Trial record 2 of 7 for:
vx659
A Study of VX-659 Combination Therapy in CF Subjects Homozygous for F508del (F/F)
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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: NCT03460990 |
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Recruitment Status :
Completed
First Posted : March 9, 2018
Results First Posted : October 17, 2019
Last Update Posted : October 17, 2019
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Sponsor:
Vertex Pharmaceuticals Incorporated
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
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Brief Summary:
This study will evaluate the efficacy of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation (F/F).
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Cystic Fibrosis | Drug: VX-659/TEZ/IVA Drug: TEZ/IVA Drug: IVA Drug: Placebo | Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 116 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-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del Mutation (F/F) |
| Actual Study Start Date : | May 1, 2018 |
| Actual Primary Completion Date : | September 26, 2018 |
| Actual Study Completion Date : | October 8, 2018 |
Resource links provided by the National Library of Medicine
Genetics Home Reference related topics:
Cystic fibrosis
MedlinePlus related topics:
Cystic Fibrosis
Drug Information available for:
Ivacaftor
| Arm | Intervention/treatment |
|---|---|
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Active Comparator: TEZ/IVA
Following a run-in period of 4 weeks with Tezacaftor (TEZ)/Ivacaftor (IVA), participants received TEZ 100 milligram (mg)/IVA 150 mg as fixed-dose combination (FDC) tablet in the morning and IVA 150 mg as mono tablet in the evening for 4 weeks in the triple combination (TC) treatment period.
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Drug: TEZ/IVA
Participants received TEZ/IVA orally once daily in the morning.
Other Names:
Drug: IVA Participants received IVA orally once daily in the evening.
Other Names:
Drug: Placebo Participants received placebo matched to VX-659/TEZ/IVA orally once daily in the morning. |
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Experimental: VX-659/TEZ/IVA TC
Following a run-in period of 4 weeks with TEZ/IVA, participants received VX-659 240 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA 150 mg as mono tablet in the evening for 4 weeks in the TC treatment period.
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Drug: VX-659/TEZ/IVA
Participants received VX-659/TEZ/IVA orally once daily in the morning.
Other Names:
Drug: IVA Participants received IVA orally once daily in the evening.
Other Names:
Drug: Placebo Participants received placebo matched TEZ/IVA orally once daily in the morning. |
Primary Outcome Measures :
- Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) [ Time Frame: From Baseline at Week 4 ]FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Secondary Outcome Measures :
- Absolute Change in Sweat Chloride (SwCl) [ Time Frame: From Baseline at Week 4 ]Sweat samples were collected using an approved collection device.
- Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score [ Time Frame: From Baseline at Week 4 ]The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
- Safety and Tolerability as Assessed Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: From first dose of study drug in TC treatment period up to 28 days after last dose of study drug or to the completion of study participation date, whichever occurs first (up to Week 8) ]
- Observed Pre-Dose Concentration (Ctrough) of VX-659, TEZ, TEZ Metabolite (M1-TEZ), and IVA [ Time Frame: From Day 1 and Week 4 ]
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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:
- Homozygous for the F508del mutation (F/F)
- 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
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): NCT03460990
Locations
Show 46 study locations
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
Study Documents (Full-Text)
Documents provided by Vertex Pharmaceuticals Incorporated:
Documents provided by Vertex Pharmaceuticals Incorporated:
Study Protocol [PDF] April 27, 2018
Statistical Analysis Plan [PDF] June 7, 2018
| Responsible Party: | Vertex Pharmaceuticals Incorporated |
| ClinicalTrials.gov Identifier: | NCT03460990 |
| Other Study ID Numbers: |
VX17-659-103 2017-004133-82 ( EudraCT Number ) |
| First Posted: | March 9, 2018 Key Record Dates |
| Results First Posted: | October 17, 2019 |
| Last Update Posted: | October 17, 2019 |
| Last Verified: | September 2019 |
| 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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VX-659 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 |