for people ages 3-21 (full criteria)
at San Francisco, California and other locations
study started
completion around



The trial of IV arginine therapy in children with Vaso-occlusive painful episodes (VOE) in sickle cell disease (SCD) is designed to further knowledge on efficacy and safety of the therapy.


The trial is designed as a double-blind, placebo controlled, randomized, phase 3, multi-center trial of IV arginine therapy in children with Vaso-occlusive painful episodes (VOE) in sickle cell disease (SCD) to further knowledge on efficacy and safety of the therapy. The exploratory objective is to more fully characterize the arginine metabolome in children with SCD during VOE, and evaluate the effects of arginine therapy on global arginine bioavailability and mitochondrial function together with important clinical outcomes (time to VOE resolution, pain scores, total parenteral opioid use, Patient-Reported Outcomes (PROs), and hospital length of stay in children with SCD and VOE.


Sickle Cell Disease, Arginine Therapy, Pain management, Sickle Cell Anemia, Arginine Hydrochloride, Normal saline


You can join if…

Open to people ages 3-21

  1. Age 3-21 years of age, inclusive; AND
  2. Established diagnosis of sickle cell disease (any genotype); AND
  3. Pain requiring medical care in an acute care setting (ED, hospital ward, day hospital, clinic) not attributable to non-sickle cell causes, treated with parenteral opioids.

You CAN'T join if...

  1. Responds to 2 doses of IV opioids sufficiently for outpatient management
  2. Greater than 12 hours from first dose of intravenous opioids to treat current pain in acute care setting
  3. Hemoglobin less than 5 gm/dL or emergent need for red blood cell transfusion for hemodynamically unstable patient; OR
  4. Ketamine use in the emergency department for treatment of VOE; OR
  5. Glutamine within 30 days; OR
  6. New SCD drug use < 3 months (e.g. Hydroxyurea, voxelotor, crizanlizumab, etc) OR
  7. Acute mental status or neurological changes; OR
  8. Acute stroke or clinical concern for stroke; OR
  9. Three or more ED visits for sickle cell related pain receiving parenteral opioids in previous 7 days (not including current ED visit); OR

    10. Hospital discharge within previous 7 days; OR 11. Hypotension requiring clinical intervention; hemodynamic instability; septic shock; OR 12. Previous randomization in this arginine phase 3 RCT; OR 13. Use of inhaled nitric oxide, sildenafil or arginine within the last month; OR 14. Non-English or non-Spanish speaking; OR 15. pregnancy; OR 16. Allergy to arginine; OR 17. PI/clinical team concerns for compliance/issues that may adversely impact study

    participation/outcome; OR

    18. Adults 18 years or older who lack medical decision-making capacity to consent


  • UCSF Benioff Children's Hospital accepting new patients
    San Francisco California 94158 United States
  • Children's Hospital Los Angeles accepting new patients
    Los Angeles California 90027 United States


accepting new patients
Start Date
Completion Date
Claudia R. Morris
Phase 3 research study
Study Type
Expecting 360 study participants
Last Updated