A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT01807923
• Date of registration: 04/03/2013
• Prospective Registration: Yes
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation TRAFFIC
• Scientific title: A 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
• Date of first enrolment: May 2013
• Target sample size: 559
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01807923
• Study type: Interventional
• Phase: Phase 3

Countries of recruitment

Australia, Canada, Czech Republic, France, Germany, Ireland, Italy, Netherlands, Sweden, United Kingdom, United States

Key inclusion & exclusion criteria

Inclusion Criteria:

- Confirmed diagnosis of CF

- Homozygous for the F508del CFTR mutation

- 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

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

Exclusion Criteria:

- 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 solid organ or hematological transplantation

- History of alcohol or drug abuse in the past year

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

- Use of strong inhibitors, moderate inducers or strong inducers of Cytochrome P450 3A
(CYP3A) within 14 days before Day 1 of dosing



Age minimum: 12 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis, Homozygous for the F508del CFTR Mutation

Intervention(s)

Drug: Ivacaftor

Drug: Lumacaftor Plus Ivacaftor Combination

Drug: Placebo

Primary Outcome(s)

Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 24 [Time Frame: Baseline, Week 16 and 24]

Secondary Outcome(s)

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

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

Percentage of Participants With Response Based on Percent Predicted FEV1 [Time Frame: Week 16 and 24]

Relative Change From Baseline in Percent Predicted FEV1 at Week 24 [Time Frame: Baseline, Week 16 and 24]

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

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

Pre-dose Concentration (Ctrough), Average Pre-dose Concentration (Ctrough,Avg), 3 to 6 Hours Post-dose Concentration (C3-6h), and Average 3 to 6 Hours Post-dose Concentration (C3-6h,Avg) [Time Frame: For C3-6h: 3 to 6 hours after morning dose on Day 1 and 15, Week 4 and 8; For C3-6h,avg 3 to 6 hours after morning dose on Day 15, Week 4 and 8; For Ctrough and Ctrough,avg: before morning dose on Week 4, 8, and 16]

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

Absolute Change From Baseline in Euro Quality of Life Scale (EuroQol) 5-Dimension-3 Level (EQ-5D-3L) Index Score at Week 24 [Time Frame: Baseline, Week 24]

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

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

Absolute Change From Baseline in EQ-5D-3L VAS Score at Week 24 [Time Frame: Baseline, Week 24]

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

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

Secondary ID(s)

VX12-809-103

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 31/08/2015
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01807923