Phase 3
Completed N=382
A Phase 3 Study of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
Source: ClinicalTrials.gov NCT03447249 ↗Enrolled (actual)
382
Serious AEs
18.1%
Results posted
Mar 2020
Primary outcomePrimary: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) — -1.0; 13.0 percentage points — p=<0.0001
◆ Published Evidence
No publication linked
No peer-reviewed publication reporting this trial's results has been linked yet. This can indicate results are unpublished — a known publication-bias signal. We re-check periodically.
Summary
This study will evaluate the efficacy of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF subjects).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) |
-0.8; 13.4 | <0.0001 sig |
| SECONDARY Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) |
-0.8; 13.4 | <0.0001 sig |
| SECONDARY Number of Pulmonary Exacerbations (PEx) |
116; 17 | <0.0001 sig |
| SECONDARY Absolute Change in Sweat Chloride (SwCl) |
-0.1; -44.6 | <0.0001 sig |
| SECONDARY Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score |
0.1; 18.0 | <0.0001 sig |
| SECONDARY Absolute Change in Body Mass Index (BMI) |
-0.05; 1.06 | <0.0001 sig |
| SECONDARY Absolute Change in Sweat Chloride |
0.0; -43.3 | <0.0001 sig |
| SECONDARY Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score |
0.1; 18.0 | <0.0001 sig |
| SECONDARY Time-to-first Pulmonary Exacerbation (PEx) |
NA; NA | — |
| SECONDARY Absolute Change in BMI Z-score for Participants <=20 Years of Age at Baseline |
-0.08; 0.31 | — |
| SECONDARY Absolute Change in Body Weight |
0.1; 3.3 | — |
| SECONDARY Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) |
175; 173; 58; 11 | — |
| SECONDARY Observed Pre-dose Concentration (Ctrough) of VX-659, TEZ, M1-TEZ, and IVA |
662; 764; 614; 638; 1220; 1390 | — |
Eligibility Criteria
Key Inclusion Criteria
- Heterozygous for F508del and an MF mutation (as defined in the protocol)
- Forced expiratory volume in 1 second (FEV1) value ≥40% and ≤90% of predicted mean for age, sex, and height
Key Exclusion Criteria
- Clinically significant cirrhosis with or without portal hypertension
- Lung infection with organisms associated with a more rapid decline in pulmonary status
- Solid organ or hematological transplantation
Other protocol defined Inclusion/Exclusion criteria may apply
Data sourced from ClinicalTrials.gov (NCT03447249). 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.