Phase 2
Completed N=45
Study of VX-121 in Healthy Subjects and in Subjects With Cystic Fibrosis
Source: ClinicalTrials.gov NCT03768089 ↗Enrolled (actual)
45
Serious AEs
0.9%
Results posted
Jul 2022
Primary outcomePrimary: Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) — 1; 4; 2; 4 participants
Summary
The purpose of this study is to evaluate safety and tolerability of VX-121 in healthy subjects and 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 (TEAEs) and Serious Adverse Events (SAEs) |
1; 4; 2; 4; 1; 2 | — |
| SECONDARY Part A: Maximum Observed Concentration (Cmax) of VX-121 |
0.134; 0.247; 0.0705; 0.061; 0.893; 1.04 | — |
| SECONDARY Part A: Area Under the Concentration Versus Time Curve From the Time of Dosing to the Last Measurable Concentration (AUC[0-last]) of VX-121 |
7.72; 20.7; 6.32; 5.36; 72.2; 66.6 | — |
| SECONDARY Part B: Maximum Observed Concentration (Cmax) of VX-121 |
0.153; 0.418; 0.803; 1.43; 0.497; 1.41 | — |
| SECONDARY Part B: Area Under the Concentration Versus Time Curve During the Dosing Interval (AUCtau) of VX-121 |
2.62; 7.11; 14.4; 27.5; 10.3; 30.1 | — |
| SECONDARY Part B: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121 |
0.391; 1.16; 2.07; 2.94; 0.484; 1.64 | — |
| SECONDARY Part C: Maximum Observed Concentration (Cmax) of VX-121, TEZ and Its Metabolites (M1-TEZ and M2-TEZ) and, IVA and Its Metabolites (M1-IVA and M6-IVA) |
0.212; 0.408; 0.0728; 1.18; 1.77; 0.364 | — |
| SECONDARY Part C: Area Under the Concentration Versus Time Curve During a Dosing Interval (AUCtau) of VX-121, TEZ and Its Metabolites (M1-TEZ and M2-TEZ) and IVA and Its Metabolites (M1-IVA and M6-IVA) |
3.51; 7.96; 1.18; 21.7; 35.4; 6.52 | — |
| SECONDARY Part C: Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ and Its Metabolites (M1-TEZ and M2-TEZ) and IVA and Its Metabolites (M1-IVA and M6-IVA) |
0.916; 1.37; 0.258; 1.24; 1.72; 0.357 | — |
| SECONDARY Part D: Maximum Observed Concentration (Cmax) of VX-121, TEZ and Its Metabolites (M1-TEZ and M2-TEZ) and IVA and Its Metabolites (M1-IVA and M6-IVA) |
0.0553; 0.389; 5.38; 6.80; 1.26; 5.56 | — |
| SECONDARY Part D: Area Under the Concentration Versus Time Curve From the Time of Dosing to the Last Measurable Concentration (AUC[0-last]) of VX-121, TEZ and Its Metabolites (M1-TEZ and M2-TEZ) and IVA and Its Metabolites (M1-IVA and M6-IVA) |
0.160; 2.50; 21.9; 37.4; 4.66; 37.8 | — |
| SECONDARY Part D: Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ and Its Metabolites (M1-TEZ and M2-TEZ) and IVA and Its Metabolites (M1-IVA and M6-IVA) |
0.258; 0.348; 0.323; 1.78; 2.01; 2.02 | — |
| SECONDARY Part D: Absolute Change in Sweat Chloride (SwCl) Concentrations |
2.6; -47.7 | — |
| SECONDARY Part D: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) |
-2.0; 8.3 | — |
Eligibility Criteria
Key Inclusion Criteria
- Part A, B, and C: Healthy Volunteers
- Female subjects must be of non-childbearing potential
- Between the ages of 18 and 55 years, inclusive
- Body mass index (BMI) of 18.0 to 32.0 kg/m2, inclusive, and a total body weight >50 kg
- Part D: Subjects with CF
- Heterozygous for F508del and an MF mutation (F/MF)
- FEV1 value ≥40% and ≤90% of predicted mean for age, sex, and height
- Body weight ≥35 kg
Key Exclusion Criteria
- Part A, B and C: Healthy Volunteers
- Any condition possibly affecting drug absorption
- History of febrile illness or other acute illness within 5 days before the first study drug dose
- Part D: Subjects with CF
- 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 (NCT03768089). 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.