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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

Tracking Information
First Submitted Date  ICMJE June 13, 2022
First Posted Date  ICMJE June 16, 2022
Last Update Posted Date January 25, 2024
Actual Study Start Date  ICMJE June 21, 2022
Estimated Primary Completion Date June 2030   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 13, 2022)
  • 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 ]
  • 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 ]
  • 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 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: June 13, 2022)
  • Part B: Absolute Change in Sweat Chloride (SwCl) [ Time Frame: From Baseline Through Week 24 ]
  • 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 ]
  • Part B: Drug Acceptability Assessment Using Modified Facial Hedonic Scale [ Time Frame: At Day 1 and Week 24 ]
  • Part B: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1) [ Time Frame: From Baseline Through Week 24 ]
  • Part B: Number of Pulmonary Exacerbation (PEx) [ Time Frame: From Baseline Through Week 24 ]
  • Part B: Number of CF-Related Hospitalizations [ Time Frame: From Baseline Through Week 24 ]
  • Part B: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score [ Time Frame: From Baseline Through Week 24 ]
  • Part B: Absolute Change in Body Mass Index (BMI) [ Time Frame: From Baseline at Week 24 ]
  • Part B: Absolute Change in BMI-for-age Z-score [ Time Frame: From Baseline at Week 24 ]
  • Part B: Absolute Change in Weight [ Time Frame: From Baseline at Week 24 ]
  • Part B: Absolute Change in Weight-for-age Z-score [ Time Frame: From Baseline at Week 24 ]
  • Part B: Absolute Change in Weight-for-length [ Time Frame: From Baseline at Week 24 ]
  • Part B: Absolute Change in Weight-for-length Z-score [ Time Frame: From Baseline at Week 24 ]
  • Part B: Absolute Change in Height [ Time Frame: From Baseline at Week 24 ]
  • Part B: Absolute Change in Height-for-age Z-score [ Time Frame: From Baseline at Week 24 ]
  • Part B: Absolute Change in Length [ Time Frame: From Baseline at Week 24 ]
  • Part B: Absolute Change in Length-for-age Z-score [ Time Frame: From Baseline at Week 24 ]
  • Part B: Proportion of Participants With SwCl <60 millimole per liter (mmol/L) [ Time Frame: From Baseline Through Week 24 ]
  • Part B: Proportion of Participants With SwCl <30 mmol/L [ Time Frame: From Baseline Through Week 24 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age
Official Title  ICMJE 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
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Cystic Fibrosis
Intervention  ICMJE 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
Study Arms  ICMJE
  • Experimental: Part A: VX-121/TEZ/D-IVA
    Participants will receive VX-121/TEZ/D-IVA in the morning.
    Intervention: Drug: VX-121/TEZ/D-IVA
  • 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.
    Intervention: Drug: VX-121/TEZ/D-IVA
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Estimated Enrollment  ICMJE
 (submitted: June 13, 2022)		 210
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE June 2030
Estimated Primary Completion Date June 2030   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 1 Year to 11 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   France,   Germany,   Netherlands,   Sweden,   Switzerland,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT05422222
Other Study ID Numbers  ICMJE VX21-121-105
2021-005930-40 ( EudraCT Number )
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
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
Current Responsible Party Vertex Pharmaceuticals Incorporated
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Vertex Pharmaceuticals Incorporated
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Vertex Pharmaceuticals Incorporated
Verification Date June 2023

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP