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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05422222
Recruitment Status : Active, not recruiting
First Posted : June 16, 2022
Last Update Posted : January 25, 2024
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Study Description
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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
Cystic Fibrosis Drug: VX-121/TEZ/D-IVA Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 210 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : June 21, 2022
Estimated Primary Completion Date : June 2030
Estimated Study Completion Date : June 2030

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arms and Interventions
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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:
  • VX-121/VX-661/VX-561
  • VX-121/VX-661/CTP-656
  • VX-121/tezacaftor/deutivacaftor
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:
  • VX-121/VX-661/VX-561
  • VX-121/VX-661/CTP-656
  • VX-121/tezacaftor/deutivacaftor


Outcome Measures
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Primary Outcome Measures :
  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 Outcome Measures :
  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 ]

Eligibility Criteria
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Ages Eligible for Study:   1 Year to 11 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05422222


Locations
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Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
More Information
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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT05422222    
Other Study ID Numbers: VX21-121-105
2021-005930-40 ( EudraCT Number )
First Posted: June 16, 2022    Key Record Dates
Last Update Posted: January 25, 2024
Last Verified: June 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
 Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases