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Trial record 1 of 1 for:    VX15-809-115
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Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT02797132
First received: May 25, 2016
Last updated: September 12, 2017
Last verified: August 2017
  Purpose
This is a Phase 3, 2-part (Part A and Part B), open-label, multicenter study evaluating the pharmacokinetics (PK), safety, tolerability, and pharmacodynamics (PD) of multiple doses of Lumacaftor/Ivacaftor (LUM/IVA) in subjects 2 through 5 years of age (inclusive) with Cystic Fibrosis (CF), homozygous for F508del. Subjects who participate in Part A may participate in Part B, if patient meet the eligibility criteria.

Condition Intervention Phase
Cystic Fibrosis Drug: lumacaftor/ ivacaftor Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 3, 2-Part, Open-label Study to Evaluate the Safety and Pharmacokinetics of Lumacaftor/Ivacaftor Combination Therapy in Subjects Aged 2 Through 5 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:
  • Part A: Pharmacokinetic (PK) parameters of lumacaftor (LUM) and ivacaftor (IVA): estimated peak concentrations (Cmax) [ Time Frame: up to 15 Days ]
  • Part A: Pharmacokinetic (PK) parameters of lumacaftor (LUM) and ivacaftor (IVA): estimated trough concentrations (Ctrough) [ Time Frame: up to 15 Days ]
  • Part B: Safety and Tolerability assessments as determined by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: Baseline up to 28 days after last dose (up to 28 weeks) ]

Secondary Outcome Measures:
  • Part A: PK parameters of LUM and IVA metabolites: estimated peak concentrations (Cmax) of lumacaftor and ivacaftor metabolites [ Time Frame: up to 15 days ]
  • Part A: PK parameters of LUM and IVA metabolites: estimated trough concentrations (Ctrough) of lumacaftor and ivacaftor metabolites [ Time Frame: up to 15 days ]
  • Part A: Safety and Tolerability assessments as determined by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: Baseline up to 10 +/- 3 days after the last dose (up to 25 +/- 3 days) ]
  • Part B: Absolute change from baseline in sweat chloride level [ Time Frame: Baseline, Week 24 ]
  • Part B: Absolute change from baseline in body mass index (BMI) and BMI for age z score [ Time Frame: Baseline, Week 24 ]
  • Part B: Absolute change from baseline in weight and weight for age z score [ Time Frame: Baseline, Week 24 ]
  • Part B: Absolute change from baseline in stature and stature for age z score [ Time Frame: Baseline, Week 24 ]
  • Part B: Absolute change from baseline in lung clearance index (LCI)2.5 [ Time Frame: Baseline, Week 24 ]
  • Part B: Absolute change from baseline in LCI5.0 [ Time Frame: Baseline, Week 24 ]
  • Part B: Time-to-first pulmonary exacerbation [ Time Frame: through Week 24 ]
  • Part B: Number of pulmonary exacerbations [ Time Frame: through Week 24 ]
  • Part B: Number of CF-related hospitalizations [ Time Frame: through Week 24 ]
  • Part B; Absolute change from Baseline in fecal elastase-1 (FE-1) levels [ Time Frame: Baseline, Week 24 ]
  • Part B: Absolute change in serum levels of immunoreactive trypsinogen (IRT) [ Time Frame: from baseline through Week 24 ]
  • Part B: Changes in sputum microbiology cultures [ Time Frame: Baseline, Week 24 ]
  • Part B: Absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1) [ Time Frame: Baseline, Week 24 ]
  • Part B: Acceptability/palatability of LUM/IVA granules measured using hedonic scale [ Time Frame: Day 1 ]
  • Part B: PK parameters of LUM, IVA, and their respective metabolites: estimated trough concentrations (Ctrough) of LUM and IVA and their metabolites [ Time Frame: up to 24 weeks ]

Enrollment: 60
Study Start Date: May 2016
Study Completion Date: September 2017
Primary Completion Date: September 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Part A: LUM/IVA Subjects < 14Kg
lumacaftor 100 mg/ivacaftor 125 mg q12h
Drug: lumacaftor/ ivacaftor
Other Name: Orkambi
Experimental: Part A: LUM/IVA Subjects >= 14Kg
lumacaftor 150 mg/ivacaftor 188 mg q12h
Drug: lumacaftor/ ivacaftor
Other Name: Orkambi
Experimental: Part B: LUM/IVA Subjects < 14Kg
lumacaftor 100 mg/ivacaftor 125 mg q12h
Drug: lumacaftor/ ivacaftor
Other Name: Orkambi
Experimental: Part B: LUM/IVA Subjects >= 14Kg
lumacaftor 150 mg/ivacaftor 188 mg q12h
Drug: lumacaftor/ ivacaftor
Other Name: Orkambi

  Eligibility

Ages Eligible for Study:   2 Years to 5 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Subjects who weigh ≥8 kilogram (kg) without shoes and wearing light clothing at the Screening Visit
  • Subjects with confirmed diagnosis of CF at the Screening Visit
  • Subjects who are homozygous for the F508del Cystic fibrosis transmembrane conductance regulator (CFTR) mutation

Exclusion Criteria:

  • Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject
  • An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 28 days before Day 1
  • A standard 12 lead ECG demonstrating QTc >450 millisecond (msec) at the Screening Visit.
  • History of solid organ or hematological transplantation.
  • Ongoing or prior participation in an investigational drug study (including studies investigating LUM and/or IVA) within 30 days of the Screening Visit.
  • History of cataract/lens opacity or evidence of cataract/lens opacity determined to be clinically significant by a licensed ophthalmologist during the ophthalmologic examination 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: NCT02797132

  Show 20 Study Locations
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
  More Information

Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT02797132     History of Changes
Other Study ID Numbers: VX15-809-115
2016-001004-33 ( EudraCT Number )
Study First Received: May 25, 2016
Last Updated: September 12, 2017

Keywords provided by Vertex Pharmaceuticals Incorporated:
CF

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 September 19, 2017