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

The recruitment status of this study is unknown because the information has not been verified recently.

Verified January 2002 by FDA Office of Orphan Products Development. Recruitment status was Recruiting

First Received on January 21, 2002. Last Updated on June 23, 2005 History of Changes

Sponsor:	FDA Office of Orphan Products Development
Information provided by:	FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:	NCT00029731

Purpose

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.

Condition	Intervention	Phase
Anemia, Sickle Cell Pneumonia	Drug: Arginine hydrochloride	Phase II

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized Endpoint Classification: Efficacy Study Intervention Model: Parallel Assignment Primary Purpose: Treatment
Official Title:	Arginine Therapy for Acute Chest Syndrome in Sickle Cell Disease

Resource links provided by NLM:

Genetics Home Reference related topics: sickle cell disease

MedlinePlus related topics: Anemia Pneumonia Sickle Cell Anemia

Drug Information available for: Arginine Arginine hydrochloride

U.S. FDA Resources

Further study details as provided by FDA Office of Orphan Products Development:

Estimated Enrollment:	30
Study Start Date:	August 2001
Estimated Study Completion Date:	July 2004

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.

Eligibility

Ages Eligible for Study:	5 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00029731

Locations

United States, California
Children's Hospital Oakland	Recruiting
Oakland, California, United States, 94609
Contact: Lori A. Styles, M.D. 510-428-3553 lstyles@mail.cho.org
Principal Investigator: Lori A. Styles, M.D.

Sponsors and Collaborators

FDA Office of Orphan Products Development

More Information

No publications provided

ClinicalTrials.gov Identifier:	NCT00029731 History of Changes
Other Study ID Numbers:	FD-R-1978-01, FD-R-001978-01
Study First Received:	January 21, 2002
Last Updated:	June 23, 2005
Health Authority:	United States: Food and Drug Administration

Keywords provided by FDA Office of Orphan Products Development:

Nitric Oxide
Administration, Oral

Additional relevant MeSH terms:

Anemia
Anemia, Sickle Cell
Pneumonia
Acute Chest Syndrome
Hematologic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic

Hemoglobinopathies
Genetic Diseases, Inborn
Lung Diseases
Respiratory Tract Diseases
Respiratory Tract Infections
Respiration Disorders

ClinicalTrials.gov processed this record on February 12, 2012