A Phase 3 Study of VX-445 Combination Therapy in Cystic Fibrosis (CF) Subjects Heterozygous for F508del and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)
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ClinicalTrials.gov Identifier: NCT04058353 |
Recruitment Status :
Completed
First Posted : August 15, 2019
Last Update Posted : July 13, 2020
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Condition or disease | Intervention/treatment | Phase |
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Cystic Fibrosis | Drug: ELX/TEZ/IVA Drug: IVA Drug: TEZ/IVA | Phase 3 |
Expanded Access : An investigational treatment associated with this study has been approved for sale to the public. More info ...
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 271 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-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes) |
Actual Study Start Date : | August 28, 2019 |
Actual Primary Completion Date : | June 12, 2020 |
Actual Study Completion Date : | June 12, 2020 |

Arm | Intervention/treatment |
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Experimental: Triple Combination Arm
Subjects will receive ELX/TEZ/IVA TC in the morning and IVA as mono tablet in the evening.
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Drug: ELX/TEZ/IVA
Fixed-dose combination (FDC) tablet for oral administration
Other Names:
Drug: IVA 150-mg tablet for oral administration
Other Names:
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Active Comparator: Control Arm
Subjects will receive either IVA as mono tablet OR TEZ/IVA as FDC in the morning and IVA as mono tablet in the evening.
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Drug: IVA
150-mg tablet for oral administration
Other Names:
Drug: TEZ/IVA FDC tablet for oral administration
Other Names:
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- Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1) for ELX/TEZ/IVA group [ Time Frame: From Baseline through Week 8 ]
- Absolute change in sweat chloride (SwCl) for ELX/TEZ/IVA group [ Time Frame: From Baseline through Week 8 ]
- Absolute change in ppFEV1 for ELX/TEZ/IVA group compared to the control group [ Time Frame: From Baseline through Week 8 ]
- Absolute change in SwCl for ELX/TEZ/IVA group compared to the control group [ Time Frame: From Baseline through Week 8 ]
- Absolute change from baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score for ELX/TEZ/IVA group [ Time Frame: From Baseline through Week 8 ]
- Absolute change in CFQ-R respiratory domain score for ELX/TEZ/IVA group compared to the control group [ Time Frame: From Baseline through Week 8 ]
- Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: From Baseline up to Week 12 ]

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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 |
Key Inclusion Criteria:
- Subject has a confirmed diagnosis of CF and is heterozygous for F508del and either a gating or residual function mutation (F/G and F/RF genotypes)
- Forced expiratory volume in 1 second (FEV1) value ≥40% and ≤90% of predicted mean for age, sex, and height
Key Exclusion Criteria:
- Clinically significant cirrhosis with or without portal hypertension
- Lung infection with organisms associated with a more rapid decline in pulmonary status
- Solid organ or hematological transplantation
Other protocol defined Inclusion/Exclusion criteria may apply

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): NCT04058353

Responsible Party: | Vertex Pharmaceuticals Incorporated |
ClinicalTrials.gov Identifier: | NCT04058353 |
Other Study ID Numbers: |
VX18-445-104 2018-002835-76 ( EudraCT Number ) |
First Posted: | August 15, 2019 Key Record Dates |
Last Update Posted: | July 13, 2020 |
Last Verified: | July 2020 |
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 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Cystic Fibrosis Fibrosis Pathologic Processes Pancreatic Diseases Digestive System Diseases Lung Diseases Respiratory Tract Diseases |
Genetic Diseases, Inborn Infant, Newborn, Diseases Ivacaftor Chloride Channel Agonists Membrane Transport Modulators Molecular Mechanisms of Pharmacological Action |