Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Homozygous for the F508del-CFTR Mutation

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00953706
• Date of registration: 04/08/2009
• Prospective Registration: Yes
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Homozygous for the F508del-CFTR Mutation DISCOVER
• Scientific title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Safety and Efficacy of VX-770 in Subjects Aged 12 Years and Older With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation
• Date of first enrolment: September 2009
• Target sample size: 140
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT00953706
• Study type: Interventional
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Patrick A Flume, MD
• Affiliation: Medical University of South Carolina

Key inclusion & exclusion criteria

Inclusion Criteria:

- Confirmed diagnosis of cystic fibrosis (CF) and homozygous for F508del-CFTR mutation

- Forced expiratory volume in 1 second (FEV1) of at least 40% of predicted normal for
age, gender, and height

- Willing to use at least 2 highly effective birth control methods during the study

- No clinically significant abnormalities that would have interfered with the study
assessments, as judged by the investigator

- Able to understand and comply with protocol requirements, restrictions, and
instructions and likely to complete the study as planned, as judged by the
investigator

Exclusion Criteria:

- History of any illness or condition that might confound the results of the study or
pose an additional risk in administering study drug to the subject

- Acute respiratory infection, pulmonary exacerbation, or changes in therapy for
pulmonary disease within 4 weeks of Day 1 of the study

- History of alcohol, medication or illicit drug abuse within one year prior to Day 1

- Abnormal liver function >=3 x the upper limit of normal

- Abnormal renal function at Screening

- History of solid organ or hematological transplantation

- Pregnant or breast-feeding (for women)

- Ongoing participation in another therapeutic clinical study or prior participation in
an investigational drug study within 30 days prior to screening

- Previous participation in a VX-809 study

- Used inhaled hypertonic saline treatment

- Concomitant use of any inhibitors or inducers of cytochrome P450 3A4 (CYP3A4)



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

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: Placebo

Drug: Ivacaftor

Primary Outcome(s)

Part A : Absolute Change From Part A Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 16 [Time Frame: Part A baseline through Week 16]

Secondary Outcome(s)

Part A : Absolute Change From Part A Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 16 [Time Frame: Part A baseline through Week 16]

Part A : Absolute Change From Part A Baseline in Sweat Chloride Concentration Through Week 16 [Time Frame: Part A baseline through Week 16]

Part B : Number of Pulmonary Exacerbation Events Per Participant Per Year [Time Frame: Part B baseline through Week 64]

Part A : Rate of Change From Baseline in Weight Through Week 16 [Time Frame: Part A baseline through Week 16]

Part B : Absolute Change From Part A and Part B Baseline in ppFEV1 Through Week 64 [Time Frame: Change from Part A baseline: Part A Baseline, Week 64; Change from Part B baseline: Part B Baseline (Week 16), Week 64]

Part B : Absolute Change From Part A and Part B Baseline in Weight Through Week 64 [Time Frame: Change from Part A baseline: Part A Baseline, Week 64; Change from Part B baseline: Part B Baseline (Week 16), Week 64]

Part B : Rate of Change From Part B Baseline in ppFEV1 Through Week 64 [Time Frame: Part B baseline through Week 64]

Part B : Number of Participants With Pulmonary Exacerbations [Time Frame: Part B baseline through Week 64]

Part B : Absolute Change From Part A and Part B Baseline in CFQ-R Respiratory Domain Score Through Week 64 [Time Frame: Change from Part A baseline: Part A Baseline, Week 64; Change from Part B baseline: Part B Baseline (Week 16), Week 64]

Part B : Number of Pulmonary Exacerbation Events [Time Frame: Part B baseline through Week 64]

Part B : Rate of Change From Part A Baseline in ppFEV1 Through Week 64 [Time Frame: Part A baseline through Week 64]

Part B : Absolute Change From Part A and Part B Baseline in Sweat Chloride Concentration Through Week 64 [Time Frame: Change from Part A baseline: Part A Baseline, Week 64; Change from Part B baseline: Part B Baseline (Week 16), Week 64]

Secondary ID(s)

VX08-770-104

2009-010261-23

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Cystic Fibrosis Foundation Therapeutics

Results

• Results available: Yes
• Date Posted: 21/08/2012
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00953706