Arginine Treatment of Acute Chest Syndrome (Pneumonia) in Sickle Cell Disease Patients

Arginine Therapy for Acute Chest Syndrome in Sickle Cell Disease

This is a study to determine if oral arginine will increase nitric oxide in sickle cell disease (SCD) patients with acute chest syndrome (ACS). It will also assess the effects of arginine in the body and how the body uses nitric oxide in ACS.

Study Overview

• Status: Completed
• Conditions: Pneumonia; Anemia, Sickle Cell
• Intervention / Treatment: Drug: Arginine hydrochloride

Detailed Description

Pneumonia in patients with SCD can be particularly severe and has come to be called acute chest syndrome. ACS is a common cause of morbidity in SCD patients and is the most common cause of death in SCD. Multiple factors are involved in the severity of acute pulmonary injury in SCD. Nitric oxide has a multitude of related functions, many of which could impact favorably on ACS in SCD. Nitric oxide is an important inflammatory mediator which is produced by the conversion of L-arginine to citrulline by nitric oxide synthase. Nitric oxide and its precursor, arginine, are known to be low in SCD patients with ACS, suggesting that therapies, such as arginine, aimed at increasing nitric oxide production will improve the clinical course of ACS. Patients will receive 1 of 3 doses of arginine hydrochloride orally 3 times a day for 3 days while hospitalized. The efficacy of arginine will be measured by the increase in nitric oxide production and the physiological effects will be assessed.

Completion date provided represents the completion date of the grant per OOPD records

• Study Type: Interventional
• Enrollment (Anticipated): 30
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Oakland, California, United States, 94609
      • Children's Hospital Oakland

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion criteria:

• Diagnosis of sickle cell disease (Hb SS, SC, or Sbeta thalassemia)

Acute chest syndrome with the following:

• New pulmonary infiltrate on chest radiography involving a full segment of the lung and 1 of the following:
• Fever
• Cough, tachypnea, retractions, rales, or wheezing
• Chest pain

Exclusion criteria:

• Inability to take or tolerate oral medications
• Hepatic dysfunction (SGPT greater than 2 times normal)
• Renal dysfunction (creatinine greater than 2 times normal)
• Mental status or neurological changes
• Allergy to arginine
• History of priapism
• Pregnancy

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment

Collaborators and Investigators

• Sponsor: UCSF Benioff Children's Hospital Oakland

Study record dates

Study Major Dates

• Study Start: August 1, 2001
• Study Completion: July 1, 2005

Study Registration Dates

• First Submitted: January 21, 2002
• First Submitted That Met QC Criteria: January 21, 2002
• First Posted (Estimate): January 22, 2002

Study Record Updates

• Last Update Posted (Estimate): March 25, 2015
• Last Update Submitted That Met QC Criteria: March 24, 2015
• Last Verified: January 1, 2002

More Information

Terms related to this study

• Keywords: Nitric Oxide; Administration, Oral
• Additional Relevant MeSH Terms: Infections; Respiratory Tract Infections; Respiratory Tract Diseases; Respiration Disorders; Lung Diseases; Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Pneumonia; Anemia, Sickle Cell; Acute Chest Syndrome
• Other Study ID Numbers: FD-R-1978-01; FD-R-001978-01