Status:

COMPLETED

Longitudinal Assessment of Exercise Capacity and Vascular Function in Patients With CF

Lead Sponsor:

Augusta University

Collaborating Sponsors:

Vertex Pharmaceuticals Incorporated

Conditions:

Cystic Fibrosis

Eligibility:

All Genders

7+ years

Brief Summary

This project is an attempt to understand how Orkambi treatment affects exercise capacity and the function of the arteries in CF patients who are homozygous F508del. Our goal is to perform the exercise...

Detailed Description

Cystic Fibrosis (CF) is the most common fatal genetic disease in North America. The most disturbing aspect of CF is the associated premature death, most often due to respiratory complications. Clinica...

Eligibility Criteria

Inclusion

  • Patients diagnosed with Cystic Fibrosis (homozygous deltaF508del)
  • Prescribed Orkambi
  • Men and women (\> 18 yrs. old)
  • Boys and girls (7 -17 yrs. old)
  • FEV1 percent predicted \> 40%
  • Resting oxygen saturation (room air) \>85%
  • Patients with or without CFRD
  • Traditional CF-treatment medications
  • Clinically stable for past 28 days (no exacerbations or change in medical status)
  • Healthy Controls

Exclusion

  • Children 6 yrs. old and younger
  • FEV1 percent predicted \< 40%
  • Resting oxygen saturation (room air) \< 85%
  • Clinical diagnosis of heart disease
  • Pulmonary artery hypertension
  • Febrile illness within two weeks of visit
  • Current smokers
  • Currently pregnant or nursing
  • Individuals on vaso-active medications (i.e. nitrates, beta blockers, ACE inhibitors, etc.)
  • Use of VX-770 within 6 months prior to Visit 1
  • History of solid organ transplantation
  • Presence of a condition or abnormality that in the opinion of the investigator would compromise the safety of the patient or the quality of the data.

Key Trial Info

Start Date :

May 1 2014

Trial Type :

OBSERVATIONAL

Allocation :

ACTUAL

End Date :

October 1 2020

Estimated Enrollment :

20 Patients enrolled

Trial Details

Trial ID

NCT03338595

Start Date

May 1 2014

End Date

October 1 2020

Last Update

January 19 2021

Active Locations (1)

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1

Georgia Prevention Institute

Augusta, Georgia, United States, 30912