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NCT Number: NCT01772758

Cystic Fibrosis and Endothelial Function: At Rest and During Exercise

Perhaps one of the most disturbing aspects of Cystic Fibrosis (CF) is the associated premature death. Oxidative stress has been observed in patients with CF and exercise intolerance has been shown to predict mortality in patients with CF, regardless of how healthy their lungs are. A critical barrier to improving the quality of life and longevity in patients with CF is our lack of knowledge regarding the different reasons why patients with CF cannot exercise to the level of their peers. We have collected preliminary data to support our central hypothesis that oxidative stress contributes to the impairment in blood vessel function at rest and during exercise which ultimately oxygen transport and delivery resulting in exercise intolerance. Exercise is therapeutic medicine for patients with CF and this investigation represents a major breakthrough in the approach to begin understanding the physiological mechanisms which contribute to exercise intolerance in these patients.

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Key information

Conditions

Cystic Fibrosis Behavior Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Motor Activity Pancreatic Diseases Respiratory Tract Diseases

Age range

7 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Georgia Prevention Institute/ Laboratory of Integrative and Exercise Physiology

Augusta, Georgia, 30912, United States

About this study

The overall goals of this proposal are to provide mechanistic evidence that oxidative stress contributes to 1) endothelial dysfunction and 2) exercise intolerance in patients with CF. This study consists of two separate sub-studies, or protocols. Protocol 1: AOC tested the effect of an antioxidant cocktail (AOC) on endothelial function at rest and during exercise in CF patients. Protocol 2: BH4 tested the effect of tetrahydrobiopterin (BH4) on endothelial function at rest and during exercise in CF patients.

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Diagnosis of CF and healthy controls
  • Men and women (> 18 yrs. old)
  • Boys and girls (7 -17 yrs. old)
  • FEV1 percent predicted > 30%
  • Resting oxygen saturation (room air) >90%
  • Patients with or without CFRD
  • Traditional CF-treatment medications
  • Ability to perform reliable/reproducible PFTs
  • Clinically stable for 2 weeks (no exacerbations or need for antibiotic treatment within 2 weeks of testing or major change in medical status)

Exclusion criteria

  • Children 6 yrs. old and younger
  • FEV1 percent predicted < 30%
  • Resting oxygen saturation (room air) < 90%
  • 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.)
  • Inability to swallow pills
  • Patients with B. Cepacia (only ~3% of our CF center patient population)

Treatment and study plan

BH4 5mg

Drug

Kuvan® or sapropterin dihydrochloride is a synthetic preparation of the dihydrochloride salt of naturally occurring tetrahydrobiopterin (BH4). Subjects received an oral dose of 5 mg/kg of Kuvan® (Biomarin Pharmaceuticals Inc.)

Other names: Tetrahydrobiopterin, Kuvan

BH4 20mg

Drug

Kuvan® or sapropterin dihydrochloride is a synthetic preparation of the dihydrochloride salt of naturally occurring tetrahydrobiopterin (BH4). Subjects received an oral dose of 20 mg/kg of Kuvan® (Biomarin Pharmaceuticals Inc.)

Other names: Tetrahydrobiopterin, Kuvan

Antioxidant cocktail

Dietary Supplement

Vitamin C (1000 mg) , Vitamin E (600 IU) , and alpha-lipoic acid (600 mg). all BID

Primary outcomes

  1. Percentage Flow-Mediated Dilation (FMD)

    Time frame: pre-treatment Baseline and 2-3 hours post-treatment

    Brachial artery FMD induced by reactive hyperemia assessed vascular endothelial function at baseline and several hours after treatment.

Sponsors and collaborators

Lead sponsor

Augusta University

Other

Registry information

Official study title

Influence of Cystic Fibrosis on Vascular Endothelial Function at Rest and During Exercise

Important dates

Study start
2011
Primary completion
2016
Study completion
2016
First posted
Jan 21, 2013
Registry last updated
Jun 20, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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