A Study to Evaluate Safety and Pharmacokinetics of VX-659 in Healthy Subjects and in Adults With Cystic Fibrosis

September 1, 2017 updated by: Vertex Pharmaceuticals Incorporated

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

Evaluate the safety and tolerability of VX-659 in healthy subjects

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

163

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • 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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

Yes

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: RANDOMIZED
  • Interventional Model: PARALLEL
  • Masking: TRIPLE

Arms and Interventions

Participant Group / 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: Ivacaftor
Other Names:
  • VX-770
  • IVA
Drug: VX-659
Drug: Tezacaftor
Other Names:
  • VX-661
  • TEZ
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: Ivacaftor
Other Names:
  • VX-770
  • IVA
Drug: VX-659
Drug: Tezacaftor
Other Names:
  • VX-661
  • TEZ
Drug: Triple Combination (TC) Matching Placebos

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
from baseline up to Day 50

Secondary Outcome Measures

Outcome Measure
Time Frame
Maximum observed concentration (Cmax) of VX-659 and selected metabolites (μg/mL)
Time Frame: from baseline up to Day 18
from baseline up to Day 18
Cmax of TEZ and selected metabolites (μg/mL)
Time Frame: from baseline up to Day 18
from baseline up to Day 18
Cmax of IVA and selected metabolites (μg/mL)
Time Frame: from baseline up to Day 18
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
from baseline up to Day 18
AUCtau of TEZ and selected metabolites (μg,h/mL)
Time Frame: from baseline up to Day 18
from baseline up to Day 18
AUCtau of IVA and selected metabolites (μg,h/mL)
Time Frame: from baseline up to Day 18
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
from baseline up to Day 18
Ctrough of TEZ and selected metabolites (μg/mL)
Time Frame: from baseline up to Day 18
from baseline up to Day 18
Ctrough of IVA and selected metabolites (μg/mL)
Time Frame: from baseline up to Day 18
from baseline up to Day 18

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Vertex Pharmaceuticals Incorporated

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

November 1, 2016

Primary Completion (ACTUAL)

August 1, 2017

Study Completion (ACTUAL)

August 1, 2017

Study Registration Dates

First Submitted

January 11, 2017

First Submitted That Met QC Criteria

January 20, 2017

First Posted (ESTIMATE)

January 24, 2017

Study Record Updates

Last Update Posted (ACTUAL)

September 5, 2017

Last Update Submitted That Met QC Criteria

September 1, 2017

Last Verified

September 1, 2017

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VX16-659-001
  • 2016-003048-35 (EUDRACT_NUMBER)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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