Phase 2
Completed N=77
A Phase 2 Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis
Source: ClinicalTrials.gov NCT03911713 ↗Enrolled (actual)
77
Serious AEs
10.7%
Results posted
Jan 2022
Primary outcomePrimary: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) — -0.8; 3.1; 2.7 percentage points
Summary
The purpose of this study is to evaluate the efficacy, safety, pharmacodynamic (PD) and pharmacokinetic (PK) effect of VX-561.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) |
-0.8; 3.1; 2.7 | — |
| SECONDARY Absolute Change in Sweat Chloride (SwCl) |
0.9; 3.3; -6.5 | — |
| SECONDARY Observed Pre-Dose Concentration (Ctrough) of VX-561 and Its Metabolites (M1-VX-561 and M6-VX-561) and IVA and Its Metabolites (M1-IVA and M6-IVA) |
26.1; 123; 458; 1100; 18.1; 108 | — |
| SECONDARY Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) |
8; 4; 8; 21; 23; 1 | — |
Eligibility Criteria
Key Inclusion Criteria
- Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D
- On ivacaftor therapy
- FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height
Key Exclusion Criteria
- History of clinically significant cirrhosis with or without portal hypertension
- History of solid organ or hematological transplantation
- Lung infection with organisms associated with a more rapid decline in pulmonary status
Other protocol defined Inclusion/Exclusion criteria may apply
Data sourced from ClinicalTrials.gov (NCT03911713). 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.