Phase 3
Completed N=111
A Study of VX-659 Combination Therapy in CF Subjects Homozygous for F508del (F/F)
Source: ClinicalTrials.gov NCT03460990 ↗Enrolled (actual)
111
Serious AEs
1.8%
Results posted
Oct 2019
Primary outcomePrimary: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) — 0.3; 10.2 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 This can indicate results are unpublished — a known publication-bias signal.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 homozygous for the F508del mutation (F/F).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) |
0.3; 10.2 | < 0.0001 sig |
| SECONDARY Absolute Change in Sweat Chloride (SwCl) |
1.5; -47.2 | <0.0001 sig |
| SECONDARY Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score |
3.0; 16.5 | <0.0001 sig |
| SECONDARY Safety and Tolerability as Assessed Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) |
31; 33; 0; 2 | — |
| SECONDARY Observed Pre-Dose Concentration (Ctrough) of VX-659, TEZ, TEZ Metabolite (M1-TEZ), and IVA |
720; 1780; 1590; 1730; 1280; 5170 | — |
Eligibility Criteria
Key Inclusion Criteria
- Homozygous for the F508del mutation (F/F)
- 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 (NCT03460990). 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.