Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age
Study Details
Study Description
Brief Summary
The purpose of this study is to evaluate the pharmacokinetics, safety, tolerability and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants with at least 1 triple combination responsive (TCR) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 3 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Part A: VX-121/TEZ/D-IVA Participants will receive VX-121/TEZ/D-IVA in the morning. |
Drug: VX-121/TEZ/D-IVA
Fixed-dose combination for oral administration.
Other Names:
|
| Experimental: Part B: VX-121/TEZ/D-IVA Participants will receive VX-121/TEZ/D-IVA in the morning with the dose(s) to be based on the outcome of Part A. |
Drug: VX-121/TEZ/D-IVA
Fixed-dose combination for oral administration.
Other Names:
|
Outcome Measures
Primary Outcome Measures
- Part A: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites [From Day 1 up to Day 22]
- Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [From Day 1 up to Day 50]
- Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [From Day 1 up to Week 28]
Secondary Outcome Measures
- Part B: Absolute Change in Sweat Chloride (SwCl) [From Baseline Through Week 24]
- Part B: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites [From Day 1 up to Week 16]
- Part B: Drug Acceptability Assessment Using Modified Facial Hedonic Scale [At Day 1 and Week 24]
- Part B: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1) [From Baseline Through Week 24]
- Part B: Number of Pulmonary Exacerbation (PEx) [From Baseline Through Week 24]
- Part B: Number of CF-Related Hospitalizations [From Baseline Through Week 24]
- Part B: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score [From Baseline Through Week 24]
- Part B: Absolute Change in Body Mass Index (BMI) [From Baseline at Week 24]
- Part B: Absolute Change in BMI-for-age Z-score [From Baseline at Week 24]
- Part B: Absolute Change in Weight [From Baseline at Week 24]
- Part B: Absolute Change in Weight-for-age Z-score [From Baseline at Week 24]
- Part B: Absolute Change in Weight-for-length [From Baseline at Week 24]
- Part B: Absolute Change in Weight-for-length Z-score [From Baseline at Week 24]
- Part B: Absolute Change in Height [From Baseline at Week 24]
- Part B: Absolute Change in Height-for-age Z-score [From Baseline at Week 24]
- Part B: Absolute Change in Length [From Baseline at Week 24]
- Part B: Absolute Change in Length-for-age Z-score [From Baseline at Week 24]
- Part B: Proportion of Participants With SwCl <60 millimole per liter (mmol/L) [From Baseline Through Week 24]
- Part B: Proportion of Participants With SwCl <30 mmol/L [From Baseline Through Week 24]
Eligibility Criteria
Criteria
Key Inclusion Criteria:
- Participants with stable CF and at least 1 TCR mutation (including F508del) in the CFTR gene
Key Exclusion Criteria:
-
History of solid organ, hematological transplantation, or cancer
-
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
Other protocol defined Inclusion/Exclusion criteria may apply.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Children's Hospital of Colorado | Aurora | Colorado | United States | 80045 |
| 2 | Boston Children's Hospital | Boston | Massachusetts | United States | 02115 |
| 3 | Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota | Minneapolis | Minnesota | United States | 55404 |
| 4 | The Children's Mercy Hospital | Kansas City | Missouri | United States | 64108 |
| 5 | Washington University School of Medicine / St. Louis Children's Hospital | Saint Louis | Missouri | United States | 63110 |
| 6 | Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center | Cleveland | Ohio | United States | 44106 |
| 7 | Oregon Health & Science University | Portland | Oregon | United States | 97239 |
Sponsors and Collaborators
- Vertex Pharmaceuticals Incorporated
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- VX21-121-105
- 2021-005930-40