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A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation

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ClinicalTrials.gov Identifier: NCT05076149
Recruitment Status : Active, not recruiting
First Posted : October 13, 2021
Last Update Posted : December 23, 2022
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Tracking Information
First Submitted Date  ICMJE September 29, 2021
First Posted Date  ICMJE October 13, 2021
Last Update Posted Date December 23, 2022
Actual Study Start Date  ICMJE October 27, 2021
Estimated Primary Completion Date July 2023   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 29, 2021)		 Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 24 [ Time Frame: From Baseline Through Week 24 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: September 29, 2021)
  • Absolute Change From Baseline in Sweat Chloride (SwCl) Through Week 24 [ Time Frame: From Baseline Through Week 24 ]
  • Proportion of Participants With SwCl <60 Millimole per Liter (mmol/L) Through Week 24 (Pooled With Data From Study VX20-121-102) [ Time Frame: From Baseline Through Week 24 ]
  • Proportion of Participants With SwCl <30 mmol/L Through Week 24 (Pooled With Data From Study VX20-121-102) [ 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 A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation
Official Title  ICMJE A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-121 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive CFTR Mutation and No F508del Mutation
Brief Summary The purpose of this study is to evaluate the efficacy and safety of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants who are homozygous for F508del, heterozygous for F508del and a gating (F/G) or residual function (F/RF) mutation, or have at least 1 other TCR CF transmembrane conductance regulator (CFTR) gene mutation and no F508del mutation.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Cystic Fibrosis
Intervention  ICMJE
  • Drug: VX-121/TEZ/D-IVA
    Fixed-dose combination tablets for oral administration.
    Other Names:
    • VX-121/VX-661/CTP-656
    • VX-121/VX-661/VX-561
    • VX-121/tezacaftor/deutivacaftor
  • Drug: ELX/TEZ/IVA
    Fixed-dose combination tablets for oral administration.
    Other Names:
    • VX-445/VX-661/VX-770
    • elexacaftor/tezacaftor/ivacaftor
  • Drug: IVA
    Tablet for oral administration.
    Other Names:
    • VX-770
    • ivacaftor
  • Drug: Placebo (matched to VX-121/TEZ/D-IVA)
    Placebo matched to VX-121/TEZ/D-IVA for oral administration.
  • Drug: Placebo (matched to ELX/TEZ/IVA)
    Placebo matched to ELX/TEZ/IVA for oral administration.
  • Drug: Placebo (matched to IVA)
    Placebo matched to IVA for oral administration.
Study Arms  ICMJE
  • Experimental: VX-121/TEZ/D-IVA
    Participants will receive VX-121/TEZ/D-IVA in the morning.
    Interventions:
    • Drug: VX-121/TEZ/D-IVA
    • Drug: Placebo (matched to ELX/TEZ/IVA)
    • Drug: Placebo (matched to IVA)
  • Active Comparator: ELX/TEZ/IVA
    Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening.
    Interventions:
    • Drug: ELX/TEZ/IVA
    • Drug: IVA
    • Drug: Placebo (matched to 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
Actual Enrollment  ICMJE
 (submitted: December 22, 2022)		 600
Original Estimated Enrollment  ICMJE
 (submitted: September 29, 2021)		 550
Estimated Study Completion Date  ICMJE February 2024
Estimated Primary Completion Date July 2023   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key Inclusion Criteria:

  • Participant has one of the following genotypes:

    • Homozygous for F508del;
    • Heterozygous for F508del and a gating (F/G) mutation;
    • Heterozygous for F508del and a residual function (F/RF) mutation;
    • At least 1 other TCR CFTR gene mutation identified as responsive to ELX/TEZ/IVA and no F508del mutation
  • Forced expiratory volume in 1 second (FEV1) value >=40% and <=90% of predicted mean for age, sex, and height for participants currently receiving CFTR protein modulator therapy; FEV1 >=40% and <=80% for participants not currently receiving CFTR protein modulator therapy

Key Exclusion Criteria:

  • History of solid organ or hematological transplantation
  • 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
  • Pregnant or breast-feeding females

Other protocol defined Inclusion/Exclusion criteria may apply
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   Austria,   Belgium,   Canada,   Denmark,   France,   Germany,   Greece,   Hungary,   Ireland,   Israel,   Italy,   Netherlands,   New Zealand,   Norway,   Poland,   Sweden,   Switzerland,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT05076149
Other Study ID Numbers  ICMJE VX20-121-103
2021-000694-85 ( 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 August 2022

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