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

About the study

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.

Study point of contact

Reshika Mendis, MBBS
[email protected]
Claudia Morris, MD
404 727-5500
[email protected]


7 Years - 21 Years


Hemoglobin SS


Phase 1/Phase 2

Study type






participation requirements

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.

participation restrictions

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
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

Last updated 2022-04-22