IMPACTS Trial: Investigation of the Modulation of Phospholipase in Acute Chest Syndrome

Anthera Pharmaceuticals30 enrolled

Overview

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.

This is a double-blind randomized, parallel group, placebo-controlled dose escalation study (2 cohorts) in patients with sickle cell disease (SCD) and vaso-occlusive crisis (VOC) who are at-risk for development of acute chest syndrome (ACS) based on the combination of VOC, fever (T ≥38.0ºC), and a serum sPLA2 concentration ≥50 ng/mL. The first group of patients will be randomized 2:1 to receive low dose A-001 or placebo as a 48-hour continuous infusion. Pharmacokinetic and clinical data from this group will undergo review by the Independent Data Monitoring Committee (IDMC). If there is no significant toxicity associated with A-001, then an additional group of patients will be enrolled and randomized 2:1 to high dose A-001 versus placebo as a 48-hour continuous infusion.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

PREVENTION

Masking

QUADRUPLE

Enrollment

Conditions

Sickle Cell Disease Vaso-occlusive Crisis Acute Chest Syndrome

Interventions

A-001DRUG

A-001

PlaceboOTHER

Placebo

Eligibility

Sex: ALLMin age: 5 Years

Medical Language ↔ Plain English

Patients are eligible for inclusion if they meet the following criteria: * Sickle cell variant (Hb SS, Hb SC, sickle β°-thalassemia, sickle β+-thalassemia) * Pain consistent with vaso-occlusive crisis * Elevated serum sPLA2 level (measured on-site) * Fever * Age ≥5 years (through adult) Patients must NOT meet any of the following exclusion criteria: * 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

Locations (6)

Howard University Hospital

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

Children's Healthcare of Atlanta

Atlanta, Georgia, United States

Children's Memorial Hospital

Chicago, Illinois, United States

SUNY Downstate Medical Center

Brooklyn, New York, United States

Outcomes

Primary Outcomes

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).

Time frame: Study end

Secondary Outcomes

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

Time frame: Study end

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

Time frame: 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

Time frame: Study end

Data from ClinicalTrials.gov

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