L-Arginine and Sickle Cell Disease

Hospital de Clinicas de Porto Alegre

Status and phase: Completed; Phase 3

Conditions: Sickle Cell Disease

Treatments: Drug: L-arginine; Drug: Placebo

Study type: Interventional

Funder types: Other

Identifiers

NCT01142219

04-487 [e-mail]

Details and patient eligibility

About

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.

Full 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.

Enrollment: 40 patients

Sex: All

Ages: 1+ year old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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40 participants in 1 patient group

L-arginine

Experimental group

Description:

0.1g/kg/day for 6 months

Treatment:

Drug: Placebo

Drug: L-arginine