Phase 3
Completed N=16
Evaluation of VX 445/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 Years of Age
Source: ClinicalTrials.gov NCT03691779 ↗Enrolled (actual)
16
Serious AEs
1.2%
Results posted
Oct 2021
Primary outcomePrimary: Part A: Maximum Observed Plasma Concentration (Cmax) of ELX, TEZ, and IVA — 6.13; 6.93; 1.01 microgram per milliliter (mcg/mL)
◆ Published Evidence
Highly cited
283citations · ~57 / year
A Phase 3 Open-Label Study of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 through 11 Years of Age with Cystic Fibrosis and at Least One <i>F508del</i> Allele.
Summary
This study will evaluate the pharmacokinetics (PK), safety, tolerability, efficacy, and pharmacodynamic effect of VX-445, tezacaftor (TEZ), and ivacaftor (IVA) when dosed in triple combination (TC) in Cystic Fibrosis (CF) subjects 6 through 11 years of age with F/F and F/MF genotypes.
Linked Publications (3)
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A Phase 3 Open-Label Study of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 through 11 Years of Age with Cystic Fibrosis and at Least One <i>F508del</i> Allele.
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Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).
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Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Part A: Maximum Observed Plasma Concentration (Cmax) of ELX, TEZ, and IVA |
6.13; 6.93; 1.01 | — |
| PRIMARY Part A: Observed Pre-dose Plasma Concentration (Ctrough) of ELX, TEZ, and IVA |
2.86; 1.06; 0.297 | — |
| PRIMARY Part A: Area Under the Concentration Versus Time Curve From 0 to 24 Hours (AUC0-24h) of ELX, TEZ, and IVA |
107; 58.4; 8.12 | — |
| PRIMARY Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) |
65; 1 | — |
| SECONDARY Part A: Cmax of ELX Metabolite (M23-ELX), TEZ Metabolite (M1-TEZ), and IVA Metabolite (M1-IVA) |
1.60; 6.26; 2.36 | — |
| SECONDARY Part A: Ctrough of ELX Metabolite (M23-ELX), TEZ Metabolite (M1-TEZ), and IVA Metabolite (M1-IVA) |
1.30; 4.64; 0.890 | — |
| SECONDARY Part A: AUC0-24h of ELX Metabolite (M23-ELX) and TEZ Metabolite (M1-TEZ) |
35.6; 133 | — |
| SECONDARY Part A: Area Under the Concentration Versus Time Curve From 0 to 6 Hours (AUC0-6h) of IVA Metabolite (M1-IVA) |
9.41 | — |
| SECONDARY Part A: Safety and Tolerability as Assessed by Number of Participants With TEAEs and SAEs |
12; 0 | — |
| SECONDARY Part B: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) |
10.2 | — |
| SECONDARY Part B: Absolute Change in Sweat Chloride (SwCl) |
-60.9 | — |
| SECONDARY Part B: Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score |
7.0 | — |
| SECONDARY Part B: Absolute Change in Body Mass Index (BMI) |
1.02 | — |
| SECONDARY Part B: Absolute Change in BMI For-Age Z-Score |
0.37 | — |
| SECONDARY Part B: Absolute Change in Weight |
3.0 | — |
| SECONDARY Part B: Absolute Change in Weight-for-age Z-Score |
0.25 | — |
| SECONDARY Part B: Absolute Change in Height |
2.3 | — |
| SECONDARY Part B: Absolute Change in Height-for-Age Z-Score |
-0.05 | — |
| SECONDARY Part B: Drug Acceptability Assessment Using Modified Facial Hedonic Scale |
16; 6; 10; 1; 0 | — |
| SECONDARY Part B: Number of Pulmonary Exacerbations Events |
4 | — |
| SECONDARY Part B: Number of CF Related Hospitalizations |
— | — |
| SECONDARY Part B: Ctrough of ELX, ELX Metabolite (M23-ELX), TEZ, TEZ Metabolite (M1-TEZ), IVA and IVA Metabolite (M1-IVA) |
2.71; 5.69; 1.59; 4.41; 1.43; 2.37 | — |
| SECONDARY Part B: Absolute Change in Lung Clearance Index 2.5 (LCI2.5) |
-1.71 | — |
Eligibility Criteria
Key Inclusion Criteria
- Homozygous or heterozygous for F508del mutation (F/F or F/MF genotypes)
- Forced expiratory volume in 1 second (FEV1) value ≥40% of predicted mean for age, sex, and height.
Key Exclusion Criteria
- Clinically significant cirrhosis with or without portal hypertension
- Lung infection with organisms associated with a more rapid decline in pulmonary status.
- Solid organ or hematological transplantation.
Other protocol defined Inclusion/Exclusion criteria may apply.
Data sourced from ClinicalTrials.gov (NCT03691779) and the linked publication. 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.