NCT01522196

Brief Summary

The purpose of this study is to determine the effectiveness of A 001 infusion in preventing acute chest syndrome in sickle cell disease (SCD) subjects with vaso-occlusive crisis, fever, and elevated serum C-reactive protein (CRP).

Trial Health

57
Monitor

Trial Health Score

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

Enrollment
2

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Feb 2012

Shorter than P25 for phase_2

Geographic Reach
1 country

1 active site

Status
terminated

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

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

January 18, 2012

Completed
13 days until next milestone

First Posted

Study publicly available on registry

January 31, 2012

Completed
1 day until next milestone

Study Start

First participant enrolled

February 1, 2012

Completed
29 days until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2012

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2012

Completed
Last Updated

March 4, 2014

Status Verified

January 1, 2014

Enrollment Period

29 days

First QC Date

January 18, 2012

Last Update Submit

January 30, 2014

Conditions

Sickle Cell DiseaseVaso-occlusive Crisis

Keywords

SCD

Outcome Measures

Primary Outcomes (1)

  • Efficacy of A 001 infusion in preventing acute chest syndrome in sickle cell disease (SCD) subjects with the combination of vaso-occlusive crisis, fever, and elevated serum C-reactive protein (CRP).

    Various time points up to Day 30

Secondary Outcomes (4)

  • Safety and tolerability of A 001 therapy when administered as a 48-hour continuous infusion to SCD subjects at-risk for acute chest syndrome.

    Various time points up to Day 30

  • Impact of A-001 treatment on sPLA2 levels and CRP.

    Various time points up to Day 30

  • Impact of A-001 treatment on the signs and symptoms of vaso-occlusive crisis.

    Various time points up to Day 30

  • Impact of A-001 treatment on opioid use for pain in the treatment of vaso-occlusive crisis.

    Various time points up to Day 30

Study Arms (2)

Varespladib

ACTIVE COMPARATOR

48 hour continuous infusion delivered intravenously (IV)

Drug: Varespladib

Placebo

PLACEBO COMPARATOR

48 hour continuous infusion delivered intravenously (IV)

Other: Placebo (Normal Saline)

Interventions

VarespladibDRUG

48 hour continuous infusion delivered intravenously (IV)at a rate of 110µg/kg/hr.

Varespladib
Placebo (Normal Saline)OTHER

48 hour continuous infusion delivered intravenously (IV)at a rate of 110µg/kg/hr.

Placebo

Eligibility Criteria

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

You may qualify if:

  • Genotypes Hb SS, Hb SC, sickle β°-thalassemia, sickle β+ -thalassemia
  • Pain consistent with vaso-occlusive crisis (clinical judgment) and not attributable to other causes
  • Serum CRP ≥5.0 mg/L at time of screening
  • Fever defined as oral temperature ≥38.0°C at time of screening
  • Age ≥5 years

You may not qualify if:

  • New or suspected new pulmonary infiltrate diagnosed by chest radiography
  • Females who are nursing, pregnant or intend to become pregnant
  • Renal dysfunction defined as a creatinine level \>1.2 mg/dL for subjects aged 18 or less or a creatinine level \>1.5 mg/dL for subjects over the age of 18
  • Hepatic dysfunction (alanine aminotransferase \[ALT\] or aspartate aminotransferase \[AST\] \>3 × upper limit of normal)
  • 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 prior to screening
  • Parenteral or oral corticosteroid therapy (inhaled steroids acceptable) within 7 days prior to screening

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Investigator Site 101

Atlanta, Georgia, 30342, United States

Location

MeSH Terms

Conditions

Anemia, Sickle CellVaso-Occlusive Crises

Interventions

varespladibSaline Solution

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Crystalloid SolutionsIsotonic SolutionsSolutionsPharmaceutical Preparations

Study Design

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

Study Record Dates

First Submitted

January 18, 2012

First Posted

January 31, 2012

Study Start

February 1, 2012

Primary Completion

March 1, 2012

Study Completion

March 1, 2012

Last Updated

March 4, 2014

Record last verified: 2014-01

Locations

US(1)