NCT01796678

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
56

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Sep 2000

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

September 1, 2000

Completed
6.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2007

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2007

Completed
5.7 years until next milestone

First Submitted

Initial submission to the registry

February 19, 2013

Completed
3 days until next milestone

First Posted

Study publicly available on registry

February 22, 2013

Completed
Last Updated

July 31, 2013

Status Verified

July 1, 2013

Enrollment Period

6.8 years

First QC Date

February 19, 2013

Last Update Submit

July 29, 2013

Conditions

Vaso-occlusive Pain Episodes

Keywords

sickle cell diseasevaso-occlusive pain episodesargininenitric oxide

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (2)

  • 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

Study Arms (2)

Arginine

EXPERIMENTAL

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

Drug: Arginine

Placebo

PLACEBO COMPARATOR

Saline or sugar pill

Drug: Placebo

Interventions

ArginineDRUG
Also known as: L-arginine, L-arginine-HCL
Arginine
PlaceboDRUG

Saline or Sugar pill was given as placebo

Placebo

Eligibility Criteria

Age3 Years - 21 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Childrens Hospital Research Center Oakland

Oakland, California, 94609, United States

Location

Related Publications (1)

  • 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.

    PMID: 23645695RESULT

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

Arginine

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Amino Acids, BasicAmino AcidsAmino Acids, Peptides, and ProteinsAmino Acids, DiaminoAmino Acids, Essential

Study Officials

  • Claudia R Morris, MD

    Childrens Hospital Oakland

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
TRIPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 19, 2013

First Posted

February 22, 2013

Study Start

September 1, 2000

Primary Completion

June 1, 2007

Study Completion

June 1, 2007

Last Updated

July 31, 2013

Record last verified: 2013-07

Locations

US(1)