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Active, not recruiting

NCT Number: NCT05422222

Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age

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.

Active, not recruiting

This study is active but is not currently recruiting participants.

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Key information

Conditions

Cystic Fibrosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

1 year–11 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

The Kids Research Institute Australia, Nedlands, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

VX-121/TEZ/D-IVA

Drug

Fixed-dose combination for oral administration.

Other names: VX-121/VX-661/VX-561, VX-121/VX-661/CTP-656, VX-121/tezacaftor/deutivacaftor

Primary outcomes

  1. Part A: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites

    Time frame: From Day 1 up to Day 22

  2. Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Time frame: From Day 1 up to Day 50

  3. Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Time frame: From Day 1 up to Week 28

Secondary outcomes

  1. Part B: Absolute Change in Sweat Chloride (SwCl)

    Time frame: From Baseline Through Week 24

  2. Part B: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites

    Time frame: From Day 1 up to Week 16

  3. Part B: Drug Acceptability Assessment Using Modified Facial Hedonic Scale

    Time frame: At Day 1 and Week 24

  4. Part B: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1)

    Time frame: From Baseline Through Week 24

  5. Part B: Number of Pulmonary Exacerbation (PEx)

    Time frame: From Baseline Through Week 24

  6. Part B: Number of CF-Related Hospitalizations

    Time frame: From Baseline Through Week 24

  7. Part B: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score

    Time frame: From Baseline Through Week 24

  8. Part B: Absolute Change in Body Mass Index (BMI)

    Time frame: From Baseline at Week 24

  9. Part B: Absolute Change in BMI-for-age Z-score

    Time frame: From Baseline at Week 24

  10. Part B: Absolute Change in Weight

    Time frame: From Baseline at Week 24

  11. Part B: Absolute Change in Weight-for-age Z-score

    Time frame: From Baseline at Week 24

  12. Part B: Absolute Change in Weight-for-length

    Time frame: From Baseline at Week 24

  13. Part B: Absolute Change in Weight-for-length Z-score

    Time frame: From Baseline at Week 24

  14. Part B: Absolute Change in Height

    Time frame: From Baseline at Week 24

  15. Part B: Absolute Change in Height-for-age Z-score

    Time frame: From Baseline at Week 24

  16. Part B: Absolute Change in Length

    Time frame: From Baseline at Week 24

  17. Part B: Absolute Change in Length-for-age Z-score

    Time frame: From Baseline at Week 24

  18. Part B: Proportion of Participants With SwCl <60 millimole per liter (mmol/L)

    Time frame: From Baseline Through Week 24

  19. Part B: Proportion of Participants With SwCl <30 mmol/L

    Time frame: From Baseline Through Week 24

Interested in participating?

Active, not recruiting

This study is active but is not currently recruiting participants.

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Sponsors and collaborators

Lead sponsor

Vertex Pharmaceuticals Incorporated

Industry

Registry information

Official study title

A Phase 3 Study Evaluating the Pharmacokinetics, Safety, and Tolerability of VX-121/Tezacaftor/Deutivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 1 Through 11 Years of Age

Important dates

Study start
2022
Primary completion
2030
Study completion
2030
First posted
Jun 16, 2022
Registry last updated
May 28, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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