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A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF)

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: NCT05033080
Recruitment Status : Active, not recruiting
First Posted : September 2, 2021
Last Update Posted : December 23, 2022
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 efficacy and safety of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants who are heterozygous for F508del and a minimal function mutation (F/MF participants).

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: VX-121/TEZ/D-IVA Drug: ELX/TEZ/IVA Drug: IVA Drug: Placebo (matched to VX-121/TEZ/D-IVA) Drug: Placebo (matched to ELX/TEZ/IVA) Drug: Placebo (matched to IVA) Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 436 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-121 Combination Therapy in Subjects With Cystic Fibrosis (CF) Who Are Heterozygous for F508del and a Minimal Function Mutation (F/MF)
Actual Study Start Date : September 14, 2021
Estimated Primary Completion Date : June 2023
Estimated Study Completion Date : January 2024

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: 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 tablets for oral administration.
Other Names:
  • VX-121/VX-661/CTP-656
  • VX-121/VX-661/VX-561
  • VX-121/tezacaftor/deutivacaftor

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.
Active Comparator: ELX/TEZ/IVA
Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening.
 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.


Outcome Measures
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Primary Outcome Measures :
  1. Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 24 [ Time Frame: From Baseline Through Week 24 ]

Secondary Outcome Measures :
  1. Absolute Change From Baseline in Sweat Chloride (SwCl) Through Week 24 [ Time Frame: From Baseline Through Week 24 ]
  2. Proportion of Participants With SwCl <60 Millimole per Liter (mmol/L) Through Week 24 (Pooled With Data From Study VX20-121-103) [ Time Frame: From Baseline Through Week 24 ]
  3. Proportion of Participants With SwCl <30 mmol/L Through Week 24 (Pooled With Data From Study VX20-121-103) [ Time Frame: From Baseline Through Week 24 ]

Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Heterozygous for F508del and a minimal function mutation (F/MF genotype)
  • Forced expiratory volume in 1 second (FEV1) value >=40% and <=90% of predicted mean for age, sex, and height for participants currently receiving ELX/TEZ/IVA therapy; FEV1 >=40% and <=80% for participants not currently receiving ELX/TEZ/IVA

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

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): NCT05033080


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: NCT05033080    
Other Study ID Numbers: VX20-121-102
2021-000712-31 ( EudraCT Number )
First Posted: September 2, 2021    Key Record Dates
Last Update Posted: December 23, 2022
Last Verified: April 2022
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
Ivacaftor
Elexacaftor
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action