Clinical trial NCT01796678: Arginine Therapy in Sickle Cell Disease-VOC Clinical Trial
Official title: Arginine Therapy in Sickle Cell Disease-VOC Clinical Trial
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Vaso-occlusive Pain Episodes
Keywords: sickle cell disease
vaso-occlusive pain episodes
arginine
nitric oxide
Lead sponsor: UCSF Benioff Children's Hospital Oakland
Sponsor class: OTHER
First posted: 2013-02-22
Enrollment: 56
Sex: ALL
Minimum age (years): 3
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Length of Hospital Stay
timeFrame: 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 outcomes:
measure: Effect on Pain Score
timeFrame: participants will be followed for the duration of hospital stay an expected average of 3-6 days
measure: Total Opioid Use (mg/kg)
timeFrame: participants will be followed for the duration of hospital stay an expected average of 3-6 days
Other outcomes:
Locations:
city: Oakland
country: United States
facility: Childrens Hospital Research Center Oakland
state: California
Study officials:
affiliation: Childrens Hospital Oakland
name: Claudia R Morris, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Morris CR, Kuypers FA, Lavrisha L, Ansari M, Sweeters N, Stewart M, Gildengorin G, Neumayr L, Vichinsky EP. A randomized, placebo-controlled trial of arginine therapy for the treatment of children with sickle cell disease hospitalized with vaso-occlusive pain episodes. Haematologica. 2013 Sep;98(9):1375-82. doi: 10.3324/haematol.2013.086637. Epub 2013 May 3.