Skip to main content
OpenTrials
Completed

NCT Number: NCT02536170

Arginine Therapy for Sickle Cell Disease Pain

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Sickle Cell Disease Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Vaso-occlusive Pain Episode

Age range

3 year–21 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Children's Healthcare of Atlanta at Egleston, Atlanta, Georgia, United States

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

L-arginine

Drug

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.

Other names: Arginine

L-arginine Loading Dose

Drug

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

Other names: Arginine

Placebo

Other

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

Primary outcomes

  1. Total Parenteral Opioid Use in IV Morphine Equivalents

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

    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.

Secondary outcomes

  1. Length of Hospital Stay

    Time frame: Discharge (Up to 8 days)

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

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

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

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

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

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

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

  4. Change in Vaso-occlusive Pain (VOE) Scores

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

    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).

  5. Length of Emergency Department (ED) Stay

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

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

  6. Rate of Emergency Department (ED) Discharge

    Time frame: Post emergency department admission (Up to 24 hours)

    Number of participants discharged from ED without a hospital ward admission.

  7. Total Opioid Dose (ORAL + Parenteral) in mg/kg IV Morphine Equivalents

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

    Total opioid dose (ORAL + Parenteral) in mg/kg IV morphine equivalents after study drug delivery up to hospital discharge (up to 8 days)

  8. Total Number of Study Drug Doses

    Time frame: Duration of study (Up to 8 days)

    The total number of study drug doses given throughout the study period.

  9. Rate of Acute Chest Syndrome

    Time frame: Duration of study (Up to 8 days)

    Number of participants who develop acute chest syndrome (not diagnosed prior to study drug delivery) throughout the study period.

  10. Rate of Blood Transfusion

    Time frame: Duration of study (Up to 8 days)

    Number of participants requiring a blood transfusion throughout the study period.

  11. Oxygen Saturation Level

    Time frame: At time of Emergency Department Admission

    Average oxygen saturation level of participants at time of ED arrival

  12. Oxygen Saturation Level

    Time frame: At time of hospital admission and at time of Hospital discharge (Up to 8 days)

    The difference in oxygen saturation levels from emergency department arrival to hospital discharge.

  13. Rate of Return Visits to Emergency Department (ED) Within 72 Hours

    Time frame: Post hospital discharge (within 72 hours)

    Number of ED visits from patients who have been discharged within the previous 72 hours.

  14. Rate of Hospital Re-admissions Within 72 Hours

    Time frame: Post hospital discharge (within 72 hours)

    Number of patients readmitted to the hospital within 72 hours of discharge.

  15. Rate of Return Visits to Emergency Department (ED) Within 30 Days

    Time frame: Post hospital discharge (within 30 days)

    Number of ED visits from patients who have been discharged within the previous 30 days.

  16. Rate of Hospital Re-admissions With 30 Days

    Time frame: Post hospital discharge (within 30 days)

    Number of patients readmitted to the hospital within 30 days of discharge.

Sponsors and collaborators

Lead sponsor

Emory University

Other

Collaborators

  • Children's Healthcare of Atlanta
  • National Center for Complementary and Integrative Health (NCCIH)

Registry information

Official study title

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

Important dates

Study start
2016
Primary completion
2021
Study completion
2021
First posted
Aug 31, 2015
Registry last updated
Sep 6, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.