NCT00951808

Brief Summary

Acute chest syndrome (ACS) is similar to severe pneumonia and is a common cause of hospitalizations for people with sickle cell disease (SCD). Blood transfusions are one treatment option for ACS. High levels of an enzyme called secretory phospholipase A2 (sPLA2) may be present in people before they develop ACS. This study will determine how well sPLA2 levels can predict the onset of ACS and whether identifying high sPLA2 levels allows enough time to prevent ACS with blood transfusions. Results from this study will help to determine the feasibility of conducting a larger study that would further examine the use of sPLA2 levels and blood transfusions to prevent ACS in people with SCD.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
237

participants targeted

Target at P75+ for not_applicable

Timeline
Completed

Started Jul 2009

Geographic Reach
1 country

24 active sites

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

July 1, 2009

Completed
1 month until next milestone

First Submitted

Initial submission to the registry

July 31, 2009

Completed
4 days until next milestone

First Posted

Study publicly available on registry

August 4, 2009

Completed
10 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2010

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2010

Completed
2.8 years until next milestone

Results Posted

Study results publicly available

April 5, 2013

Completed
Last Updated

April 24, 2013

Status Verified

April 1, 2013

Enrollment Period

11 months

First QC Date

July 31, 2009

Results QC Date

April 25, 2012

Last Update Submit

April 16, 2013

Conditions

Keywords

Anemia, Sickle CellAcute Chest SyndromeSecretory phospholipase A2(sPLA2)

Outcome Measures

Primary Outcomes (1)

  • Acute Chest Syndrome

    First occurence of positive infiltrate on chest x-ray

    Chest x-rays (CXR) were ordered for trial eligibility, as a result of clinical indications, or at discharge or 72 hours if no prior CXR.

Study Arms (3)

Blood Transfusion Trial Cohort

ACTIVE COMPARATOR

Twenty participants will receive a blood transfusion while in the hospital.

Biological: Single blood transfusion

Standard Care Trial Cohort

ACTIVE COMPARATOR

Twenty participants will not receive a blood transfusion and will receive standard care.

Behavioral: Standard care

Standard Care Observational Cohort

ACTIVE COMPARATOR

Approximately 300 participants who are ineligible for or decline the blood transfusion part of the study will participate in the observational portion of the study and receive standard care.

Behavioral: Standard care

Interventions

Participants will receive a single transfusion of 7-13cc/kg packed red blood cells (RBCs) while in the hospital.

Also known as: transfusion
Blood Transfusion Trial Cohort
Standard careBEHAVIORAL

Participants will receive standard care for ACS while in the hospital.

Also known as: standard of care
Standard Care Observational CohortStandard Care Trial Cohort

Eligibility Criteria

Age2 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Hemoglobin diagnosis of SS (two copies of the hemoglobin S gene), SC (one copy of the hemoglobin S gene and one copy of the hemoglobin C gene), or S-β thalassemia (β+ or β0)
  • No clinically apparent ACS
  • No prior participation in either part of the study
  • sPLA2 level greater than 100 ng/mL within the same 24-hour window that coincides with fever and chest radiograph negative for new pulmonary infiltrate within the last 12 hours of the 24-hour window
  • Fever greater than 38.0º C within the same 24-hour window that coincides with elevated sPLA2 level (greater than 100 ng/mL) and chest radiograph negative for new pulmonary infiltrate within the last 12 hours of the 24-hour window
  • Chest radiograph negative for new pulmonary infiltrate within the last 12 hours of the 24-hour window of an abnormal sPLA2 level and fever
  • Hemoglobin levels equal or less than 10 g/dL at time of study entry
  • Informed consent of parent(s) or legal guardian; informed consent or assent of participant as applicable

You may not qualify if:

  • Existing diagnosis of a new pulmonary infiltrate diagnosed by chest radiography (pleural effusion not obscuring lung parenchyma will not exclude the person from the study)
  • Any coexisting medical condition for which the physician feels that a transfusion may be needed within 24 hours (e.g., severe anemia, stroke)
  • Red Blood Cell (RBC) transfusion in the 60 days before study entry
  • Unwillingness to sign consent form, or if a minor, unwillingness of parent/guardian to sign consent form
  • Treatment with any investigational drug or device in the 30 days before study entry (hydroxyurea is allowable)
  • History of alloimmunization that would prevent the participant from receiving blood within 8 hours of eligibility for study entry or history of a life-threatening transfusion reaction
  • Objection to transfusion for religious or other reasons from either the participant or guardian
  • History of treatment with systemic steroids within 1 week of study entry (inhaled steroids are acceptable)
  • Pregnant

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (24)

Children's Hospital and Research Center

Oakland, California, United States

Location

A.I. duPont Hospital for Children

Wilmington, Delaware, United States

Location

Children's National Medical Center

Washington D.C., District of Columbia, United States

Location

Howard University Hospital

Washington D.C., District of Columbia, United States

Location

Emory University School of Medicine

Atlanta, Georgia, United States

Location

Medical College of Georgia

Augusta, Georgia, United States

Location

Children's Memorial Hospital

Chicago, Illinois, United States

Location

University of Illinois Sickle Cell Center

Chicago, Illinois, United States

Location

Kosair Children's Hospital

Louisville, Kentucky, United States

Location

Johns Hopkins

Baltimore, Maryland, United States

Location

Boston Medical Center

Boston, Massachusetts, United States

Location

Brigham & Women's Hospital

Boston, Massachusetts, United States

Location

Children's Hospital Boston

Boston, Massachusetts, United States

Location

University of Mississippi Medical Center

Jackson, Mississippi, United States

Location

Interfaith Medical Center

Brooklyn, New York, United States

Location

New York Methodist Hospital

Brooklyn, New York, United States

Location

The University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States

Location

Duke University Medical Center

Durham, North Carolina, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, United States

Location

Ohio State University

Columbus, Ohio, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Location

St. Christopher's Hospital for Children

Philadelphia, Pennsylvania, United States

Location

Virginia Commonwealth University Health Systems

Richmond, Virginia, United States

Location

Related Publications (3)

  • Miller ST, Kim HY, Weiner D, Wager CG, Gallagher D, Styles L, Dampier CD; Investigators of the Sickle Cell Disease Clinical Research Network (SCDCRN). Inpatient management of sickle cell pain: a 'snapshot' of current practice. Am J Hematol. 2012 Mar;87(3):333-6. doi: 10.1002/ajh.22265. Epub 2012 Jan 9.

  • Styles L, Wager CG, Labotka RJ, Smith-Whitley K, Thompson AA, Lane PA, McMahon LE, Miller R, Roseff SD, Iyer RV, Hsu LL, Castro OL, Ataga KI, Onyekwere O, Okam M, Bellevue R, Miller ST; Sickle Cell Disease Clinical Research Network (SCDCRN). Refining the value of secretory phospholipase A2 as a predictor of acute chest syndrome in sickle cell disease: results of a feasibility study (PROACTIVE). Br J Haematol. 2012 Jun;157(5):627-36. doi: 10.1111/j.1365-2141.2012.09105.x. Epub 2012 Mar 30.

  • Miller ST, Kim HY, Weiner DL, Wager CG, Gallagher D, Styles LA, Dampier CD, Roseff SD; Investigators of the Sickle Cell Disease Clinical Research Network (SCDCRN). Red blood cell alloimmunization in sickle cell disease: prevalence in 2010. Transfusion. 2013 Apr;53(4):704-9. doi: 10.1111/j.1537-2995.2012.03796.x. Epub 2012 Jul 13.

MeSH Terms

Conditions

Anemia, Sickle CellAcute Chest Syndrome

Interventions

Blood TransfusionStandard of Care

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLung DiseasesRespiratory Tract DiseasesRespiration Disorders

Intervention Hierarchy (Ancestors)

Biological TherapyTherapeuticsQuality Indicators, Health CareQuality of Health CareHealth Services AdministrationHealth Care Quality, Access, and Evaluation

Limitations and Caveats

The feasibility study has been completed, but the randomization trial has been terminated early due to the lack of enrollment. Therefore, the results were reported with the subjects who completed the feasibility study and were not reported by Arm.

Results Point of Contact

Title
Hae-Young Kim
Organization
New England Research Institutes

Study Officials

  • Sonja McKinlay, PhD

    Carelon Research

    PRINCIPAL INVESTIGATOR
  • Margaret C. Bell, MPH, MS

    Carelon Research

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
Yes

Study Design

Study Type
interventional
Phase
not applicable
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 31, 2009

First Posted

August 4, 2009

Study Start

July 1, 2009

Primary Completion

June 1, 2010

Study Completion

July 1, 2010

Last Updated

April 24, 2013

Results First Posted

April 5, 2013

Record last verified: 2013-04

Locations