A Study of Varespladib Infusion in Subjects With Sickle Cell Disease.

Phase 2

Terminated

• Conditions: Sickle Cell Disease; Vaso-occlusive Crisis
• Interventions: Other: Placebo (Normal Saline); Drug: Varespladib

• Registration Number: NCT01522196
• Lead Sponsor: Anthera Pharmaceuticals

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).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2012-01-18
• Study First Submitted QC: 2012-01-27
• Study First Posted: 2012-01-31
• Study Start: 2012-02
• Primary Completion: 2012-03
• Study Completion: 2012-03
• Status Verified: 2014-01
• Disposition First Submitted: 2014-01-30
• Disposition First Submitted QC: 2014-01-30
• Last Update Submitted: 2014-01-30
• Disposition First Posted: 2014-03-04
• Last Update Posted: 2014-03-04

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 2

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo (Normal Saline)	48 hour continuous infusion delivered intravenously (IV)
Varespladib	Varespladib	48 hour continuous infusion delivered intravenously (IV)

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (1)

Investigator Site 101; 🇺🇸 Atlanta, Georgia, United States