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Enrolling by Invitation

NCT Number: NCT05844449

Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older

The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of vanzacaftor/tezacaftor/deutivacaftor (VNZ/TEZ/D-IVA) in participants with cystic fibrosis (CF).

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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 and older

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:

Parts A and B:

  • Participants who have completed study drug treatment in the parent study (VX21-121-105; NCT Number: NCT05422222)

Part B:

-Meets at least 1 of the following criteria:

  • Completed study drug treatment in Part A
  • Had study drug interruption(s) in Part A, but did not permanently discontinue study drug and completed study visits up to the last scheduled visit of the treatment period of Part A

Key Exclusion Criteria:

  • Hepatic cirrhosis with portal hypertension, moderate hepatic impairment, or severe hepatic impairment that might pose an additional risk in administering study drug
  • History of solid organ, hematological transplantation, or cancer
  • History of drug intolerance in the parent study

Other protocol defined Inclusion/Exclusion criteria may apply.

Treatment and study plan

VNZ/TEZ/D-IVA

Drug

Fixed-dose combination tablets or granules for oral administration.

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

Primary outcomes

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

    Time frame: From Baseline up to Week 100

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

    Time frame: From Baseline up to Week 196

Secondary outcomes

  1. Part A (All Cohorts): Absolute Change in Sweat Chloride (SwCl)

    Time frame: From Baseline Through Week 96

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

    Time frame: From Baseline Through Week 192

  3. Part A (Cohort 1): Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1)

    Time frame: From Baseline Through Week 100

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

    Time frame: From Baseline Through Week 196

  5. Part A (All Cohorts): Number of Pulmonary Exacerbation (PEx)

    Time frame: From Baseline Through Week 100

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

    Time frame: From Baseline Through Week 196

  7. Part A (All Cohorts): Number of CF- Related Hospitalizations

    Time frame: From Baseline Through Week 100

  8. Part B: Number of CF- Related Hospitalizations

    Time frame: From Baseline Through Week 196

  9. Part A (Cohort 1): Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score

    Time frame: From Baseline Through Week 100

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

    Time frame: From Baseline Through Week 196

  11. Part A (Cohorts 1 and 2): Absolute Change in Body Mass Index (BMI)

    Time frame: From Baseline Through Week 100

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

    Time frame: From Baseline Through Week 196

  13. Part A (Cohorts 1 and 2): Absolute Change in BMI-for-age Z-score

    Time frame: From Baseline Through Week 100

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

    Time frame: From Baseline Through Week 196

  15. Part A (Cohort 3): Absolute Change in Weight-for-length

    Time frame: From Baseline Through Week 100

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

    Time frame: From Baseline Through Week 196

  17. Part A (Cohort 3): Absolute Change in Weight-for-length Z-score

    Time frame: From Baseline Through Week 100

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

    Time frame: From Baseline Through Week 196

  19. Part A (All Cohorts): Absolute Change in Weight

    Time frame: From Baseline Through Week 100

  20. Part B: Absolute Change in Weight

    Time frame: From Baseline Through Week 196

  21. Part A (All Cohorts): Change in Weight-for-age Z-score

    Time frame: From Baseline Through Week 100

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

    Time frame: From Baseline Through Week 196

  23. Part A (Cohorts 1 and 2): Absolute Change in Height

    Time frame: From Baseline Through Week 100

  24. Part B: Absolute Change in Height

    Time frame: From Baseline Through Week 196

  25. Part A (Cohorts 1 and 2): Absolute Change in Height-for-age Z-score

    Time frame: From Baseline Through Week 100

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

    Time frame: From Baseline Through Week 196

  27. Part A (Cohort 3): Absolute Change in Length

    Time frame: From Baseline Through Week 100

  28. Part B: Absolute Change in Length

    Time frame: From Baseline Through Week 196

  29. Part A (Cohort 3): Absolute Change in Length-for-age Z-score

    Time frame: From Baseline Through Week 100

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

    Time frame: From Baseline Through Week 196

Sponsors and collaborators

Lead sponsor

Vertex Pharmaceuticals Incorporated

Industry

Registry information

Official study title

A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 1 Year of Age and Older

Important dates

Study start
2023
Primary completion
2029
Study completion
2029
First posted
May 6, 2023
Registry last updated
Apr 29, 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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