NCT03486236

Brief Summary

This study evaluated the safety and pharmacokinetics of multiple ascending doses of VX-440 in combination with tezacaftor/ivacaftor (TEZ/IVA) (triple combination \[TC\]) administered for 13 days to healthy male and female subjects

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
16

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Jul 2016

Shorter than P25 for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

July 20, 2016

Completed
2 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 14, 2016

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 14, 2016

Completed
1.5 years until next milestone

First Submitted

Initial submission to the registry

March 27, 2018

Completed
7 days until next milestone

First Posted

Study publicly available on registry

April 3, 2018

Completed
Last Updated

April 3, 2018

Status Verified

March 1, 2018

Enrollment Period

2 months

First QC Date

March 27, 2018

Last Update Submit

March 27, 2018

Conditions

Outcome Measures

Primary Outcomes (1)

  • Safety and tolerability were based on the number and assessment of adverse events (AEs) and serious adverse events (SAEs)

    from baseline through safety follow-up visit (up to 29 days)

Secondary Outcomes (3)

  • Maximum observed concentration (Cmax) of VX-440, TEZ and its metabolites (M1-TEZ and M2-TEZ), and IVA and its metabolites (M1-IVA and M6-IVA)

    from Day 1 through Day 18

  • Area under the concentration versus time curve during a dosing interval (AUCtau) of VX-440, TEZ and its metabolites (M1-TEZ and M2-TEZ), and IVA and its metabolites (M1-IVA and M6-IVA)

    from Day 1 through Day 18

  • Observed pre-dose concentration (Ctrough) of VX-440, TEZ and its metabolites (M1-TEZ and M2-TEZ), and IVA and its metabolites (M1-IVA and M6-IVA)

    from Day 1 through Day 18

Study Arms (4)

Cohort C1

EXPERIMENTAL
Drug: VX-440Drug: TEZDrug: IVA

Cohort C1: Triple Placebo

PLACEBO COMPARATOR
Drug: Matched Placebos

Cohort C2

EXPERIMENTAL
Drug: VX-440Drug: TEZDrug: IVA

Cohort C2: Triple Placebo

PLACEBO COMPARATOR
Drug: Matched Placebos

Interventions

VX-440DRUG

VX-440 was administered in TC with TEZ and IVA.

Cohort C1Cohort C2
TEZDRUG

TEZ was administered as part of a fixed-dose combination (FDC) tablet (TEZ/IVA)

Also known as: Tezacaftor, VX-661
Cohort C1Cohort C2
IVADRUG

IVA was administered as part of a FDC tablet (TEZ/IVA) and as a mono tablet

Also known as: Ivacaftor, VX-770
Cohort C1Cohort C2

Placebos matched to VX-440, TEZ, and IVA.

Cohort C1: Triple PlaceboCohort C2: Triple Placebo

Eligibility Criteria

Age18 Years - 55 Years
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64)

You may qualify if:

  • Female subjects of non-childbearing potential only.
  • Body mass index (BMI) of 18.0 to 31.0 kg/m2, inclusive, and a total body weight \>50 kg.
  • Normal pulmonary function measurements, defined as forced expiratory volume in 1 second (FEV1) and forced vital capacity (FVC) both ≥80% of their predicted value at screening.

You may not qualify if:

  • For female subjects: Pregnant or nursing subjects.
  • Glucose-6-phosphate dehydrogenase (G6PD) deficiency.
  • History of hemolysis.
  • Total bilirubin level \>2 × ULN at Screening.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

The Medicines Evaluation Unit

Manchester, United Kingdom

Location

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

tezacaftorivacaftor

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, Diseases

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 27, 2018

First Posted

April 3, 2018

Study Start

July 20, 2016

Primary Completion

September 14, 2016

Study Completion

September 14, 2016

Last Updated

April 3, 2018

Record last verified: 2018-03

Locations