Acute Chest Syndrome (ACS) in At-Risk patients with Sickle Cell Disease (SCD) and Vaso-occlusive Crisis (VOC) MedDRA version: 9.1 Level: LLT Classification code 10051895 Term: Acute chest syndrome
Conditions
Interventions
Product Name: Varespladib Sodium
Product Code: A-001
Pharmaceutical Form: Intravenous infusion
INN or Proposed INN: Varespladib sodium
CAS Number: 172733-42-5
Current Sponsor code: A-001
Other descrip
Sponsors
Anthera Pharmaceuticals Incorporated
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Hb SS, Hb SC, sickle ß-thalassemia, sickle ß+ -thalassemia - Pain consistent with VOC (clinical judgment) and not attributable to other causes. - Serum sPLA2 level =100 ng/mL (measured on-site) - Fever defined as oral temperature =38.0°C - US and UK: age =12 years (through adult) Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: - New (or suspected new) pulmonary infiltrate diagnosed by chest radiography - Pregnancy or breastfeeding - Known hypersensitivity to any of the active constituents of the A-001 or the IV multivitamin preparation (placebo) - Renal dysfunction: creatinine >1.2 mg/dL for age 18 or less, creatinine >1.5 mg/dL for over age 18 - Hepatic dysfunction (ALT >2 × 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 of entry into the study - Unwillingness to sign consent form, or if a minor, unwillingness of parent/guardian to sign consent form - Parenteral or oral corticosteroid therapy (inhaled steroids acceptable) within 7 days of enrollment - Treatment with any investigational drug or device in the last 30 days - Any other current or past medical condition that might significantly affect pharmacodynamic responses to the administered drug, or any imminently life-threatening illness
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine the safety and tolerability of different doses of A-001 therapy when administered as a 2-day continuous infusion to SCD patients at-risk for ACS.;Secondary Objective: To determine the pharmacokinetic profile of A-001 in SCD patients. To confirm the ability of A-001 infusion to suppress serum sPLA2 activity in SCD patients with elevated serum sPLA2. To determine the efficacy of A-001 infusion in preventing ACS in SCD patients with the combination of VOC, fever, and elevated serum sPLA2. To determine the efficacy of A-001 infusion in reducing pain during VOC. To select the dose(s) to carry forward into pivotal trials. ;Primary end point(s): Safety and tolerability of A-001 in SCD patients with VOC | — |
Countries
France, United Kingdom
Outcome results
None listed