Arginine treatment for pain relief in children with sickle cell disease

Arginine Therapy for the Treatment of Vaso-Occlusive Events in Children With Severe Sickle Cell Disease

Phase1; Phase2 Interventional Emory University · NCT02447874

This study is testing if giving arginine to children with sickle cell disease can help reduce their pain during painful episodes and lessen their need for pain medications.

Quick facts

PhasePhase1; Phase2
Study typeInterventional
Enrollment21 (estimated)
Ages7 Years to 21 Years
SexAll
SponsorEmory University Academic / other
Locations2 sites (Atlanta, Georgia and 1 other locations)
Trial IDNCT02447874 on ClinicalTrials.gov

What this trial studies

This study investigates the effects of arginine supplementation on pain management in children with sickle cell disease experiencing vaso-occlusive events. It aims to determine if increasing arginine levels can reduce pain scores, decrease reliance on pain medications, and shorten hospital stays or emergency visits. The study includes a loading dose and continuous infusion of arginine to enhance its therapeutic effects. Participants will be monitored for pain relief and overall health outcomes during treatment.

Who should consider this trial

Good fit: Ideal candidates are children aged 7-21 years with a diagnosis of sickle cell disease requiring acute pain management.

Not a fit: Patients with other types of sickle cell disease or those not requiring acute medical care may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve pain management and quality of life for children with sickle cell disease.

How similar studies have performed: Previous studies have indicated that arginine may have beneficial effects in pediatric sickle cell patients, suggesting potential for success in this approach.

Eligibility criteria

Show full inclusion / exclusion criteria
Inclusion Criteria:

* 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 Criteria:

* 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

Where this trial is running

Atlanta, Georgia and 1 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Sickle Cell Disease
Last reviewed 2026-06-10 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.