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

Recruitment Status
ENROLLING BY INVITATION
(See Contacts and Locations)Verified March 2026 by Vertex Pharmaceuticals Incorporated
Sponsor
Vertex Pharmaceuticals Incorporated
Information Provided by (Responsible Party)
Vertex Pharmaceuticals Incorporated
Clinicaltrials.gov Identifier
NCT05844449
Other Study ID Numbers:
VX22-121-106
First Submitted
April 23, 2023
First Posted
May 5, 2023
Last Update Posted
April 28, 2026
Last Verified
March 2026

ClinicalTrials.gov processed this data on April 2026. Link to the current ClinicalTrials.gov record .

History of Changes

Study Details

Study Description

Condition or DiseaseIntervention/Treatment
Cystic Fibrosis
Drug: VNZ/TEZ/D-IVA

Study Design

Study TypeInterventional
Actual Enrollment174 participants
Design AllocationN/A
Interventional ModelSingle Group Assignment
MaskingNone (Open Label)
Primary PurposeTreatment
Official TitleA 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
Study Start DateAugust 10, 2023
Actual Primary Completion Date3yrs 2mos from now
Actual Study Completion Date3yrs 2mos from now

Groups and Cohorts

Group/CohortIntervention/Treatment
VNZ/TEZ/D-IVA
Part A: Participants (Cohort 1: 6 through 11 years of age (inclusive); Cohort 2: 2 through 5 years of age (inclusive) and Cohort 3: 1 to less than (\<) 2 years of age) will receive VNZ/TEZ/D-IVA once daily at doses determined based on their age and weight. The age range is based on date of informed consent in parent study. Part B: Participants in Cohort 1 who meet the eligibility criteria will receive VNZ/TEZ/D-IVA for an additional 96 weeks.
Drug: VNZ/TEZ/D-IVA
Fixed-dose combination tablets or granules for oral administration.

Outcome Measures

Primary Outcome Measures
  1. Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
  2. Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
Secondary Outcome Measures
  1. Part A (All Cohorts): Absolute Change in Sweat Chloride (SwCl)
  2. Part B: Absolute Change in Sweat Chloride (SwCl)
  3. Part A (Cohort 1): Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1)
  4. Part B: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1)
  5. Part A (All Cohorts): Number of Pulmonary Exacerbation (PEx)
  6. Part B: Number of Pulmonary Exacerbation (PEx)
  7. Part A (All Cohorts): Number of CF- Related Hospitalizations
  8. Part B: Number of CF- Related Hospitalizations
  9. Part A (Cohort 1): Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score
  10. Part B: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score
  11. Part A (Cohorts 1 and 2): Absolute Change in Body Mass Index (BMI)
  12. Part B: Absolute Change in Body Mass Index (BMI)
  13. Part A (Cohorts 1 and 2): Absolute Change in BMI-for-age Z-score
  14. Part B: Absolute Change in BMI-for-age Z-score
  15. Part A (Cohort 3): Absolute Change in Weight-for-length
  16. Part B: Absolute Change in Weight-for-length
  17. Part A (Cohort 3): Absolute Change in Weight-for-length Z-score
  18. Part B: Absolute Change in Weight-for-length Z-score
  19. Part A (All Cohorts): Absolute Change in Weight
  20. Part B: Absolute Change in Weight
  21. Part A (All Cohorts): Change in Weight-for-age Z-score
  22. Part B: Change in Weight-for-age Z-score
  23. Part A (Cohorts 1 and 2): Absolute Change in Height
  24. Part B: Absolute Change in Height
  25. Part A (Cohorts 1 and 2): Absolute Change in Height-for-age Z-score
  26. Part B: Absolute Change in Height-for-age Z-score
  27. Part A (Cohort 3): Absolute Change in Length
  28. Part B: Absolute Change in Length
  29. Part A (Cohort 3): Absolute Change in Length-for-age Z-score
  30. Part B: Absolute Change in Length-for-age Z-score

Eligibility Criteria

Ages Eligible for Study(Child, Adult, Older Adult)
Sexes Eligible for StudyAll
Accepts Healthy VolunteersNo
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.

Contacts and Locations

Sponsors and CollaboratorsVertex Pharmaceuticals Incorporated
Locations
Children's Hospital of Orange County | Orange California, United States, 92868Stanford University - Palo Alto - Pulmonology | Palo Alto California, United States, 94304Children's Hospital of Colorado - Pulmonology | Aurora Colorado, United States, 80045The Emory Clinic - Clifton Road - Pulmonology | Atlanta Georgia, United States, 30322Ann & Robert H. Lurie Children's Hospital of Chicago - Hematology | Chicago Illinois, United States, 60611JW Riley Hospital for Children - Pulmonology | Indianapolis Indiana, United States, 46202Boston Children's Hospital | Boston Massachusetts, United States, 02115Children's Health Care d/b/a Children's Hospitals and Clinics of Minnesota | Minneapolis Minnesota, United States, 55404The Children's Mercy Hospital | Kansas City Missouri, United States, 64108St. Louis Children's Hospital - Pulmonology | St Louis Missouri, United States, 63110Cohen Children's Medical Center - Lakeville Road | Lake Success New York, United States, 11042Cincinnati Children's Hospital Medical Center - Pulmonology | Cincinnati Ohio, United States, 45229UH Cleveland Medical Center - Pulmonology | Cleveland Ohio, United States, 44106Nationwide Children's Hospital | Columbus Ohio, United States, 43205Children's Hospital of Pittsburgh - Pulmonology | Pittsburgh Pennsylvania, United States, 15224Texas Children's Hospital - Wallace Tower - Pulmonology | Houston Texas, United States, 77030Vermont Lung Center | Colchester Vermont, United States, 05446American Family Childrens Hospital | Madison Wisconsin, United States, 53792The Kids Research Institute Australia | Nedlands , Australia, Women's & Children's Hospital | North Adelaide , Australia, The Royal Children's Hospital Melbourne | Parkville , Australia, Children's Health Queensland Hospital and Health Service | South Brisbane , Australia, Hospital for Sick Children - Pulmonology | Toronto , Canada, British Columbia Children's Hospital | Vancouver , Canada, Hopital Femme Mere-Enfant | Bron , France, Hopital Necker Enfants Malades - Pulmonology | Paris , France, Charite Paediatric Pulmonology Department | Berlin , Germany, Universitatsklinikum Essen | Essen , Germany, Medizinische Hochschule Hannover - Clinic for Pediatric Pneumology, Allergology and Neonatology, CF-Centre | Hanover , Germany, Sophia Children's Hospital | Rotterdam , Netherlands, Starship Child Health | Grafton , New Zealand, Sahlgrenska Universitetssjukhuset - Göteborg CF-center | Gothenburg , Sweden, Karolinska University Hospital - Pulmonology | Stockholm , Sweden, Inselspital Bern | Bern , Switzerland, Kinderspital Zurich - Abteilung Pneumologie | Zurich , Switzerland, Noah's Ark Children's Hospital for Wales | Cardiff , United Kingdom, Great Ormond Street Hospital for Children | London , United Kingdom,