Study of the Effect of VX-770 on Hyperpolarized Helium-3 MagneticResonance Imaging in Subjects With Cystic Fibrosis and the G551DMutatio

Phase 1

• Conditions: cystic fibrosis; MedDRA version: 19.1Level: SOCClassification code 10010331Term: Congenital, familial and genetic disordersSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2017-000672-28-Outside-EU/EEA
• Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2017-03-27
• Last Update Posted: 3 April 2017

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: 17

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
Are the trial subjects under 18? yes
Number of subjects for this age range: 4
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 13
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified