L-Arginine and Sickle Cell Disease

Phase 3

Completed

• Conditions: Sickle Cell Disease
• Interventions: Drug: L-arginine; Drug: Placebo

• Registration Number: NCT01142219
• Lead Sponsor: Hospital de Clinicas de Porto Alegre

Brief Summary

One of the main problems in sickle cell disease is the decreased bioavailability of nitric oxide and arginine. This study was designed to assess if treating sickle cell disease patients with L-arginine would improve pulmonary arterial pressure and other aspects.

Detailed Description

This is a phase III, randomized, double-blind, placebo-controlled clinical trial with sickle cell disease patients older than 1 year of age. The patients were randomly assigned to take 0.1 g/kg/day of either L-arginine or placebo orally. Adverse events were monitored by a safety committee. The variables were assessed while patients were in remission, as part of their routine care: weight, blood pressure, full blood cell count, creatinine and dosage of methemoglobin at baseline and at each follow-up visit. Peripheral oxygen saturation (SpO2), fetal hemoglobin, lactate dehydrogenase and tricuspid regurgitant jet velocity were measured through transthoracic Doppler echocardiogram before and after treatment.

Study Timeline

• Study Start: 2006-09
• Primary Completion: 2008-01
• Study Completion: 2009-10
• Status Verified: 2010-06
• Study First Submitted: 2010-06-10
• Study First Submitted QC: 2010-06-10
• Last Update Submitted: 2010-06-10
• Study First Posted: 2010-06-11
• Last Update Posted: 2010-06-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

• Diagnosis of sickle cell disease confirmed through hemoglobin electrophoresis
• Genotypes SS, SC and Sβ thalassemia
• Age > 1 year

Exclusion Criteria

• Liver dysfunction (alanine aminotransferase ALT > 3 times the normal levels
• Renal dysfunction (creatinine > twice the normal levels)
• Increase in methemoglobin levels (> 5 times the normal levels)
• History of recent stroke (< 1 month) and priapism
• Pregnancy
• Allergy to L-arginine
• Use of sildenafil, calcium channel blockers, nitroglycerin or other nitrates

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
L-arginine	Placebo	0.1g/kg/day for 6 months
L-arginine	L-arginine	0.1g/kg/day for 6 months

Primary Outcome Measures

Name	Time	Method
Tricuspid regurgitant jet velocity >2.5 m/s	6 months	Tricuspid regurgitant jet velocity was used to assess pulmonary arterial hypertension before and after treatment with L-arginine.

Secondary Outcome Measures

Name	Time	Method
Lactate dehydrogenase levels	6 months	The effect of L-argigine administration on hemolysis was assessed through the variation of serum levels of lactate dehydrogenase.

Trial Locations

Locations (1)

Hospital de Clínicas de Porto Alegre; 🇧🇷 Porto Alegre, RS, Brazil