L-Arginine and Sickle Cell Disease

Sickle Cell Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01142219
• Other study ID #: 04-487 cep@hcpa.ufrgs.br
• Status: Completed
• Phase: Phase 3
• First received: June 10, 2010
• Last updated: June 10, 2010
• Start date: September 2006
• Est. completion date: October 2009

Study information

• Verified date: June 2010
• Source: Hospital de Clinicas de Porto Alegre
• Contact: n/a
• Is FDA regulated: No
• Health authority: Brazil: Ethics Committee
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 40
• Est. completion date: October 2009
• Est. primary completion date: January 2008
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 1 Year and older

Eligibility

Inclusion Criteria:

• Diagnosis of sickle cell disease confirmed through hemoglobin electrophoresis

• Genotypes SS, SC and Sß 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

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

Intervention

Drug:
• L-arginine
L-arginine was administered orally at a dose of 0.1g/kg/day during 6 months.
• Placebo
Mannitol was administered orally at a dose of 01 g/kg/day for six months.

Locations

Country	Name	City	State
Brazil	Hospital de Clínicas de Porto Alegre	Porto Alegre	RS

Sponsors (4)

Lead Sponsor	Collaborator
Hospital de Clinicas de Porto Alegre	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)

Country where clinical trial is conducted

Brazil, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Tricuspid regurgitant jet velocity >2.5 m/s	Tricuspid regurgitant jet velocity was used to assess pulmonary arterial hypertension before and after treatment with L-arginine.	6 months	No
Secondary	Lactate dehydrogenase levels	The effect of L-argigine administration on hemolysis was assessed through the variation of serum levels of lactate dehydrogenase.	6 months	No