Phase 2 Randomized Control Trial of Arginine Therapy for Pediatric Sickle Cell Disease Pain

Study point of contact

Reshka Mendis, MBBS
352 277-7781
[email protected]
Claudia Morris, MD
404-727-5500
[email protected]

Locations

3 United States sites

Age

3 to 21 Years

Phase

Phase 2

Study type

Interventional

Gender

All

Interventions

Drug

Other

Compensation

Unknown

About the study

The aim of this study is to determine whether giving extra arginine, a simple amino acid, to
patients with sickle cell disease seeking treatment for a pain crisis (vaso-occlusive painful
events (VOE) will decrease pain scores, decrease the need for pain medications or decrease
length of hospital stay or emergency department visit. Funding Source – FDA OOPD.

participation requirements

– Established diagnosis of sickle cell disease (SCD); all genotypes

– Pain requiring medical care in an acute care setting (such as the emergency department
or ED, hospital ward, day hospital, clinic) not attributable to non-sickle cell
causes, that is moderate-to-severe requiring parenteral opioids

participation restrictions

– Decision to discharge home from the acute care setting

– Hemoglobin less than 5 gm/dL or immediate need for red cell transfusion anticipated
within next 12 hours

– Hepatic dysfunction of SGPT greater than 3 times the upper value

– Renal dysfunction of creatinine greater than 1.0

– Mental status or neurological changes

– Acute stroke or clinical concern for stroke

– Pregnancy

– Allergy to arginine

– Two (2) or more ED visits for VOE within the last 7 days prior to CURRENT ED visit

– Hospitalization within 14 days

– Previous randomization in this arginine RCT (patient consented and screen failed
before receiving study drug or placebo remains eligible for future participation).

– Use of inhaled nitric oxide, sildenafil or arginine within the last month

– PICU admission from the emergency department

– Hypotension requiring treatment with clinical intervention

– Acidosis with Co2≤ 16

– Newly started on HU for <3 months - Not an appropriate candidate in the investigator's judgment - Patient refusal

Locations

  • Atlanta, Georgia, United States, Children's Healthcare of Atlanta at Hugh Spalding, 30303 [Recruiting]
  • Atlanta, Georgia, United States, Children's Healthcare of Atlanta at Egleston, 30322 [Recruiting]
  • Atlanta, Georgia, United States, Children's Healthcare of Atlanta at Scottish Rite, 30342 [Not yet recruiting]