Completed

TRANSPORTA Phase 3, Randomized, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation

What is being tested

Placebo

+ Lumacaftor Plus Ivacaftor Combination

+ Ivacaftor

Drug

Who is being recruted

Digestive System Diseases+5

+ Infant, Newborn, Diseases

+ Lung Diseases

From 12 to 65 Years

+9 Eligibility Criteria

See all eligibility criteria

How is the trial designed

Treatment Study

Placebo-ControlledPhase 3

Interventional

Study Start: April 2013

See protocol details

Summary

Principal SponsorVertex Pharmaceuticals Incorporated

Last updated: September 27, 2016

Sourced from a government-validated database.Claim as a partner

Study start date: April 1, 2013

Actual date on which the first participant was enrolled.

This was a Phase 3, randomized, double-blind, placebo-controlled, parallel-group multicenter study of orally administered lumacaftor in combination with ivacaftor in participants aged 12 years and older with CF who are homozygous for the F508del-CFTR mutation. The study included a Screening Period (Day -28 through Day -1), a Treatment Period (Day 1 [first dose of study drug] to Week 24 ± 5 days), and a Safety Follow-up Visit (4 weeks ± 7 days after the Week 24 Visit).

NCT01807949

Principal SponsorVertex Pharmaceuticals Incorporated

Last updated: September 27, 2016

Sourced from a government-validated database.Claim as a partner

Protocol

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

Design Details

563 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.

Conditions

Criteria

Any sex

Biological sex of participants that are eligible to enroll.

From 12 to 65 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Digestive System DiseasesInfant, Newborn, DiseasesLung DiseasesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesPancreatic DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCystic Fibrosis

Criteria

4 inclusion criteria required to participate

Confirmed diagnosis of CF

Forced expiratory volume in 1 second (FEV1) greater than or equal to ( >= ) 40 percent (%) and less than or equal to (= < ) 90% of predicted normal for age, sex, and height

Homozygous for the F508del CFTR mutation

Willing to remain on a stable CF medication regimen through Week 24 or, if applicable, the Safety Follow up Visit

5 exclusion criteria prevent from participating

An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before first dose of study drug

History of alcohol or drug abuse in the past year

History of solid organ or hematological transplantation

Ongoing or prior participation in an investigational drug study (including studies investigating lumacaftor and/or ivacaftor) within 30 days of screening

Show More Criteria

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.

Treatment Groups

Study Objectives

3 intervention groups are designated in this study

33.333% chance of being blinded to the placebo group

Treatment Groups

Group I

Placebo

Placebo matched to lumacaftor (LUM, VX-809) and ivacaftor (IVA, VX-770) tablet every 12 hours (q12h), up to Week 24.

Group II

Experimental

LUM 600 milligram (mg) plus IVA 250 mg fixed-dose combination (FDC) tablet in the morning and IVA 250 mg film-coated tablet in the evening, up to Week 24.

Group III

Experimental

LUM 400 mg plus IVA 250 mg FDC tablet in the morning and in the evening, up to Week 24.

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 82 locations

Oakland, United StatesOpen in Google Maps

Sacramento, United States

Aurora, United States

Hartford, United States

Completed82 Study Centers