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
404-785-4525
[email protected]

Locations

2 United States sites

Age

7 to 21 Years

Genotypes

SS

Phase

Phase 1/Phase 2

Study type

Interventional

Gender

All

Interventions

Drug

Compensation

Unknown

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

Locations

  • 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]