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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

Timeline

Screening 3 weeks

Treatment Varies

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

Awards & highlights

R34 pK/PD Trial 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.Check my eligibility

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.See study design

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.

R34 pK/PD Trial 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.

R34 pK/PD Trial 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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change in nitric oxide metabolites

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

Secondary outcome measures

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

Vomiting

Hyperammonemia

100%

80%

60%

40%

20%

Study treatment Arm

Low-dose Arginine Plus Buphenyl

High-dose Arginine Alone

R34 pK/PD Trial Design

3Treatment 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: 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 III: 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

~480

0 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

National Center for Complementary and Integrative Health (NCCIH)NIH

832 Previous Clinical Trials

668,952 Total Patients Enrolled

Emory UniversityLead Sponsor

1,639 Previous Clinical Trials

2,560,625 Total Patients Enrolled

Children's Healthcare of AtlantaOTHER

165 Previous Clinical Trials

105,109 Total Patients Enrolled

Media Library

Arginine (Amino Acid) Clinical Trial Eligibility Overview. Trial Name: NCT02447874 — Phase 1 & 2

Sickle Cell Disease Research Study Groups: Standard dose, Loading dose + continuous infusion, Loading dose + standard dose

Sickle Cell Disease Clinical Trial 2023: Arginine Highlights & Side Effects. Trial Name: NCT02447874 — Phase 1 & 2

Arginine (Amino Acid) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02447874 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there any vacancies for participants in this clinical trial?

"Correct. Clinicaltrials.gov records show that this medical trial, which was initialy posted on the 1st of May 2015 and last updated on 22nd April 2022 is now accepting participants. Approximately 21 people are needed from two distinct locations for enrollment into this study."

Answered by AI

Is this trial the inaugural venture of its kind?

"Presently, 14 trials evaluating Arginine are in progress across 24 cities and 11 distinct nations. This drug's clinical journey began with Baxter Healthcare Corporation sponsoring a Phase 4 study back in 2007; the research involved 4640 test-subjects and was ultimately successful. Subsequently, 58 further investigations have been conducted over the intervening years."

Answered by AI

What are the criteria for eligibility in this trial?

"This medical trial requires 21 participants aged between 7 and 21, who have anemia caused by sickle cell disease. Additionally, they must weigh at least 25 kg (55lbs) and require opioid medications to manage the pain associated with their condition due to a visit to an emergency department or clinic."

Answered by AI

Have there been previous experiments regarding Arginine's effects?

"Currently, 14 trials are investigating the effects of Arginine with 4 studies in their final phase. While most investigations for this substance occur out of Santiago, Región Metropolitana, there are 41 different sites observing its properties."

Answered by AI

How many human participants have registered for this research effort?

"Affirmative. As per clinicaltrials.gov, this medical experiment is enrolling participants at the present time. Originally posted on May 1st 2015, it was most recently updated on April 22nd 2022 and requires 21 patients from 2 separate locations to complete the trial."

Answered by AI

Is there an age restriction on the participants of this trial?

"This research is in search of participants aged 7 or above but under 21 years old."

Answered by AI

What therapeutic applications is Arginine typically applied to?

"Arginine is a popular treatment for pituitary dwarfism, as well as conditions related to deficient enzymes, inadequate stimulation of the pituitary gland, and amino acid deficiency."

Answered by AI

Recent research and studies

BloodJournal

Impact of Arginine Therapy on Mitochondrial Function in Children with Sickle Cell Disease During Vaso-occlusive Pain

Morris, Claudia R., Lou Ann S. Brown, Michael Reynolds, Carlton D. Dampier, Peter A. Lane, Amanda Watt, Polly Kumari, et al.. 2020. “Impact of Arginine Therapy on Mitochondrial Function in Children with Sickle Cell Disease During Vaso-occlusive Pain”. Blood. American Society of Hematology. doi:10.1182/blood.2019003672.

BloodJournal

Impact of Arginine Therapy on Mitochondrial Function in Children with Sickle Cell Disease During Vaso-occlusive Pain

clinicaltrials.govStudy

Arginine Therapy for the Treatment of Pain in Children With Sickle Cell Disease

Claudia R. Morris 2015. "Arginine Therapy for the Treatment of Pain in Children With Sickle Cell Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02447874.

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