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Arginine Therapy in Sickle Cell Disease-VOC Clinical Trial

Primary Purpose

Vaso-occlusive Pain Episodes

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Arginine
Placebo
Sponsored by
UCSF Benioff Children's Hospital Oakland
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Vaso-occlusive Pain Episodes focused on measuring sickle cell disease, vaso-occlusive pain episodes, arginine, nitric oxide

Eligibility Criteria

3 Years - 21 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Childrens Hospital Research Center Oakland

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Arginine

Placebo

Arm Description

100 mg/kg T.I.D 3x a day IV or PO

Saline or sugar pill

Outcomes

Primary Outcome Measures

Length of Hospital Stay

Secondary Outcome Measures

Effect on Pain Score
Total Opioid Use (mg/kg)

Full Information

First Posted
February 19, 2013
Last Updated
July 29, 2013
Sponsor
UCSF Benioff Children's Hospital Oakland
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1. Study Identification

Unique Protocol Identification Number
NCT01796678
Brief Title
Arginine Therapy in Sickle Cell Disease-VOC Clinical Trial
Official Title
Arginine Therapy in Sickle Cell Disease-VOC Clinical Trial
Study Type
Interventional

2. Study Status

Record Verification Date
July 2013
Overall Recruitment Status
Completed
Study Start Date
September 2000 (undefined)
Primary Completion Date
June 2007 (Actual)
Study Completion Date
June 2007 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
UCSF Benioff Children's Hospital Oakland

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Vaso-occlusive Pain Episodes
Keywords
sickle cell disease, vaso-occlusive pain episodes, arginine, nitric oxide

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
56 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Arginine
Arm Type
Experimental
Arm Description
100 mg/kg T.I.D 3x a day IV or PO
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Saline or sugar pill
Intervention Type
Drug
Intervention Name(s)
Arginine
Other Intervention Name(s)
L-arginine, L-arginine-HCL
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Saline or Sugar pill was given as placebo
Primary Outcome Measure Information:
Title
Length of Hospital Stay
Time Frame
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 Measure Information:
Title
Effect on Pain Score
Time Frame
participants will be followed for the duration of hospital stay an expected average of 3-6 days
Title
Total Opioid Use (mg/kg)
Time Frame
participants will be followed for the duration of hospital stay an expected average of 3-6 days

10. Eligibility

Sex
All
Minimum Age & Unit of Time
3 Years
Maximum Age & Unit of Time
21 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Claudia R Morris, MD
Organizational Affiliation
Childrens Hospital Oakland
Official's Role
Principal Investigator
Facility Information:
Facility Name
Childrens Hospital Research Center Oakland
City
Oakland
State/Province
California
ZIP/Postal Code
94609
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
23645695
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.
Results Reference
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Arginine Therapy in Sickle Cell Disease-VOC Clinical Trial

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