The Short Term Safety and Efficacy of Inhaled L-arginine in Patients With Cystic Fibrosis

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00405665
• Date of registration: 28/11/2006
• Prospective Registration: No
• Primary sponsor: The Hospital for Sick Children
• Public title: The Short Term Safety and Efficacy of Inhaled L-arginine in Patients With Cystic Fibrosis
• Scientific title: Pilot Study of the Short Term Safety and Efficacy of Inhaled L-arginine in Patients With Cystic Fibrosis
• Date of first enrolment: November 2006
• Target sample size: 20
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00405665
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

Canada

Contacts

• Name: Felix Ratjen, MD
• Affiliation: The Hospital for Sick Children, Toronto Canada

Key inclusion & exclusion criteria

Inclusion Criteria:

- Diagnosis of CF as defined by two or more clinical features of CF and a documented
sweat chloride concentration > 60 mEq/L and/or two well characterized disease causing
CFTR gene mutations

- 14 years of age and older at enrollment

- Clinically stable at enrollment

- Ability to comply with medication use, study visits and study procedures

- FEV1 % predicted > 40% < 80 % as calculated by reference equations

Exclusion Criteria:

- Respiratory culture positive for: B. cepacia complex within past year or at screening

- Use of systemic corticosteroids within 30 days of screening

- Use of intravenous antibiotics or oral quinolones within 14 days of screening

- History of biliary cirrhosis, portal hypertension, or splenomegaly

- Other major organ dysfunction

- History of lung transplantation or currently on lung transplant list

- Supplemental oxygen therapy

- Oxygen saturation < 95 % on room air

- Positive pregnancy test at screening

- Investigational drug use within 30 days of screening

- History of alcohol, illicit drug or medication abuse within 1 year of screening

- Acute respiratory symptoms

- Inability to take any form of bronchodilator

- Wheezing at the time of study



Age minimum: 14 Years
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: L-arginine

Primary Outcome(s)

Change in FEV1 (in liters) from baseline [Time Frame: At the end of the 14 day treatment period]

Adverse events such as gastrointestinal complaints, wheezing, hepatitis or shortness of breath [Time Frame: 70 weeks]

Secondary Outcome(s)

Change in FVC and change in FEV25-75 from baseline to completion of the 2 week treatment period. [Time Frame: Will be measured at the end of the 14 day treatment period]

Changes in inflammatory markers in sputum from baseline including neutrophils (sputum), neutrophil elastase (sputum) and interleukin (IL)-8 concentrations (sputum). [Time Frame: Will me measured at the end of the 14 day treatment period]

Change in exhaled nitric oxide (FeNO) [Time Frame: 70 days]

Changes in sputum concentrations of L-arginine metabolites [Time Frame: 70 days]

Secondary ID(s)

1000009282

Source(s) of Monetary Support

Please refer to primary and secondary sponsors