Arginine Therapy for Pain in Children with Sickle Cell Disease

This study is looking at whether giving extra arginine, a natural amino acid, can help children with sickle cell disease who are experiencing pain crises (vaso-occlusive painful events or VOE). Patients with sickle cell disease often have low levels of arginine, which might be linked to their pain. Researchers want to see if increasing arginine levels through intravenous (IV) infusions can reduce pain, lessen the need for pain medications like morphine, or shorten hospital stays. You might be able to join if you are 7 to 21 years old, have a specific type of sickle cell disease (Hb-SS or Sβᴼ-thalassemia), weigh at least 55 pounds, and are experiencing pain that requires medical care. The study's current status is unclear.

Study design
This is an interventional study with a planned enrollment of 21 participants. It is not specified if the study is randomized or blinded.
What's involved
You would receive arginine intravenously (in the vein) either three times a day for up to seven days, as an initial dose followed by regular doses, or as a continuous IV infusion. The study will measure your plasma arginine concentration and nitric oxide levels over an average of up to 7 days.
Compensation
Not stated in the trial record.
Follow-up
Your plasma arginine concentration and nitric oxide metabolites will be measured from Day 1 through study completion, which is an average of up to 7 days.

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NCT02447874

Arginine Therapy for the Treatment of Pain in Children With Sickle Cell Disease

Recruiting
PHASE1Ages 7–21InterventionalTreatment
Emory University
~21 participants
Updated 2026-07-29 on ClinicalTrials.gov
What's tested:ArginineArginine (Loading)Arginine (Continuous)

At a glance

Recruiting sites
2 of 2 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Pharmacokinetics of IV arginine, measured by plasma arginine concentration over time
Measured over Day 1 through study completion, an average of up to 7 days
+1 more outcome measured
Sickle Cell Disease
2 sites across 1 states
Georgia2
  • Claudia Morris, MD · PRINCIPAL_INVESTIGATOR · Emory University

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Eligibility criteria

Inclusion

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.

Exclusion

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
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
Use of inhaled nitric oxide, sildenafil or arginine within the last 14 days
Not an appropriate candidate in the investigator's judgement
  • Pharmacokinetics of IV arginine, measured by plasma arginine concentration over timeDay 1 through study completion, an average of up to 7 days

    Total time plasma arginine levels are maintained above the half-saturating concentration (Km) of cationic amino acid transporter protein-1 (CAT-1), which is 150 µM (normal range of extracellular plasma arginine concentration). pK samples will be collected at 6 time-points within 8 hours: prior to arginine treatment (time 0), and at 60, 90, 120 minutes, 4 and 8 hours after the initiation of arginine therapy, and then every 24 hours up to 7 days.

  • Change in nitric oxide metabolitesBaseline, day 1 through study completion, an average of up to 7 days

    The formation of NO metabolites will be measured by determination of its stable end products in serum; nitrite (NO2-) and nitrate (NO3-). Change in nitric oxide metabolites will be calculated as the difference in metabolites from the time prior to arginine treatment (baseline) to the end of the intervention period.