271 Participants Needed

Arginine Therapy for Sickle Cell Disease

(STArT Trial)

Recruiting at 9 trial locations
Claudia R. Morris | Faculty | People ...
Overseen ByClaudia Morris, MD
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 2 JurisdictionsThis treatment is already approved in other countries

Trial Summary

What is the purpose of this trial?

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.

Research Team

Claudia R. Morris | Faculty | People ...

Claudia Morris, MD

Principal Investigator

Emory University

Eligibility Criteria

The STArT trial is for children and young adults aged 3-21 with sickle cell disease experiencing pain episodes needing medical care. They must not have had certain treatments or hospitalizations recently, be stable without severe anemia, and able to understand English or Spanish.

Inclusion Criteria

I have been diagnosed with sickle cell disease.
I have had severe pain from sickle cell disease treated with strong painkillers.
I am between 3 and 21 years old.

Exclusion Criteria

Previous randomization in this arginine phase 3 RCT; OR
I am 18 or older but cannot make medical decisions for myself.
pregnancy; OR
See 15 more

Treatment Details

Interventions

  • Arginine Hydrochloride
Trial OverviewThis study tests IV arginine therapy versus saline (a placebo) in managing pain during vaso-occlusive episodes in sickle cell patients. It aims to determine the effectiveness and safety of arginine compared to a standard treatment.
Participant Groups
2Treatment groups
Experimental Treatment
Placebo Group
Group I: L-Arginine HydrochlorideExperimental Treatment1 Intervention
Participants receiving L-arginine hydrochloride in parenteral form. Participants receive up to 21 doses, with participants who are discharged early receiving fewer doses.
Group II: PlaceboPlacebo Group1 Intervention
Participants receiving normal saline as a placebo for L-arginine hydrochloride for up to 21 doses, with participants who are discharged early receiving fewer doses.

Arginine Hydrochloride is already approved in European Union, United States for the following indications:

🇪🇺
Approved in European Union as L-arginine for:
  • Nutritional supplement
  • COVID-19-related conditions
🇺🇸
Approved in United States as L-arginine for:
  • Nutritional supplement
  • COVID-19-related conditions

Find a Clinic Near You

Who Is Running the Clinical Trial?

Emory University

Lead Sponsor

Trials
1,735
Recruited
2,605,000+

Claudia R. Morris

Lead Sponsor

Trials
1
Recruited
360+

National Heart, Lung, and Blood Institute (NHLBI)

Collaborator

Trials
3,987
Recruited
47,860,000+