---
title: Arginine Therapy for Sickle Cell Disease Pain
nct_id: NCT02536170
overall_status: COMPLETED
phase: PHASE2
sponsor: Emory University
study_type: INTERVENTIONAL
primary_condition: Sickle Cell Disease
countries: United States
canonical_url: "https://parkinsonspathways.com/agent/trials/NCT02536170.md"
clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT02536170"
ct_last_update_post_date: 2023-09-06
last_seen_at: "2026-05-12T07:04:53.185Z"
source: ClinicalTrials.gov (mirrored, no enrichment)
---
# Arginine Therapy for Sickle Cell Disease Pain

**Official Title:** Phase 2 Randomized Control Trial of Arginine Therapy for Pediatric Sickle Cell Disease Pain

**NCT ID:** [NCT02536170](https://clinicaltrials.gov/study/NCT02536170)

## Key Facts

- **Status:** COMPLETED
- **Phase:** PHASE2
- **Study Type:** INTERVENTIONAL
- **Target Enrollment:** 108
- **Lead Sponsor:** Emory University
- **Collaborators:** Children's Healthcare of Atlanta, National Center for Complementary and Integrative Health (NCCIH)
- **Conditions:** Sickle Cell Disease, Vaso-occlusive Pain Episode
- **Start Date:** 2016-02
- **Completion Date:** 2021-02-21
- **CT.gov Last Update:** 2023-09-06

## Brief Summary

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.

## Detailed Description

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.

## Eligibility

- **Minimum age:** 3 Years
- **Maximum age:** 21 Years
- **Sex:** ALL
- **Healthy Volunteers:** No

```
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
```

## Arms

- **L-Arginine** (EXPERIMENTAL) — Participants will be randomized to receive an intravenous (IV) infusion of L-arginine (100 mg/kg) three times a day until time of discharge from the emergency department (ED) or hospital.
- **Loading Dose and L-Arginine** (EXPERIMENTAL) — Participants will be randomized to receive an intravenous (IV) infusion of one-time loading dose of L-arginine (200 mg/kg) followed by standard dose (100 mg/kg) three times a day until time of discharge from the emergency department (ED) or hospital.
- **Placebo** (PLACEBO_COMPARATOR) — Participants will be randomized to receive an intravenous (IV) infusion of placebo (normal saline 1-2 ml/kg) three times a day until time of discharge from the emergency department (ED) or hospital.

## Interventions

- **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.
- **L-arginine Loading Dose** (DRUG) — One loading dose of L-arginine will be dispensed intravenously (IV) at 200 mg/kg
- **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

- **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

- **Length of Hospital Stay** _(time frame: Discharge (Up to 8 days))_
- **Time to Vaso-occlusive Pain Event (VOE) Resolution in Emergency Department** _(time frame: Post study drug delivery (Up to 8 hours))_
- **Time to Vaso-occlusive Pain Event (VOE) Resolution in Hospital** _(time frame: Post study drug delivery until discharge (up to 8 days))_
- **Change in Vaso-occlusive Pain (VOE) Scores** _(time frame: Baseline, Time of discharge (Up to 8 days))_
- **Length of Emergency Department (ED) Stay** _(time frame: Until discharge or Hospital Admission (Up to 24 hours))_
- **Rate of Emergency Department (ED) Discharge** _(time frame: Post emergency department admission (Up to 24 hours))_
- **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 Number of Study Drug Doses** _(time frame: Duration of study (Up to 8 days))_
- **Rate of Acute Chest Syndrome** _(time frame: Duration of study (Up to 8 days))_
- **Rate of Blood Transfusion** _(time frame: Duration of study (Up to 8 days))_
- **Oxygen Saturation Level** _(time frame: At time of Emergency Department Admission)_
- **Oxygen Saturation Level** _(time frame: At time of hospital admission and at time of Hospital discharge (Up to 8 days))_
- **Rate of Return Visits to Emergency Department (ED) Within 72 Hours** _(time frame: Post hospital discharge (within 72 hours))_
- **Rate of Hospital Re-admissions Within 72 Hours** _(time frame: Post hospital discharge (within 72 hours))_
- **Rate of Return Visits to Emergency Department (ED) Within 30 Days** _(time frame: Post hospital discharge (within 30 days))_
- **Rate of Hospital Re-admissions With 30 Days** _(time frame: Post hospital discharge (within 30 days))_

## 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

## Recent Field Changes (last 30 days)

- `status.overallStatus` — added _(2026-05-12)_
- `status.primaryCompletionDate` — added _(2026-05-12)_
- `status.completionDate` — added _(2026-05-12)_
- `status.lastUpdatePostDate` — added _(2026-05-12)_
- `design.phases` — added _(2026-05-12)_
- `design.enrollmentCount` — added _(2026-05-12)_
- `eligibility.criteria` — added _(2026-05-12)_
- `eligibility.minAge` — added _(2026-05-12)_
- `eligibility.maxAge` — added _(2026-05-12)_
- `eligibility.sex` — added _(2026-05-12)_
- `outcomes.primary` — added _(2026-05-12)_
- `outcomes.secondary` — added _(2026-05-12)_
- `armsInterventions.arms` — added _(2026-05-12)_
- `armsInterventions.interventions` — added _(2026-05-12)_
- `sponsor.lead` — added _(2026-05-12)_
- `sponsor.collaborators` — added _(2026-05-12)_
- `results.hasResults` — added _(2026-05-12)_
- `locations.children's healthcare of atlanta at hugh spalding|atlanta|georgia|united states` — added _(2026-05-12)_
- `locations.children's healthcare of atlanta at egleston|atlanta|georgia|united states` — added _(2026-05-12)_
- `locations.children's healthcare of atlanta at scottish rite|atlanta|georgia|united states` — added _(2026-05-12)_

---

*Canonical: https://parkinsonspathways.com/agent/trials/NCT02536170.md*  
*Source data (authoritative): https://clinicaltrials.gov/study/NCT02536170*  
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