Arginine Therapy for the Treatment of Pain in Children With Sickle Cell Disease (R34 pK/PD)
Sickle Cell Disease
About this trial
This is an interventional treatment trial for Sickle Cell Disease
Eligibility Criteria
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
- Hepatic dysfunction: serum glutamic pyruvic transaminase (SGPT) > 3X upper value
- 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
- Previous randomization in this arginine pK study (patient consented and screened failed before receiving study drug or placebo remains eligible for future participation)
- Use of inhaled nitric oxide, sildenafil or arginine within the last month
Sites / Locations
- Children's Healthcare fo Atlanta at Hughes SpaldingRecruiting
- Children's Healthcare of Atlanta at EglestonRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Experimental
Experimental
Experimental
Standard dose
Loading dose + standard dose
Loading dose + continuous infusion
Subjects with sickle cell disease (SCD) and vaso-occlusive painful events (VOE) will be randomized to receive an intravenous (IV) infusion of a standard dose of arginine (100 mg/kg) three times a day for seven days or until discharged from the hospital, whichever occurs first
Subjects with sickle cell disease and vaso-occlusive painful events (VOE) will be randomized to receive an intravenous (IV) infusion of an initial loading dose of arginine (200 mg/kg) given over 30 minutes and then receive an intravenous (IV) infusion of a standard dose of arginine (100 mg/kg) three times a day for seven days or until discharged from the hospital, whichever occurs first
Subjects with sickle cell disease and vaso-occlusive painful events (VOE) will be randomized to receive an intravenous (IV) infusion of an initial loading dose of arginine (200 mg/kg) given over 30 minutes and then receive a continuous intravenous (IV) infusion of 300 mg/kg/24hr for 7 days or until discharged from the hospital, whichever occurs first