Phase 3
N=398
A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF)
Cystic Fibrosis
Bottom Line
View on ClinicalTrials.gov: NCT05033080 ↗Enrolled (actual)
398
Serious AEs
17.3%
Results posted
Jun 2024
Primary outcome: Primary: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) — 0.3; 0.5 percentage points — p=< 0.0001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- VX-121/TEZ/D-IVA (Drug); ELX/TEZ/IVA (Drug); IVA (Drug); Placebo (matched to VX-121/TEZ/D-IVA) (Drug); Placebo (matched to ELX/TEZ/IVA) (Drug); Placebo (matched to IVA) (Drug)
- Age
- Pediatric, Adult, Older Adult · 12+ yrs
- Sex
- All
- Sponsor
- Vertex Pharmaceuticals Incorporated
- Primary completion
- May 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) |
0.3; 0.5 | < 0.0001 sig |
| SECONDARY Absolute Change in Sweat Chloride (SwCl) |
0.9; -7.5 | < 0.0001 sig |
| SECONDARY Percentage of Participants With SwCl <60 mmol/L (Pooled With Data From Study VX20-121-103) |
76.6; 85.8 | < 0.0001 sig |
| SECONDARY Percentage of Participants With SwCl <30 mmol/L (Pooled With Data From Study VX20-121-103) |
22.5; 30.5 | < 0.0001 sig |
Summary
The purpose of this study evaluates the efficacy and safety of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants who were heterozygous for F508del and a minimal function mutation (F/MF participants).
Eligibility Criteria
Key Inclusion Criteria
- Heterozygous for F508del and a minimal function mutation (F/MF genotype)
- Forced expiratory volume in 1 second (FEV1) value >=40% and =40% and <=80% for participants not currently receiving ELX/TEZ/IVA
Key Exclusion Criteria
- History of solid organ or hematological transplantation
- Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)
- Lung infection with organisms associated with a more rapid decline in pulmonary status
- Pregnant or breast-feeding females
Other protocol defined Inclusion/Exclusion criteria may apply
Data sourced from ClinicalTrials.gov (NCT05033080). 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.