Arginine Therapy for Sickle Cell Disease Pain

Emory University108 enrolled

Overview

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.

The purpose of this study is to determine the effects of IV L-arginine hydrochloride therapy in children with sickle cell disease (SCD) and vaso-occlusive pain events (VOE). Specifically, the impact on total opioid use (mg/kg) over the duration of their emergency department (ED) visit and hospital stay will be evaluated.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

108

Conditions

Sickle Cell Disease Vaso-occlusive Pain Episode

Interventions

L-arginineDRUG

L-arginine will be dispensed intravenously (IV) in the standard dose of 100 mg/kg three times a day until discharge from the emergency department (ED) or hospital.

L-arginine Loading DoseDRUG

One loading dose of L-arginine will be dispensed intravenously (IV) at 200 mg/kg

PlaceboOTHER

Placebo of intravenous (IV) normal saline 1-2 ml/kg three times a day until discharge from the emergency department (ED) or hospital.

Eligibility

Sex: ALLMin age: 3 YearsMax age: 21 Years

Medical Language ↔ Plain English

Inclusion Criteria: * 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 Exclusion Criteria: * 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 (3)

Children's Healthcare of Atlanta at Hugh Spalding

Atlanta, Georgia, United States

Children's Healthcare of Atlanta at Egleston

Atlanta, Georgia, United States

Children's Healthcare of Atlanta at Scottish Rite

Atlanta, Georgia, United States

Outcomes

Primary Outcomes

Total Parenteral Opioid Use in IV Morphine Equivalents

The total amount of parenteral opioids used by participants measured in mg/kg of IV morphine equivalents. The total is calculated after study drug delivery for participants in the emergency department (ED) and during hospital stay.

Time frame: Post study drug delivery to discharge from the hospital (Up to 8 days)

Secondary Outcomes

Length of Hospital Stay

The total number of hours spent in the hospital from study drug delivery to time of discharge.

Time frame: Discharge (Up to 8 days)

Time to Vaso-occlusive Pain Event (VOE) Resolution in Emergency Department

The total number of hours between study drug delivery and the last parenteral opioid.

Time frame: Post study drug delivery (Up to 8 hours)

Time to Vaso-occlusive Pain Event (VOE) Resolution in Hospital

The total number of hours between study drug delivery and time of last parenteral opioid use, pain relief improved to tolerate oral pain medications

Time frame: Post study drug delivery until discharge (up to 8 days)

Change in Vaso-occlusive Pain (VOE) Scores

Pain associated with VOE will be measured on a scale of 0-10, by asking subjects to rate their pain level on a subjective scale from 0 to 10, with the ends representing the extreme limits of "no-pain" (0) and "worst pain" (10).

Time frame: Baseline, Time of discharge (Up to 8 days)

Length of Emergency Department (ED) Stay

Total hours from time of ED triage to ED discharge or hospital admission.

Time frame: Until discharge or Hospital Admission (Up to 24 hours)

Data from ClinicalTrials.gov

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