Phase 2
Completed N=34
A Phase 2 Study to Evaluate Effects of VX-661/Ivacaftor on Lung and Extrapulmonary Systems in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
Source: ClinicalTrials.gov NCT02508207 ↗Enrolled (actual)
34
Serious AEs
0.0%
Results posted
Jul 2018
Primary outcomePrimary: Absolute Change From Baseline in Mucociliary Clearance (MCC) at Day 28 — -0.5; -0.9 percentage of whole-lung clearance — p=0.7151
Summary
To evaluate the clinical mechanisms of action in lung and extrapulmonary systems of VX-661 (tezacaftor; TEZ) in combination with ivacaftor (IVA) (TEZ/IVA) in participants with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Absolute Change From Baseline in Mucociliary Clearance (MCC) at Day 28 |
-0.5; -0.9 | 0.7151 |
| SECONDARY Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28 |
-0.4; 2.4 | 0.0004 sig |
| SECONDARY Absolute Change From Baseline in Small-bowel Area Under the Curve (AUC) Over 1-minute Mean pH Increments at Day 29 |
0.3; -0.2 | 0.3345 |
| SECONDARY Absolute Change From Baseline in Sweat Chloride at Day 29 |
-0.2; -8.4 | 0.0002 sig |
| SECONDARY Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) |
0; 0; 2; 24 | — |
Eligibility Criteria
Inclusion Criteria
- Male and female participants, homozygous for the F508del CFTR mutation
- Confirmed diagnosis of CF by sweat chloride testing
- Forced Expiratory Volume in 1 Second (FEV1) ≥40% and ≤90% of predicted normal for age, sex, and height at Screening Visit
- 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 subject.
- An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 28 days before Day 1
- History or evidence of clinically significant findings on ophthalmologic examination during the Screening Period.
- History of solid organ or hematological transplantation
- Pregnant or nursing females
- Participants who have had radiation exposure within 1 year before the first mucociliary clearance (MCC) procedure that would cause them to exceed federal regulations by participating in this study
- In the opinion of the investigator, unable to adequately perform inhalation maneuvers during the MCC procedures
Data sourced from ClinicalTrials.gov (NCT02508207). 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.