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

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT02514473
• Date of registration: 23/07/2015
• Prospective Registration: No
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: A Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects With CF, Homozygous for the F508del-CFTR Mutation
• Scientific 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
• Date of first enrolment: July 2015
• Target sample size: 206
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02514473
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Australia, Belgium, Canada, Denmark, France, Germany, Sweden, United Kingdom, United States

Key inclusion & exclusion 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



Age minimum: 6 Years
Age maximum: 11 Years
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: VX-770

Drug: VX-809

Drug: Placebo

Primary Outcome(s)

Absolute Change From Baseline in Lung Clearance Index 2.5 (LCI2.5) Through Week 24 [Time Frame: Baseline, Through Week 24]

Secondary Outcome(s)

Absolute Change From Baseline in Lung Clearance Index 5.0 (LCI5.0) Through Week 24 [Time Frame: Baseline, Through Week 24]

Average Absolute Change From Baseline in Sweat Chloride at Day 15 and Week 4 [Time Frame: Baseline, Day 15 and Week 4]

Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) [Time Frame: Baseline up to Week 28]

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

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

Absolute Change From Baseline in BMI-for-age Z-score at Week 24 [Time Frame: Baseline, Week 24]

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

Absolute Change From Baseline in Height at Week 24 [Time Frame: Baseline, Week 24]

Absolute Change From Baseline in Treatment Satisfaction Questionnaire for Medication (TSQM) Domains Through Week 24 [Time Frame: Baseline, Through Week 24]

Number of Pulmonary Exacerbation Events [Time Frame: Baseline through Week 24]

Time-to-first Pulmonary Exacerbation [Time Frame: Baseline through Week 24]

Percentage of Participants With At Least 1 Pulmonary Exacerbation Event [Time Frame: Baseline through Week 24]

Relative Change From Baseline in ppFEV1 Through Week 24 [Time Frame: Baseline, Through Week 24]

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

Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 24 [Time Frame: Baseline, Through Week 24]

Absolute Change From Baseline in Height-for-age Z-score at Week 24 [Time Frame: Baseline, Week 24]

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

Average Pre-dose Concentration (Ctrough,Ave) and Average 3 to 6 Hours Post-dose Concentration (C3-6h,Ave) For Lumacaftor and Ivacaftor [Time Frame: For Ctrough,ave: before morning dose on Week 4 and 24; For C3-6h,ave: 3 to 6 hours after morning dose on Day 1, 15 and Week 4]

Secondary ID(s)

VX14-809-109

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 23/10/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02514473