NCT00434473

Brief Summary

The study will be conducted at 15-20 US centers in a randomized, placebo-controlled, double-blind fashion. Enrollees will be hospitalized sickle cell disease (SCD) patients at-risk for acute chest syndrome (ACS) based on the presence of vaso-occlusive crisis (VOC), fever (T ≥38.0°C) and serum sPLA2 concentration ≥50 ng/mL.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
30

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Dec 2006

Typical duration for phase_2

Geographic Reach
1 country

6 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

December 1, 2006

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

February 9, 2007

Completed
4 days until next milestone

First Posted

Study publicly available on registry

February 13, 2007

Completed
2.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2009

Completed
4 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2009

Completed
Last Updated

March 4, 2014

Status Verified

January 1, 2014

Enrollment Period

2.7 years

First QC Date

February 9, 2007

Last Update Submit

January 30, 2014

Conditions

Outcome Measures

Primary Outcomes (1)

  • To determine the safety and tolerability of different doses of A 001 therapy when administered as a 2-day continuous infusion to sickle cell disease (SCD) patients at-risk for the acute chest syndrome (ACS).

    Study end

Secondary Outcomes (3)

  • To determine the pharmacokinetic profile of A-001 in SCD patients

    Study end

  • To confirm the ability of A-001 infusion to suppress serum sPLA2 activity in SCD patients with elevated serum sPLA2

    Study end

  • To determine the efficacy of A-001 infusion in preventing ACS in SCD patients with the combination of vaso-occlusive crisis (VOC), fever, and elevated serum sPLA2

    Study end

Study Arms (2)

Placebo

PLACEBO COMPARATOR
Other: Placebo

A-001

EXPERIMENTAL
Drug: A-001

Interventions

A-001DRUG

A-001

A-001
PlaceboOTHER

Placebo

Placebo

Eligibility Criteria

Age5 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may not qualify if:

  • New lung infiltrate by chest radiography
  • Pregnancy or breastfeeding
  • Significant renal dysfunction
  • Significant hepatic dysfunction
  • Acute neurologic dysfunction
  • Any medical condition for which transfusion may be needed imminently, and/or hemoglobin \<5 g/dL
  • Red blood cell transfusion within 30 days of entry into the study

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (6)

Howard University Hospital

Washington D.C., District of Columbia, 20060, United States

Location

Children's Healthcare of Atlanta

Atlanta, Georgia, 30342, United States

Location

Children's Memorial Hospital

Chicago, Illinois, 60614, United States

Location

SUNY Downstate Medical Center

Brooklyn, New York, 11203, United States

Location

Duke University Comprehensive Sickle Cell Center

Durham, North Carolina, 27713, United States

Location

Virginia Commonwealth University

Richmond, Virginia, 23219, United States

Location

MeSH Terms

Conditions

Anemia, Sickle CellVaso-Occlusive CrisesAcute Chest Syndrome

Condition Hierarchy (Ancestors)

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

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
PREVENTION
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 9, 2007

First Posted

February 13, 2007

Study Start

December 1, 2006

Primary Completion

August 1, 2009

Study Completion

December 1, 2009

Last Updated

March 4, 2014

Record last verified: 2014-01

Locations