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

NCT03338595 | Completed DMC

• 1 other identifier: CF-Long
• Study Type: observational
• Target: 20
• Locations: 1 country
• Sites: 1

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 and vascular measurements every 3 months after a patient starts taking Orkambi.

Conditions

Cystic Fibrosis

Keywords

arterial stiffness; flow-mediated dilation; endothelial function; Orkambi; exercise capacity

Outcome Measures

Primary Outcomes (1)

• Maximal exercise capacity

  Subjects will perform the maximal exercise tests on an electronically braked cycle ergometer using the Godfrey protocol. Expired gases will be collected using a Parvo Medics True One metabolic cart for determination of exercise capacity (VO2 peak).

  1 year

Secondary Outcomes (1)

• Flow mediated dilation

  1 year

Eligibility Criteria

• Age: 7 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

CF patients who are homozygous F508del and have been prescribed Orkambi.

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Augusta University: lead
• Vertex Pharmaceuticals Incorporated: collaborator

Study Sites (1)

Georgia Prevention Institute

Augusta, Georgia, 30912, United States

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

plasma samples

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Ryan Harris, PhD

  Augusta University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: March 31, 2017
• First Posted: November 9, 2017
• Study Start: May 1, 2014
• Primary Completion: October 1, 2020
• Study Completion: October 1, 2020
• Last Updated: January 19, 2021

Record last verified: 2021-01

Data Sharing

• IPD Sharing: Will share

Locations

US (1)