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A Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects With CF, Homozygous for the F508del-CFTR Mutation

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT02514473
First received: July 23, 2015
Last updated: October 24, 2016
Last verified: March 2016
  Purpose
To evaluate the efficacy and safety of lumacaftor in combination with ivacaftor in subjects aged 6 Through 11 years with cystic fibrosis (CF), homozygous for the F508del CF transmembrane conductance regulator (CFTR) mutation

Condition Intervention Phase
Cystic Fibrosis Drug: VX-809 Drug: Placebo Drug: VX-770 Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 3, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

Resource links provided by NLM:


Further study details as provided by Vertex Pharmaceuticals Incorporated:

Primary Outcome Measures:
  • Absolute change in lung clearance index (LCI) [ Time Frame: From Baseline through Week 24 ]

Secondary Outcome Measures:
  • Average absolute change in sweat chloride [ Time Frame: From Baseline at Day 15 and at Week 4 ]
  • Absolute change in body mass index (BMI) [ Time Frame: From Baseline at Week 24 ]
  • Absolute change in Cystic Fibrosis Questionnaire Revised (CFQ R) respiratory domain score [ Time Frame: From Baseline through Week 24 ]
  • Absolute change in LCI5.0 [ Time Frame: From Baseline through Week 24 ]
  • Absolute change in sweat chloride [ Time Frame: From Baseline at Week 24 ]
  • Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1) [ Time Frame: From Baseline through Week 24 ]
  • Relative change in ppFEV1 [ Time Frame: From Baseline through Week 24 ]
  • Absolute change in BMI-for-age z-score [ Time Frame: From Baseline at Week 24 ]
  • Absolute change in weight [ Time Frame: From Baseline at Week 24 ]
  • Absolute change in weight-for-age z-score [ Time Frame: From Baseline at Week 24 ]
  • Absolute change in height [ Time Frame: From Baseline at Week 24 ]
  • Absolute change in height-for-age z-score [ Time Frame: From Baseline at Week 24 ]
  • Absolute change in Treatment Satisfaction Questionnaire for Medication (TSQM) domains [ Time Frame: From Baseline Through Week 24 ]
  • Time-to-first pulmonary exacerbation [ Time Frame: through Week 24 ]
  • Event of having at least 1 pulmonary exacerbation [ Time Frame: through Week 24 ]
  • Number of pulmonary exacerbations [ Time Frame: through Week 24 ]
  • Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: through Week 24 ]
  • Pharmacokinetic parameters: estimated peak concentrations and trough concentrations (C3-6h and Ctrough) of lumacaftor, M28-lumacaftor, ivacaftor, M1 ivacaftor and M6 ivacaftor [ Time Frame: through Week 24 ]

Enrollment: 206
Study Start Date: July 2015
Study Completion Date: September 2016
Primary Completion Date: September 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: LUM/IVA
Fixed-dose combination with lumacaftor (LUM) 200 mg every 12 hours (q12h)/ ivacaftor (IVA) 250 mg q12h
Drug: VX-809
Other Name: lumacaftor
Drug: VX-770
Other Name: ivacaftor
Placebo Comparator: Placebo
Matching placebo q12h
Drug: Placebo

  Eligibility

Ages Eligible for Study:   6 Years to 11 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Subjects who weigh ≥15 kg without shoes a the Screening Visit
  • Subjects with confirmed diagnosis of CF at the Screening Visit.
  • Subjects who are homozygous for the F508del CFTR mutation
  • Subjects with ppFEV1 of ≥70 percentage points adjusted for age, sex, and height
  • Subjects with a screening LCI2.5 result greater than or equal to 7.5

Exclusion Criteria:

  • History of any comorbidity reviewed at the Screening Visit that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject.
  • Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject
  • Clinically significant abnormalities in hemoglobin, liver function, or renal function at the Screening Visit.
  • An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 28 days before Day 1
  • History of solid organ or hematological transplantation at the Screening Visit
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02514473

  Show 54 Study Locations
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
  More Information

Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT02514473     History of Changes
Other Study ID Numbers: VX14-809-109
Study First Received: July 23, 2015
Last Updated: October 24, 2016

Additional relevant MeSH terms:
Fibrosis
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases

ClinicalTrials.gov processed this record on July 26, 2017