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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT04058353 |
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Date of registration:
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14/08/2019 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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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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Scientific title:
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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) |
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Date of first enrolment:
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August 28, 2019 |
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Target sample size:
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271 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT04058353 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
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Phase:
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Phase 3
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Countries of recruitment
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Australia
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Belgium
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Canada
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Denmark
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France
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Germany
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Ireland
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Israel
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Italy
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Netherlands
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Spain
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United Kingdom
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United States
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Key inclusion & exclusion criteria
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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
Age minimum:
12 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Cystic Fibrosis
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Intervention(s)
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Drug: IVA
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Drug: TEZ/IVA
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Drug: ELX/TEZ/IVA
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Primary Outcome(s)
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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]
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Secondary Outcome(s)
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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]
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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]
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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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Absolute change in sweat chloride (SwCl) for ELX/TEZ/IVA group
[Time Frame: From Baseline through Week 8]
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Absolute change in ppFEV1 for ELX/TEZ/IVA group compared to the control group
[Time Frame: From Baseline through Week 8]
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Absolute change in SwCl for ELX/TEZ/IVA group compared to the control group
[Time Frame: From Baseline through Week 8]
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Secondary ID(s)
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VX18-445-104
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2018-002835-76
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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