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Completed

NCT Number: NCT01142219

L-Arginine and Sickle Cell Disease

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.

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Key information

Conditions

Sickle Cell Disease Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Hemolysis Hypertension, Pulmonary Lung Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Pulmonary Arterial Hypertension Respiratory Tract Diseases

Age range

1 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Hospital de Clínicas de Porto Alegre

Porto Alegre, Rio Grande do Sul, 90035-001, Brazil

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

L-arginine

Drug

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

Placebo

Drug

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

Primary outcomes

  1. Tricuspid regurgitant jet velocity >2.5 m/s

    Time frame: 6 months

    Tricuspid regurgitant jet velocity was used to assess pulmonary arterial hypertension before and after treatment with L-arginine.

Secondary outcomes

  1. Lactate dehydrogenase levels

    Time frame: 6 months

    The effect of L-argigine administration on hemolysis was assessed through the variation of serum levels of lactate dehydrogenase.

Sponsors and collaborators

Lead sponsor

Hospital de Clinicas de Porto Alegre

Other

Collaborators

  • Ajinomoto Interamericana Indústria e Comércio Ltda. (donation of L-arginine)
  • Associação de Amigos da Hematologia (HEMOAMIGOS)
  • Fundo de Incentivo à Pesquisa do Hospital de Clinicas de Porto Alegre (FIPE)

Registry information

Official study title

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

Important dates

Study start
2006
Primary completion
2008
Study completion
2009
First posted
Jun 11, 2010
Registry last updated
Jun 11, 2010

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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