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Cystic Fibrosis and Endothelial Function: At Rest and During Exercise

NCT01772758 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 64

Last updated 2019-06-20

Study results available
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Summary

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.

Conditions

Interventions

DRUG

BH4 5mg

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.)

DRUG

BH4 20mg

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.)

DIETARY_SUPPLEMENT

Antioxidant Cocktail

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

Sponsors & Collaborators

  • Augusta University

    lead OTHER

Principal Investigators

  • Ryan Harris, PhD, CES · Augusta University

Study Design

Allocation
NON_RANDOMIZED
Purpose
OTHER
Masking
DOUBLE
Model
PARALLEL

Eligibility

Min Age
7 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2011-08-31
Primary Completion
2016-06-21
Completion
2016-06-21

Countries

  • United States

Study Locations

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01772758 on ClinicalTrials.gov