Sickle Cell Disease Treatment With Arginine Therapy (STArT) Trial

Completed · Phase 3 · Has a placebo group

Conditions studied: Sickle Cell Disease

In brief

The trial is designed to test intravenous (IV) arginine therapy in children with sickle cell disease (SCD) and vaso-occlusive painful episodes (VOE) to further knowledge on efficacy and safety of this orphan drug.

Key facts

Study ID

NCT04839354

Run by

Claudia R. Morris

People needed

271

Starts

2021-06-21

Expected to finish

2024-07-11

Last updated by the study team

2026-06-23

Who can join

Age: 3 and older, up to 21. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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

You may not qualify if…

• Responds to 2 doses of IV opioids sufficiently for outpatient management
• Greater than 12 hours from first dose of intravenous opioids to treat current pain in acute care setting
• Hemoglobin less than 5 gm/dL or emergent need for red blood cell transfusion for hemodynamically unstable patient
• Ketamine use in the emergency department for treatment of VOE
• Glutamine within 30 days
• New SCD drug use < 3 months (e.g. Hydroxyurea, voxelotor, crizanlizumab, etc)
• Acute mental status or neurological changes
• Acute stroke or clinical concern for stroke
• Three or more ED visits for sickle cell related pain receiving parenteral opioids in previous 7 days (not including current emergency department visit)
• Hospital discharge within previous 7 days
• Hypotension requiring clinical intervention; hemodynamic instability; septic shock
• Previous randomization in this arginine phase 3 randomized controlled trial
• Use of inhaled nitric oxide, sildenafil or arginine within the last month
• Non-English speaking or requires a translator for clinical care
• Pregnancy
• Allergy to arginine
• PI/clinical team concerns for compliance/issues that may adversely impact study participation/outcome
• Adults 18 years or older who lack medical decision-making capacity to consent

Where it is running

• Children's Hospital Los Angeles — Los Angeles, California, United States
• UCSF Benioff Children's Hospital — San Francisco, California, United States
• Children's National Medical Center — Washington D.C., District of Columbia, United States
• Children's Healthcare of Atlanta at Hughes Spalding — Atlanta, Georgia, United States
• Children's Healthcare of Atlanta at Egleston — Atlanta, Georgia, United States
• Washington University/St. Louis Children's Hospital — St Louis, Missouri, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Texas Children's Hospital/Baylor College of Medicine — Houston, Texas, United States
• Medical College of Wisconsin/Wisconsin Children's Hospital — Wauwatosa, Wisconsin, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.