Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With Advanced Lung Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT02390219
• Date of registration: 11/03/2015
• Prospective Registration: No
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With Advanced Lung Disease
• Scientific title: A Phase 3b, Open-Label Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With Cystic Fibrosis and Advanced Lung Disease, Homozygous for the F508del-CFTR Mutation
• Date of first enrolment: March 2015
• Target sample size: 46
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02390219
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

United States

Key inclusion & exclusion criteria

Inclusion Criteria:

- Homozygous for the F508del-CFTR mutation; historical genotype must be documented in
the participant's source documents.

- Percent predicted FEV1 <40 of adjusted for age, sex, and height at Screening

Exclusion Criteria:

- Participant currently receiving invasive mechanical ventilation.

- History of any comorbidity 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
participant

- Any clinically significant laboratory abnormalities at screening that would interfere
with the study assessments or pose an undue risk for the subject

- A 12-lead electrocardiograms (ECG) demonstrating QTcF >450 msec at Screening

- 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 CYP3A

- Pregnant and nursing females: Females of childbearing potential must have a negative
pregnancy test at Screening and Day 1.

- Sexually active subjects of reproductive potential who are not willing to follow the
contraception requirements

- Use of beta blockers or the equivalent at Screening.



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

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Advanced Lung Disease

Intervention(s)

Drug: Ivacaftor

Drug: Lumacaftor

Primary Outcome(s)

Number of Participants With Treatment Emergent Adverse Events (AEs) or Serious Adverse Events (SAEs) [Time Frame: Day 1 up to Week 28]

Secondary Outcome(s)

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

Number of Hospitalizations [Time Frame: Baseline through Week 24]

Duration For Which Participants Received Intravenous (IV) Antibiotics [Time Frame: Baseline through Week 24]

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

Secondary ID(s)

VX14-809-106

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 01/11/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02390219