RECRUITINGPhase 1 / Phase 2INTERVENTIONAL

Arginine Therapy for the Treatment of Pain in Children With Sickle Cell Disease

Arginine Therapy for the Treatment of Vaso-Occlusive Events in Children With Severe Sickle Cell Disease

About This Trial

The purpose of this study is to determine whether giving extra arginine to patients with sickle cell disease seeking treatment for vaso-occlusive painful events (VOE) will decrease pain scores, decrease need for pain medications or decrease length of hospital stay or emergency department visit.

Who May Be Eligible (Plain English)

Who May Qualify: - Established diagnosis of sickle cell disease--Hemoglobin SS (Hb-SS) or Sβᴼ-thalassemia - 7-21 years of age - Weight \>= 25kg (55lbs) - Pain requiring medical care in an acute care setting (emergency department (ED), hospital ward, day hospital, clinic) requiring parenteral opioids, not attributable to non-sickle cell causes. Who Should NOT Join This Trial: - Decision to discharge home from acute care setting. - Diagnosis of sickle cell disease with any of the following types: hemoglobin SC disease (HbSC), hemoglobin beta thalassemia (Hb-Beta Thal), hemoglobin SD disease (HbSD), hemoglobin SE disease (HbSE), hemoglobin SO disease (HbSO), hemoglobin AS carrier (Hb AS) - Hemoglobin less than 5 gm/dL - Immediate Red cell transfusion anticipated - Renal dysfunction: Creatinine \>1.0 or 2 x baseline - Mental status or neurological changes - Acute stroke or clinical concern for stroke - Pregnancy - Allergy to arginine - Previous hospitalization \< 7 days - Use of inhaled nitric oxide, sildenafil or arginine within the last 14 days - Not an appropriate candidate in the investigator's judgement Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language

Inclusion Criteria: * Established diagnosis of sickle cell disease--Hemoglobin SS (Hb-SS) or Sβᴼ-thalassemia * 7-21 years of age * Weight \>= 25kg (55lbs) * Pain requiring medical care in an acute care setting (emergency department (ED), hospital ward, day hospital, clinic) requiring parenteral opioids, not attributable to non-sickle cell causes. Exclusion Criteria: * Decision to discharge home from acute care setting. * Diagnosis of sickle cell disease with any of the following types: hemoglobin SC disease (HbSC), hemoglobin beta thalassemia (Hb-Beta Thal), hemoglobin SD disease (HbSD), hemoglobin SE disease (HbSE), hemoglobin SO disease (HbSO), hemoglobin AS carrier (Hb AS) * Hemoglobin less than 5 gm/dL * Immediate Red cell transfusion anticipated * Renal dysfunction: Creatinine \>1.0 or 2 x baseline * Mental status or neurological changes * Acute stroke or clinical concern for stroke * Pregnancy * Allergy to arginine * Previous hospitalization \< 7 days * Use of inhaled nitric oxide, sildenafil or arginine within the last 14 days * Not an appropriate candidate in the investigator's judgement

Treatments Being Tested

DRUG

Arginine

Arginine will be dispensed intravenously (in the vein) in the standard dose of arginine as 100 mg/kg three times a day for seven days or until discharge. * Loading dose: 200 mg/kg once * Continuous IV: 300 mg/kg/24 hours

DRUG

Arginine (Loading)

Arginine will be dispensed intravenously (in the vein) as an initial bolus (loading) at each specified group dose once, followed by a standard dose of 100mg/kg every 8 hours until discharge or for a total of 21 doses of arginine, whichever comes first.

DRUG

Arginine (Continuous)

Arginine will be dispensed intravenously (in the vein) as a continuous IV infusion of 300 mg/kg/24hr

Locations (2)

Children's Healthcare fo Atlanta at Hughes Spalding

Atlanta, Georgia, United States

Children's Healthcare of Atlanta at Arthur M. Blank Hospital

Atlanta, Georgia, United States