Arginine for Sickle Cell Disease

Children's Healthcare of Atlanta at Egleston, Atlanta, GA
Sickle Cell DiseaseArginine - Drug
7 - 21
All Sexes

Study Summary

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

Eligible Conditions
  • Sickle Cell Disease

Treatment Effectiveness

Study Objectives

2 Primary · 12 Secondary · Reporting Duration: Day 1 through study completion, an average of up to 7 days

Day 7
Change in asymmetric dimethylarginine (ADMA) levels
Day 7
Change in nitric oxide metabolites
Change in red blood cell (RBC) arginine
Day 1
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
Maximum observed plasma concentration of arginine
Terminal elimination half-life (t1/2) for arginine
Day 7
Pharmacokinetics of IV arginine, measured by plasma arginine concentration over time
Day 7
Daily urine arginine
Day 7
Biomarkers of hemolysis
Erythrocyte glutathione levels
Global arginine bioavailability (GABR)
Level of cytokines
Modeling nitric oxide (NOx) level versus plasma arginine level

Trial Safety

Side Effects for

Low-dose Arginine Plus Buphenyl
This histogram enumerates side effects from a completed 2012 Phase 2 trial (NCT00345605) in the Low-dose Arginine Plus Buphenyl ARM group. Side effects include: Vomiting with 8%, Hyperammonemia with 8%.

Awards & Highlights

No Placebo Group
All patients enrolled in this trial will receive the new treatment.

Trial Design

3 Treatment Groups

Standard dose
1 of 3
Loading dose + continuous infusion
1 of 3
Loading dose + standard dose
1 of 3

Experimental Treatment

21 Total Participants · 3 Treatment Groups

Primary Treatment: Arginine · No Placebo Group · Phase 1 & 2

Standard dose
Experimental Group · 1 Intervention: Arginine · Intervention Types: Drug
Loading dose + continuous infusionExperimental Group · 2 Interventions: Arginine (Loading), Arginine (Continuous) · Intervention Types: Drug, Drug
Loading dose + standard doseExperimental Group · 2 Interventions: Arginine (Loading), Arginine · Intervention Types: Drug, Drug
First Studied
Drug Approval Stage
How many patients have taken this drug
Not yet FDA approved

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: day 1 through study completion, an average of up to 7 days

Who is running the clinical trial?

National Center for Complementary and Integrative Health (NCCIH)NIH
797 Previous Clinical Trials
657,631 Total Patients Enrolled
Emory UniversityLead Sponsor
1,559 Previous Clinical Trials
2,765,750 Total Patients Enrolled
Children's Healthcare of AtlantaOTHER
159 Previous Clinical Trials
48,231 Total Patients Enrolled
Claudia Morris, MD4.8319 ReviewsPrincipal Investigator - Emory University
Northside Hospital
Medical School - Emory University, Doctor of Medicine
Emory University, Residency in Obstetrics and Gynecology
4 Previous Clinical Trials
508 Total Patients Enrolled
5Patient Review
So far, Dr. Ayres has been great to work with and I hope to continue our relationship.

Eligibility Criteria

Age 7 - 21 · All Participants · 0 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:

Frequently Asked Questions

Are there any vacancies for participants in this clinical trial?

"Correct. 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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

How many human participants have registered for this research effort?

"Affirmative. As per, 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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.