Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02797132
• Date of registration: 25/05/2016
• Prospective Registration: No
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del
• Scientific 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
• Date of first enrolment: May 2016
• Target sample size: 62
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02797132
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Canada, United States

Key inclusion & exclusion 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



Age minimum: 2 Years
Age maximum: 5 Years
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: LUM/IVA

Primary Outcome(s)

Part B: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) [Time Frame: Day 1 up to Week 26]

Part A: Pre-dose Concentration (Ctrough) of LUM and IVA [Time Frame: Day 15]

Secondary Outcome(s)

Part B: Absolute Change From Baseline in Stature-for-Age Z-Score [Time Frame: Baseline, Week 24]

Part B: Absolute Change From Baseline in Serum Levels of Immunoreactive Trypsinogen (IRT) Through Week 24 [Time Frame: Baseline, Through Week 24]

Part B: Absolute Change From Baseline in Stature (Height) at Week 24 [Time Frame: Baseline, Week 24]

Part A: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) [Time Frame: Day 1 up to Day 25]

Part B: Absolute Change From Baseline in Body Mass Index (BMI) at Week 24 [Time Frame: Baseline, Week 24]

Part B: Number of Pulmonary Exacerbations [Time Frame: Through Week 24]

Part B: Acceptability/Palatability of LUM/IVA Granules Measured Using Hedonic Scale [Time Frame: Day 1]

Part B: Number of Cystic Fibrosis (CF)-Related Hospitalizations [Time Frame: Through Week 24]

Part A: Pre-dose Concentration (Ctrough) of LUM and IVA Metabolites [Time Frame: Day 15]

Part B: Number of Participants With at Least One Pulmonary Exacerbation Pulmonary Exacerbation Through Week 24 [Time Frame: Through Week 24]

Part B: Number of Participants With Microbiology Culture Status (Positive or Negative) at Week 24 [Time Frame: Baseline and Week 24]

Part B: Absolute Change From Baseline in Body Mass Index (BMI) For-Age Z-Score at Week 24 [Time Frame: Baseline, Week 24]

Part B: Absolute Change From Baseline in Fecal Elastase-1 (FE-1) Levels at Week 24 [Time Frame: Baseline, Week 24]

Part B: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Week 24 [Time Frame: Baseline, Week 24]

Part B: Absolute Change From Baseline in Lung Clearance Index (LCI) 2.5 at Week 24 [Time Frame: Baseline, Week 24]

Part B: Absolute Change From Baseline in Lung Clearance Index (LCI) 5.0 at Week 24 [Time Frame: Baseline, Week 24]

Part B: Absolute Change From Baseline in Sweat Chloride at Week 24 [Time Frame: Baseline, Week 24]

Part B: Absolute Change From Baseline in Weight at Week 24 [Time Frame: Baseline, Week 24]

Part B: Absolute Change From Baseline in Weight-for-age Z-Score at Week 24 [Time Frame: Baseline, Week 24]

Part B: Pre-dose Concentration (Ctrough) of LUM and IVA and Its Metabolites [Time Frame: Week 24]

Part B: Absolute Change in Sweat Chloride From Week 24 at Week 26 [Time Frame: Week 24, Week 26]

Secondary ID(s)

2016-001004-33

VX15-809-115

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 30/10/2018
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02797132