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Amino Acid

Arginine Therapy for Sickle Cell Disease (R34 pK/PD Trial)

Phase 1 & 2

Recruiting

Led By Claudia Morris, MD

Research Sponsored by Emory University

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Established diagnosis of sickle cell disease--Hemoglobin SS (Hb-SS) or Sβᴼ-thalassemia

7-21 years of age

Must not have

Immediate Red cell transfusion anticipated

Hepatic dysfunction: serum glutamic pyruvic transaminase (SGPT) > 3X upper value

Timeline

Screening 3 weeks

Treatment Varies

Follow Up day 1 through study completion, an average of up to 7 days

Awards & highlights

No Placebo-Only Group

Summary

This trial will test whether giving extra arginine to sickle cell patients during a vaso-occlusive event will decrease pain.

Who is the study for?

This trial is for children and young adults aged 7-21 with Sickle Cell Disease (specifically Hemoglobin SS or Sβᴼ-thalassemia), weighing at least 55lbs, who are experiencing pain that requires strong painkillers. They must not be pregnant, have had a recent hospitalization, severe liver or kidney issues, certain other types of sickle cell disease, or an allergy to arginine.

What is being tested?

The study tests if extra arginine can help reduce pain levels in patients with sickle cell disease during painful episodes. It looks at whether this treatment can lessen the need for pain medication or shorten the time spent in hospitals or emergency departments.

What are the potential side effects?

While specific side effects aren't listed here, arginine supplements may commonly cause stomach discomfort and diarrhea. In people with sickle cell disease, there's also a potential risk of complications due to changes in blood flow.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with sickle cell disease, either Hb-SS or Sβᴼ-thalassemia.

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I am between 7 and 21 years old.

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I have pain from sickle cell that needed strong painkillers in a hospital or clinic.

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I weigh at least 55 pounds.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I expect to need a blood transfusion soon.

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My liver enzyme SGPT levels are more than three times the normal upper limit.

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My kidney function is reduced, with creatinine levels above normal.

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I have changes in my mental status or neurological condition.

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My last hospital stay was less than a week.

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I have been diagnosed with a type of sickle cell disease.

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I have had a recent stroke or symptoms suggesting a stroke.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ day 1 through study completion, an average of up to 7 days

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~day 1 through study completion, an average of up to 7 days

This trial's timeline: 3 weeks for screening, Varies for treatment, and day 1 through study completion, an average of up to 7 days for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change in nitric oxide metabolites

Pharmacokinetics of IV arginine, measured by plasma arginine concentration over time

Secondary study objectives

Apparent clearance of arginine

Area Under the Plasma Concentration -Time Curve (AUC) From Time 0 to the Time of the Last Quantifiable Concentration for Arginine

Biomarkers of hemolysis

+9 more

Side effects data

From 2012 Phase 2 trial • 12 Patients • NCT00345605

Hyperammonemia

Vomiting

100%

80%

60%

40%

20%

Study treatment Arm

Low-dose Arginine Plus Buphenyl

High-dose Arginine Alone

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

6Treatment groups

Experimental Treatment

Group I: Standard doseExperimental Treatment1 Intervention

Subjects with sickle cell disease (SCD) and vaso-occlusive painful events (VOE) will be randomized to receive an intravenous (IV) infusion of a standard dose of arginine (100 mg/kg) three times a day for seven days or until discharged from the hospital, whichever occurs first

Group II: Non-Randomized Loading dose 500 mg/kg + standard doseExperimental Treatment1 Intervention

Arginine will be dispensed intravenously (in the vein) as an initial bolus (loading) arginine dose at 500 mg/kg once, followed by a standard dose of 100mg/kg every 8 hours until discharge or for a total of 21 doses of arginine, whichever comes first.

Group III: Non-Randomized Loading dose 400mg/kg + standard doseExperimental Treatment1 Intervention

Arginine will be dispensed intravenously (in the vein) as an initial bolus (loading) arginine dose at 400 mg/kg once, followed by a standard dose of 100mg/kg every 8 hours until discharge or for a total of 21 doses of arginine, whichever comes first.

Group IV: Non-Randomized Loading dose 300 mg/kg + standard doseExperimental Treatment1 Intervention

Arginine will be dispensed intravenously (in the vein) as an initial bolus (loading) arginine dose at 300 mg/kg once, followed by a standard dose of 100mg/kg every 8 hours until discharge or for a total of 21 doses of arginine, whichever comes first.

Group V: Loading dose + standard doseExperimental Treatment2 Interventions

Subjects with sickle cell disease and vaso-occlusive painful events (VOE) will be randomized to receive an intravenous (IV) infusion of an initial loading dose of arginine (200 mg/kg) given over 30 minutes and then receive an intravenous (IV) infusion of a standard dose of arginine (100 mg/kg) three times a day for seven days or until discharged from the hospital, whichever occurs first

Group VI: Loading dose + continuous infusionExperimental Treatment2 Interventions

Subjects with sickle cell disease and vaso-occlusive painful events (VOE) will be randomized to receive an intravenous (IV) infusion of an initial loading dose of arginine (200 mg/kg) given over 30 minutes and then receive a continuous intravenous (IV) infusion of 300 mg/kg/24hr for 7 days or until discharged from the hospital, whichever occurs first

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Arginine

2016

Completed Phase 4

~760

0 / 11Eligibility criteria met