A Study to Evaluate Safety and Pharmacokinetics of VX-659 in Healthy Subjects and in Adults With Cystic Fibrosis
![]() |
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: NCT03029455 |
Recruitment Status :
Completed
First Posted : January 24, 2017
Last Update Posted : September 5, 2017
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Cystic Fibrosis | Drug: VX-659 Drug: Tezacaftor Drug: Ivacaftor Drug: VX-659 Matching Placebo Drug: Triple Combination (TC) Matching Placebos | Phase 1 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 163 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Triple (Participant, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official Title: | A Phase 1, Randomized, Double Blind, Placebo Controlled, Dose Escalation, and Bioavailability Study Evaluating the Safety and Pharmacokinetics of VX-659 in Healthy Subjects and in Subjects With Cystic Fibrosis |
Study Start Date : | November 2016 |
Actual Primary Completion Date : | August 2017 |
Actual Study Completion Date : | August 2017 |

Arm | Intervention/treatment |
---|---|
Experimental: Part A: VX-659 or Matching Placebo
Part A includes single-dose escalation.
|
Drug: VX-659 Drug: VX-659 Matching Placebo |
Experimental: Part B: VX-659 or Matching Placebo
Part B includes multiple-dose escalation.
|
Drug: VX-659 Drug: VX-659 Matching Placebo |
Experimental: Part C: VX-659 in TC with TEZ/IVA or Matching Triple Placebo
Part C includes multiple dose escalation of VX-659 administered in Triple Combination (TC).
|
Drug: VX-659 Drug: Tezacaftor Other Names:
Drug: Ivacaftor Other Names:
Drug: Triple Combination (TC) Matching Placebos |
Experimental: Part D: VX-659 in TC with TEZ/IVA or Matching Triple Placebo
Part D includes subjects with CF. Participants will receive TC or matching placebos.
|
Drug: VX-659 Drug: Tezacaftor Other Names:
Drug: Ivacaftor Other Names:
Drug: Triple Combination (TC) Matching Placebos |
- Safety and Tolerability assessments as determined by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: from baseline up to Day 50 ]
- Maximum observed concentration (Cmax) of VX-659 and selected metabolites (μg/mL) [ Time Frame: from baseline up to Day 18 ]
- Cmax of TEZ and selected metabolites (μg/mL) [ Time Frame: from baseline up to Day 18 ]
- Cmax of IVA and selected metabolites (μg/mL) [ Time Frame: from baseline up to Day 18 ]
- Area under the concentration versus time curve during a dosing interval (AUCtau) of VX-659 and selected metabolites (μg,h/mL) [ Time Frame: from baseline up to Day 18 ]
- AUCtau of TEZ and selected metabolites (μg,h/mL) [ Time Frame: from baseline up to Day 18 ]
- AUCtau of IVA and selected metabolites (μg,h/mL) [ Time Frame: from baseline up to Day 18 ]
- Observed pre-dose concentration (Ctrough) of VX-659 and selected metabolites (μg/mL) [ Time Frame: from baseline up to Day 18 ]
- Ctrough of TEZ and selected metabolites (μg/mL) [ Time Frame: from baseline up to Day 18 ]
- Ctrough of IVA and selected metabolites (μg/mL) [ Time Frame: from baseline up to Day 18 ]

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | Yes |
Inclusion Criteria:
Healthy Volunteers: PARTS A, B, and C
- Males and Females of non-childbearing potential.
- Between the ages of 18 and 60 years inclusive
- Healthy, as defined per protocol.
- Body mass index (BMI) of 18.0 to 32.0 kg/m2 inclusive
- Body weight >50 kg
CF Patients: PART D
- Body weight ≥35 kg.
- Males and Females of non-childbearing potential.
- Sweat chloride value ≥ 60 mmol/L at screening.
- Heterozygous for F508del and a minimal function CFTR mutation
- Forced expiratory volume in 1 second (FEV1) ≥40% and ≤90% of predicted at screening
Exclusion Criteria:
Healthy Volunteers: PARTS A, B, and C
- History of any illness or any clinical condition that in the opinion of the investigator might confound the results of the study or pose additional risk to the subject.
- Any condition possibly affecting drug absorption.
- History of febrile illness within 14 days before the first study drug dose.
- Glucose-6-phosphate dehydrogenase (G6PD) deficiency assessed at Screening.
CF Patients: PART D
- History of any comorbidity that, in the opinion of the investigator, might confound the results of the study or pose an additional risk to the subject.
- History of cirrhosis with portal hypertension.
- Risk factors for Torsade de Pointes.
- G6PD deficiency assessed at Screening.
- Abnormal Laboratory Values.
- Lung infection with organisms associated with a more rapid decline in pulmonary status
- History of solid organ or hematological transplantation.

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): NCT03029455
United Kingdom | |
Birmingham, United Kingdom | |
Cambridge, United Kingdom | |
Exeter, United Kingdom | |
Glasgow, United Kingdom | |
Leeds, United Kingdom | |
Liverpool, United Kingdom | |
London, United Kingdom | |
Manchester, United Kingdom | |
Newcastle upon Tyne, United Kingdom | |
Southampton, United Kingdom |
Responsible Party: | Vertex Pharmaceuticals Incorporated |
ClinicalTrials.gov Identifier: | NCT03029455 |
Other Study ID Numbers: |
VX16-659-001 2016-003048-35 ( EudraCT Number ) |
First Posted: | January 24, 2017 Key Record Dates |
Last Update Posted: | September 5, 2017 |
Last Verified: | September 2017 |
Cystic Fibrosis Fibrosis Pathologic Processes Pancreatic Diseases Digestive System Diseases Lung Diseases Respiratory Tract Diseases |
Genetic Diseases, Inborn Infant, Newborn, Diseases Ivacaftor VX-659 Chloride Channel Agonists Membrane Transport Modulators Molecular Mechanisms of Pharmacological Action |