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L-arginine

Phase 2

Sickle Cell Disease | Small molecule | Hematology |National Healthcare Corp.|Last Updated: Sep 6, 2023

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment108

FDA Designations

No designations recorded

Clinical trial landscape

L-arginine · 1 trial · 2 indications

Phase 2 1
NCT02536170Arginine Therapy for Sickle Cell Disease PainSickle Cell Disease
COMPLETED108 Analytics
PHASE2COMPLETED
Arginine Therapy for Sickle Cell Disease Pain
Sickle Cell DiseaseUnlock trial analytics

Study Endpoints

Primary Endpoints

Total Parenteral Opioid Use in IV Morphine Equivalents
Post study drug delivery to discharge from the hospital (Up to 8 days)

The total amount of parenteral opioids used by participants measured in mg/kg of IV morphine equivalents. The total is calculated after study drug delivery for participants in the emergency department (ED) and during hospital stay.

Secondary Endpoints

Length of Hospital Stay
Discharge (Up to 8 days)
Time to Vaso-occlusive Pain Event (VOE) Resolution in Emergency Department
Post study drug delivery (Up to 8 hours)
Time to Vaso-occlusive Pain Event (VOE) Resolution in Hospital
Post study drug delivery until discharge (up to 8 days)
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Study Design & Arms

AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
L-ArginineEXPERIMENTALParticipants will be randomized to receive an intravenous (IV) infusion of L-arginine (100 mg/kg) three times a day until time of discharge from the emergency department (ED) or hospital.
Loading Dose and L-ArginineEXPERIMENTALParticipants will be randomized to receive an intravenous (IV) infusion of one-time loading dose of L-arginine (200 mg/kg) followed by standard dose (100 mg/kg) three times a day until time of discharge from the emergency department (ED) or hospital.
PlaceboPLACEBO_COMPARATORParticipants will be randomized to receive an intravenous (IV) infusion of placebo (normal saline 1-2 ml/kg) three times a day until time of discharge from the emergency department (ED) or hospital.

Interventions

NameTypeDescription
L-arginineDRUGL-arginine will be dispensed intravenously (IV) in the standard dose of 100 mg/kg three times a day until discharge from the emergency department (ED) or hospital.
L-arginine Loading DoseDRUGOne loading dose of L-arginine will be dispensed intravenously (IV) at 200 mg/kg
PlaceboOTHERPlacebo of intravenous (IV) normal saline 1-2 ml/kg three times a day until discharge from the emergency department (ED) or hospital.
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Eligibility Criteria

Age Range3 Years to 21 Years
SexALL
Healthy VolunteersNo
Study Sites3

Inclusion Criteria: * Established diagnosis of sickle cell disease (SCD); all genotypes * Pain requiring medical care in an acute care setting (such as the emergency department or ED, hospital ward, day hospital, clinic) not attributable to non-sickle cell causes, that is moderate-to-severe requiri...

Countries:United States
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Frequently asked questions about L-arginine

What is L-arginine used for in sickle cell disease?

L-arginine is an investigational small molecule being studied for the treatment of sickle cell disease, specifically for vaso-occlusive pain episodes. It is currently in Phase 2 clinical development and is not yet approved by the FDA. The drug is being developed by National Healthcare Corp. (NHC).

How does L-arginine work?

L-arginine is an amino acid that serves as a precursor for nitric oxide production. In sickle cell disease, it is thought to improve blood flow and reduce vaso-occlusion by enhancing nitric oxide bioavailability, which helps relax blood vessels and decrease red blood cell sickling. This mechanism is being evaluated in clinical trials.

Who makes L-arginine?

L-arginine is being developed by National Healthcare Corp. (ticker: NHC). The company is conducting clinical trials to evaluate the drug for the treatment of sickle cell disease, specifically for vaso-occlusive pain episodes. The drug is currently in Phase 2 development.

What phase is L-arginine in?

L-arginine is in Phase 2 clinical development for sickle cell disease. It is an investigational drug and has not been approved by the FDA. One Phase 2 trial has been completed, and the drug is not currently in active clinical trials.

What clinical trials is L-arginine in?

L-arginine has one completed Phase 2 clinical trial, NCT02536170, titled "Arginine Therapy for Sickle Cell Disease Pain." This randomized, double-blind, placebo-controlled trial enrolled 108 participants with sickle cell disease and vaso-occlusive pain episodes in the United States. The trial included patients as young as 3 years old.

Is L-arginine the same as arginine?

L-arginine is a form of the amino acid arginine. In the context of sickle cell disease research, L-arginine is the specific formulation being studied. It is also known simply as arginine, and the clinical trial NCT02536170 refers to it as arginine therapy.