L-Arginine and Sickle Cell Disease

A Randomized, Controlled, Double-blind Clinical Trial of L-arginine as Adjuvant Therapy for Sickle Cell Disease

Status

Completed

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01142219

Enrollment

Registered

2010-06-11

Start date

2006-09-30

Completion date

2009-10-31

Last updated

2010-06-11

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Sickle Cell Disease

Keywords

sickle cell disease, L-arginine, hemolysis, nitric oxide, pulmonary arterial hypertension

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.

Interventions

DRUGL-arginine

L-arginine was administered orally at a dose of 0.1g/kg/day during 6 months.

DRUGPlacebo

Mannitol was administered orally at a dose of 01 g/kg/day for six months.

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

TRIPLE (Subject, Caregiver, Investigator)

Eligibility

Sex/Gender

ALL

Age

1 Years to No maximum

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
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

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

Countries

Brazil

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026