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

Cystic Fibrosis Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03029455
• Other study ID #: VX16-659-001
• Secondary ID: 2016-003048-35
• Status: Completed
• Phase: Phase 1
• First received: January 11, 2017
• Last updated: September 1, 2017
• Start date: November 2016
• Est. completion date: August 2017

Study information

• Verified date: September 2017
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 163
• Est. completion date: August 2017
• Est. primary completion date: August 2017
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• VX-659

• Tezacaftor

• Ivacaftor

• VX-659 Matching Placebo

• Triple Combination (TC) Matching Placebos

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety and Tolerability assessments as determined by number of subjects with adverse events (AEs) and serious adverse events (SAEs)		from baseline up to Day 50
Secondary	Maximum observed concentration (Cmax) of VX-659 and selected metabolites (µg/mL)		from baseline up to Day 18
Secondary	Cmax of TEZ and selected metabolites (µg/mL)		from baseline up to Day 18
Secondary	Cmax of IVA and selected metabolites (µg/mL)		from baseline up to Day 18
Secondary	Area under the concentration versus time curve during a dosing interval (AUCtau) of VX-659 and selected metabolites (µg,h/mL)		from baseline up to Day 18
Secondary	AUCtau of TEZ and selected metabolites (µg,h/mL)		from baseline up to Day 18
Secondary	AUCtau of IVA and selected metabolites (µg,h/mL)		from baseline up to Day 18
Secondary	Observed pre-dose concentration (Ctrough) of VX-659 and selected metabolites (µg/mL)		from baseline up to Day 18
Secondary	Ctrough of TEZ and selected metabolites (µg/mL)		from baseline up to Day 18
Secondary	Ctrough of IVA and selected metabolites (µg/mL)		from baseline up to Day 18