Sickle Cell Disease, Vaso-occlusive Crisis
Conditions
Keywords
SCD
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).
Interventions
DRUGVarespladib
48 hour continuous infusion delivered intravenously (IV)at a rate of 110µg/kg/hr.
OTHERPlacebo (Normal Saline)
48 hour continuous infusion delivered intravenously (IV)at a rate of 110µg/kg/hr.
Sponsors
Anthera Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
PREVENTION
Masking
TRIPLE (Subject, Caregiver, Investigator)
Eligibility
Sex/Gender
ALL
Age
5 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 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
Exclusion criteria
* 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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
| Measure | Time frame |
|---|---|
| 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 |
Countries
United States
Outcome results
None listed