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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00434473
Recruitment Status : Completed
First Posted : February 13, 2007
Last Update Posted : March 4, 2014
Information provided by (Responsible Party):
Anthera Pharmaceuticals

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Vaso-occlusive Crisis Acute Chest Syndrome Drug: A-001 Other: Placebo Phase 2

Detailed Description:

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  (Clinical Trial)
Actual Enrollment : 30 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Prevention
Official Title: IMPACTS Trial: Investigation of the Modulation of Phospholipase in Acute Chest Syndrome (Dose Escalation Study: Varespladib Infusion [A-001] for the Prevention of Acute Chest Syndrome in At-Risk Patients With Sickle Cell Disease and Vaso-occlusive Crisis)
Study Start Date : December 2006
Actual Primary Completion Date : August 2009
Actual Study Completion Date : December 2009

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Placebo Comparator: Placebo Other: Placebo

Experimental: A-001 Drug: A-001

Primary Outcome Measures :
  1. 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 Outcome Measures :
  1. To determine the pharmacokinetic profile of A-001 in SCD patients [ Time Frame: Study end ]
  2. To confirm the ability of A-001 infusion to suppress serum sPLA2 activity in SCD patients with elevated serum sPLA2 [ Time Frame: Study end ]
  3. 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 ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Ages Eligible for Study:   5 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00434473

United States, District of Columbia
Howard University Hospital
Washington, District of Columbia, United States, 20060
United States, Georgia
Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30342
United States, Illinois
Children's Memorial Hospital
Chicago, Illinois, United States, 60614
United States, New York
SUNY Downstate Medical Center
Brooklyn, New York, United States, 11203
United States, North Carolina
Duke University Comprehensive Sickle Cell Center
Durham, North Carolina, United States, 27713
United States, Virginia
Virginia Commonwealth University
Richmond, Virginia, United States, 23219
Sponsors and Collaborators
Anthera Pharmaceuticals

Responsible Party: Anthera Pharmaceuticals Identifier: NCT00434473     History of Changes
Other Study ID Numbers: AN-SCD1121
First Posted: February 13, 2007    Key Record Dates
Last Update Posted: March 4, 2014
Last Verified: January 2014

Additional relevant MeSH terms:
Anemia, Sickle Cell
Acute Chest Syndrome
Pathologic Processes
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn
Lung Diseases
Respiratory Tract Diseases
Respiration Disorders