Phase 2
Completed N=86
A Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic Fibrosis
Source: ClinicalTrials.gov NCT03912233 ↗Enrolled (actual)
86
Serious AEs
4.7%
Results posted
Apr 2023
Primary outcomePrimary: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) — 9; 8; 16; 20 participants
Summary
The purpose of this study is to evaluate the safety, tolerability and efficacy of VX-121 combination therapy in subjects with cystic fibrosis (CF).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) |
9; 8; 16; 20; 8; 16 | — |
| PRIMARY Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) |
1.9; 4.6; 14.2; 9.8; -0.1; 15.9 | — |
| SECONDARY Absolute Change in Sweat Chloride (SwCl) Concentrations |
2.3; -42.8; -45.8; -49.5; -2.6; -45.5 | — |
| SECONDARY Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score |
3.3; 17.6; 21.2; 29.8; -5.0; 19.4 | — |
| SECONDARY Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ and Its Metabolite (M1-TEZ) and, VX-561 and Its Metabolites (M1-VX-561 and M6-VX-561) |
317; 366; 520; 582; 974; 1050 | — |
Eligibility Criteria
Key Inclusion Criteria
- Part 1: Heterozygous for F508del and an MF mutation (F/MF)
- Part 2: Homozygous for F508del (F/F)
- FEV1 value ≥40% and ≤90% of the predicted mean for age, sex, and height
Key Exclusion Criteria
- History of clinically significant cirrhosis with or without portal hypertension
- Lung infection with organisms associated with a more rapid decline in pulmonary status
- History of solid organ or hematological transplantation
Other protocol-defined Inclusion/Exclusion criteria may apply
Data sourced from ClinicalTrials.gov (NCT03912233). 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.