Study of VX-809 Alone and in Combination With VX-770 in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT01225211
• Date of registration: 15/10/2010
• Prospective Registration: No
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: Study of VX-809 Alone and in Combination With VX-770 in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation
• Scientific title: A Phase 2, Multicenter, Double-Blinded, Placebo-Controlled, Multiple-Dose Study to Evaluate Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Lumacaftor Monotherapy, and Lumacaftor and Ivacaftor Combination Therapy in Subjects With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation
• Date of first enrolment: October 2010
• Target sample size: 312
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01225211
• Study type: Interventional
• Phase: Phase 2

Countries of recruitment

Australia, Belgium, France, Germany, Ireland, New Zealand, United Kingdom, United States

Key inclusion & exclusion criteria

Inclusion Criteria:

- Male or female participants with confirmed diagnosis of CF

- Must have the F508del-CFTR mutation on at least 1 allele.

- FEV1 greater than equal (>=) 40% of predicted normal for age, gender, and height
(Knudson standards)(Cohort 1, 2, and 3); FEV1 40-90% of predicted normal for age,
gender, and height (Hankinson standards (Cohort 4)

- Participant of child-bearing potential and who are sexually active must meet the
contraception requirements

Exclusion Criteria:

- History of any illness or condition 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 (e.g., cirrhosis with portal hypertension).

- An acute illness including acute upper or lower respiratory infection, pulmonary
exacerbation or changes in therapy (including antibiotics) for pulmonary disease
within 14 days (Cohort 1, 2, and 3) or 28 days (Cohort 4) before receiving the first
dose of study drug.

- History of solid organ or hematological transplantation.

- History of alcohol abuse or drug addiction in the past year, including cannabis,
cocaine, and opiates.

- Ongoing participation in another therapeutic clinical study, or prior participation in
an investigational drug study without appropriate washout

- Pregnant or nursing females; females of childbearing potential who are unwilling or
unable to use an acceptable method of non-hormonal contraception

- Participants enrolled in Cohort 1 or Cohort 2 will not be eligible for Cohort 3

- Ongoing or prior participation in an investigational drug study within 30 days of the
Screening Visit

- Heterozygous participants who participated in Cohort 2 and meet the eligibility
criteria for Cohort 4 may participate in Cohort 4

- Evidence of lens opacity or cataract as determined by the ophthalmologic examination
(Cohort 4 only)



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: Ivacaftor Placebo

Drug: Lumacaftor Placebo

Drug: Lumacaftor

Drug: Ivacaftor

Primary Outcome(s)

Cohort 1: Safety and Tolerability Based on Adverse Events (AEs) [Time Frame: Cohort 1: Day 1 up to 28 days after last dose (Last dose = Day 21)]

Cohort 2 And 3: Absolute Change From Day 28 in Sweat Chloride at Day 56 [Time Frame: Cohort 2 and 3: Day 28, Day 56]

Cohort 1: Absolute Change From Day 14 in Sweat Chloride at Day 21 [Time Frame: Cohort 1: Day 14, Day 21]

Cohort 4: Safety and Tolerability Assessed by Number of Participants With AEs and SAEs [Time Frame: Cohort 4: Day 1 up to 28 days after last dose (Last dose = Day 56)]

Cohort 2 and 3: Safety and Tolerability Based on Adverse Events (AEs) [Time Frame: Cohort 2 and 3: Day 1 up to 28 days after last dose (Last dose = Day 56)]

Cohort 4: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 56 [Time Frame: Cohort 4: Baseline, Day 56]

Secondary Outcome(s)

Cohort 1: Absolute Change From Day 14 in FEV1 at Day 21 [Time Frame: Cohort 1: Day 14, Day 21]

Cohort 4: Relative Change From Baseline in Percent Predicted FEV1 at Day 56 [Time Frame: Cohort 4: Baseline, Day 56]

Cohort 2 and 3: Absolute Change From Baseline in ppFEV1 at Day 28 and 56 [Time Frame: Cohort 2 and 3: Baseline, Day 28 and 56]

Cohort 2 and 3: Relative Change From Day 28 in ppFEV1 at Day 56 [Time Frame: Cohort 2 and 3: Day 28, Day 56]

Cohort 4: Absolute Change From Baseline in Body Mass Index (BMI) at Day 56 [Time Frame: Cohort 4: Baseline, Day 56]

Cohort 4: Absolute Change From Baseline in CFQ-R Respiratory Domain Score at Day 56 [Time Frame: Cohort 4: Baseline, Day 56]

Cohort 1: Absolute Change From Day 14 in ppFEV1 at Day 21 [Time Frame: Cohort 1: Day 14, Day 21]

Cohort 2 And 3: Absolute Change From Baseline in Sweat Chloride at Day 14 [Time Frame: Cohort 2: Baseline, Day 14]

Cohort 2 and 3: Relative Change From Baseline in FEV1 at Day 28 and 56 [Time Frame: Cohort 2 and 3: Baseline, Day 28 and 56]

Cohort 4: Absolute Change From Baseline in Weight at Day 56 [Time Frame: Cohort 4: Baseline, Day 56]

Cohort 4: Absolute Change From Baseline in Sweat Chloride at Day 56 [Time Frame: Cohort 4: Baseline, Day 56]

Cohort 2 and 3: Absolute Change From Day 28 in ppFEV1 at Day 56 [Time Frame: Cohort 2 and 3: Day 28, Day 56]

Cohort 1: Absolute Change From Baseline in Sweat Chloride at Day 14 [Time Frame: Cohort 1: Baseline, Day 14]

Cohort 2 and 3: Absolute Change From Day 28 in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Day 56 [Time Frame: Cohort 2 and 3: Day 28, Day 56]

Secondary ID(s)

2010-020413-90

VX09-809-102

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 05/10/2015
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01225211