A Study of VX-659 Combination Therapy in CF Subjects Homozygous for F508del (F/F)
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| ClinicalTrials.gov Identifier: NCT03460990 |
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Recruitment Status :
Completed
First Posted : March 9, 2018
Last Update Posted : December 4, 2018
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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 Drug: TEZ Drug: IVA Drug: TEZ/IVA | Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 116 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | Triple (Participant, Care Provider, Investigator) |
| 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
| Arm | Intervention/treatment |
|---|---|
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Experimental: Triple Combination
Subjects will receive 240 mg VX-659 / 100 mg TEZ / 150 mg IVA as FDC tablets in the morning and 150 mg IVA as mono tablet in the evening
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Drug: VX-659
Fixed-dose combination (FDC) tablet (VX-659/TEZ/IVA) Drug: TEZ FDC tablet (VX-659/TEZ/IVA)
Other Name: tezacaftor; VX-661 Drug: IVA FDC tablet (VX-659/TEZ/IVA)
Other Name: ivacaftor; VX-770 Drug: IVA IVA tablet
Other Name: ivacaftor; VX-770 |
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Active Comparator: TEZ/IVA
Subjects will receive 100 mg TEZ/150 mg IVA as FDC tablets in the morning and 150 mg IVA as mono tablet in the evening
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Drug: TEZ/IVA
FDC tablet TEZ/IVA
Other Name: ivacaftor/tezacaftor Drug: IVA IVA tablet
Other Name: ivacaftor; VX-770 |
Primary Outcome Measures :
- Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1) [ Time Frame: from baseline at Week 4 ]
Secondary Outcome Measures :
- Absolute change in CF Questionnaire-Revised (CFQ-R) respiratory domain score [ Time Frame: from baseline at Week 4 ]
- Absolute change in Sweat Chloride (SwCl) [ Time Frame: from baseline at Week 4 ]
- Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: from baseline through 4-week safety follow-up (up to12 weeks) ]
- Observed pre-dose concentration (Ctrough) of VX-659, TEZ, M1-TEZ, and IVA [ Time Frame: from Day 1 through 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
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Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
| Responsible Party: | Vertex Pharmaceuticals Incorporated |
| ClinicalTrials.gov Identifier: | NCT03460990 History of Changes |
| Other Study ID Numbers: |
VX17-659-103 2017-004133-82 ( EudraCT Number ) |
| First Posted: | March 9, 2018 Key Record Dates |
| Last Update Posted: | December 4, 2018 |
| Last Verified: | December 2018 |
| 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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Fibrosis Cystic 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 |