Arginine Therapy in Sickle Cell Disease-VOC Clinical Trial

Study Overview
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Brief Summary
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Vaso-occlusion contributes significantly to morbidity in sickle cell disease (SCD). Vaso-occlusive painful episodes (VOE) are common and debilitating, causing the majority of emergency department visits. Currently efforts to treat painful episodes with use of non-steroidal pain relievers and intravenous narcotics offer symptomatic relief only, without targeting the underlying mechanisms of vaso-occlusion.Investigators have found that an arginine deficiency and low NO bioavailability occurs during painful events in SCD. Since arginine is the obligate substrate for NO production, and an acute deficiency is associated with VOE, investigators hypothesized that arginine supplementation may be a safe and beneficial treatment for sickle cell pain.

Official Title
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Arginine Therapy in Sickle Cell Disease-VOC Clinical Trial

Conditions
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Vaso-occlusive Pain Episodes

Intervention / Treatment
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* Drug: Arginine
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Established Diagnosis of Sickle Cell Disease(SS,SC,S-beta thal) Admitted to Hospital for pain Pain requiring hospitalization for parenteral narcotics, not attributable to non-sickle cell causes >3 year and older Exclusion Criteria: Hemoglobin less than 5gm/dL or immediate need for red cell transfusion Hepatic Dysfunction: increased in SGPT to >2x normal value Renal Dysfunction: increased in creatinine to >2x normal value or >1.5 Mental status or neurological changes Pregnancy >10 Hospitalizations per year or history of dependance to narcotics Inability to take oral medications or allergy to arginine Inability to use a PCA device < 3 years of age

Ages Eligible for Study
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Minimum Age: 3 Years
Maximum Age: 21 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Arginine<br>100 mg/kg T.I.D 3x a day IV or PO | Drug: Arginine<br> <br> * Other names: L-arginine, L-arginine-HCL;|
| Placebo Comparator: Placebo<br>Saline or sugar pill | Drug: Placebo<br>* Saline or Sugar pill was given as placebo<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Length of Hospital Stay |  | participants will be followed for the duration of hospital stay an expected average of 3-6 days, with re-admission data being collect for up to 30 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Effect on Pain Score |  | participants will be followed for the duration of hospital stay an expected average of 3-6 days | 
| Total Opioid Use (mg/kg) |  | participants will be followed for the duration of hospital stay an expected average of 3-6 days | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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sickle cell disease, vaso-occlusive pain episodes, arginine, nitric oxide