Pregnant and nursing females
- Sexually active participants of reproductive potential who are not willing to follow the contraception requirements
Data sourced from ClinicalTrials.gov (NCT02392234) 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.