Arginine Therapy for Sickle Cell Disease

(STArT Trial)

Recruiting at 9 trial locations

Overseen By Claudia Morris, MD

Age: < 65

Sex: Any

Trial Phase: Phase 3

Sponsor: Emory University

Must not be taking: Ketamine, Glutamine, others

Disqualifiers: Stroke, Pregnancy, Hypotension, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 1-2 weeks

271 Participants Needed

What You Need to Know Before You Apply

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.

Who Is on the Research Team?

Claudia Morris, MD

Principal Investigator

Emory University

Are You a Good Fit for This Trial?

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

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive up to 21 doses of IV arginine or placebo over 7 to 8 days

1-2 weeks

Daily visits for IV administration

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Regular follow-up visits

Long-term Follow-up

Participants are followed for up to 28 days following hospital discharge to assess long-term outcomes

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Arginine Hydrochloride

Trial Overview This 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.

How Is the Trial Designed?

Group I: L-Arginine Hydrochloride Experimental Treatment 1 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: Placebo Placebo Group 1 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

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+