Study of the Effect of VX-770 on Hyperpolarized Helium-3 Magnetic Resonance Imaging in Subjects With Cystic Fibrosis and the G551D Mutation

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT01161537
• Date of registration: 09/07/2010
• Prospective Registration: Yes
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: Study of the Effect of VX-770 on Hyperpolarized Helium-3 Magnetic Resonance Imaging in Subjects With Cystic Fibrosis and the G551D Mutation
• Scientific title: A Phase 2, Single-Blind, Placebo-Controlled Study to Evaluate the Effect of VX-770 on Hyperpolarized Helium-3 Magnetic Resonance Imaging in Subjects With Cystic Fibrosis, the G551D Mutation, and FEV1 =40% Predicted
• Date of first enrolment: October 2010
• Target sample size: 13
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01161537
• Study type: Interventional
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Talissa Altes, MD
• Affiliation: University of Virginia

Key inclusion & exclusion criteria

Inclusion Criteria:

- Male or female with Cystic Fibrosis

- Must have the G551D-CFTR mutation on at least 1 allele

- FEV1 =40% of predicted normal for age, gender, and height at Screening

- 12 years of age or older

- Must be able to swallow tablets

Exclusion Criteria:

- History of solid organ or hematological transplantation

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

- Use of inhaled hypertonic saline treatment within 14 days prior to the Screening Visit

- Extensive body tattoos or other physical features that will confound MRI



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

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: Placebo

Drug: VX-770

Primary Outcome(s)

Part B: Change From Baseline in Total Ventilation Defect Defined by Hyperpolarized Helium 3 Magnetic Resonance Imaging (3He-MRI) at Week 48 [Time Frame: Part B: Baseline (Day -1), Week 48]

Part A: Change From Baseline in Total Ventilation Defect Defined by Hyperpolarized Helium 3 Magnetic Resonance Imaging (3He-MRI) at Day 43 [Time Frame: Part A: Baseline (pre-dose Day 15), Day 43]

Secondary Outcome(s)

Part B: Absolute Change From Baseline in in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score At Week 48 [Time Frame: Part B: Baseline (Day -1), Week 48]

Part A: Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score At Day 43 [Time Frame: Baseline (pre-dose Day 15), Day 43]

Part A: Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Day 43 [Time Frame: Part A: Baseline (pre-dose Day 15), Day 43]

Part A: Absolute Change From Baseline in Sweat Chloride at Day 43 [Time Frame: Part A: Baseline (pre-dose Day 15), Day 43]

Part B: Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 48 [Time Frame: Part B: Baseline (Day -1), Week 48]

Part A: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Related AEs [Time Frame: Part A: Day 1 up to Day 57]

Part B: Absolute Change From Baseline in Sweat Chloride at Week 48 [Time Frame: Part B: Baseline (Day -1), Week 48]

Part B: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Related AEs [Time Frame: Part B: Day 1 up to Week 48]

Secondary ID(s)

VX10-770-107

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: 31/07/2014
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01161537