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

Study point of contact

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


2 United States sites


7 to 21 Years


Hemoglobin SS


Phase 1/Phase 2

Study type








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.

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

– 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


  • Atlanta, Georgia, United States, Children's Healthcare fo Atlanta at Hughes Spalding, 30303 [Recruiting]
  • Atlanta, Georgia, United States, Children's Healthcare of Atlanta at Egleston, 30322 [Recruiting]
Last updated 2021-03-12 Enroll Now