Phase 3
N=573
A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation
Cystic Fibrosis
Bottom Line
View on ClinicalTrials.gov: NCT05076149 ↗Enrolled (actual)
573
Serious AEs
14.0%
Results posted
Jun 2024
Primary outcome: Primary: Absolute Change in Percent Predicted Forced Expiratory Volume in 1second (ppFEV1) — 0.0; 0.2 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 1second (ppFEV1) |
0.0; 0.2 | < 0.0001 sig |
| SECONDARY Absolute Change in Sweat Chloride (SwCl) |
-2.3; -5.1 | =0.0034 sig |
| SECONDARY Percentage of Participants With SwCl <60 Millimole Per Liter (mmol/L) (Pooled With Data From Study VX20-121-102) |
76.6; 85.8 | < 0.0001 sig |
| SECONDARY Percentage of Participants With SwCl <30 mmol/L (Pooled With Data From Study VX20-121-102) |
22.5; 30.5 | < 0.0001 sig |
Summary
The purpose of this study is to evaluate the efficacy and safety of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants who are homozygous for F508del, heterozygous for F508del and a gating (F/G) or residual function (F/RF) mutation, or have at least 1 other TCR CF transmembrane conductance regulator (CFTR) gene mutation and no F508del mutation.
Eligibility Criteria
Key Inclusion Criteria
- Participant has one of the following genotypes:
- Homozygous for F508del;
- Heterozygous for F508del and a gating (F/G) mutation;
- Heterozygous for F508del and a residual function (F/RF) mutation;
- At least 1 other TCR CFTR gene mutation identified as responsive to ELX/TEZ/IVA and no F508del mutation
- Forced expiratory volume in 1 second (FEV1) value >=40% and =40% and <=80% for participants not currently receiving CFTR protein modulator therapy
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 (NCT05076149). 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.