Just what is a pilot study probe study?
"This pilot study is the first in a series of potential probe studies XOMA is considering to explore various indications for NEUPREX,"
Xoma has not and probably will not explain what this means nor the implications. The entire p.r. refers to P I, P II clinical trials.
Press Release Source: XOMA Ltd.
XOMA and Children's Medical Center Dallas Announce Initiation of Clinical Trial with NEUPREX -- rBPI-21 -- in Pediatric Open Heart Surgery Patients
Thursday October 2, 7:30 am ET
BERKELEY, Calif. and DALLAS--(BUSINESS WIRE)--Oct. 2, 2003--XOMA Ltd. (Nasdaq:XOMA - News) and Children's Medical Center Dallas announced today the initiation of an open-label, single center, dose escalating, investigator-sponsored, Phase I/II clinical trial of NEUPREX® (rBPI-21) in pediatric patients with congenital heart abnormalities requiring open heart surgery (OHS) associated with cardiopulmonary bypass (CPB). NEUPREX is an injectable formulation of a modified recombinant fragment of BPI (bactericidal/permeability-increasing protein), a naturally occurring human host-defense protein.
Pediatric patients undergoing OHS associated with CPB have a higher rate of serious post-operative inflammatory complications than adults. These complications, which affect the heart, lungs, kidney and central nervous system, have been associated with increased blood levels of endotoxin. NEUPREX's endotoxin-neutralizing mechanism of action could potentially be important for limiting the post-operative complications following OHS associated with CPB. The study plans to investigate dosing, efficacy endpoints and safety to assess the potential for conducting larger, additional studies.
"Congenital heart disease remains the leading cause of death among all congenital defects, with hundreds of lives lost and even more children left with disabilities each year in the U.S.," said Dr. Erica Molitor-Kirsch, Attending Physician, Critical Care Services and lead investigator of the study. "This pilot study exploring the use of NEUPREX in pediatric patients undergoing cardiopulmonary bypass could be an important step in reducing the mortality and serious complications associated with this pediatric patient population. Improving the outcome of these children could also potentially decrease the cost of care, which exceeds $2.2 billion for inpatient surgery alone in the U.S."
"This pilot study is the first in a series of potential probe studies XOMA is considering to explore various indications for NEUPREX," said John L. Castello, XOMA's chairman, president and chief executive officer. "We believe NEUPREX has established a promising safety and activity profile in clinical trials involving over 2000 patients in a variety of indications. We're pleased that Children's Medical Center Dallas physicians also believe that it could potentially be a beneficial treatment for pediatric patients undergoing extensive surgical interventions and look forward to reviewing the results following the study's completion."
About Congenital Heart Disease, Open Heart Surgery (OHS) and
Cardiopulmonary Bypass (CPB)
Every year, an estimated 35,000 babies are born with some form of congenital heart disease. More than twice as many children die from congenital heart disease in the U.S. each year than from childhood cancers. Approximately one-third of children born with congenital heart disease will require surgery in the first year of life. The majority, more than 6,000 infants each year, will require complex open heart surgery (OHS) which necessitates the use of cardiopulmonary bypass (CPB), a heart-lung bypass machine.
During CPB, the heart-lung machine takes over the functions of the heart and lungs (which have been cooled down and stopped), allowing a surgeon to operate on the heart while the machine sustains circulation. However, CPB itself poses significant risks which are particularly prominent in pediatric patients. CPB induces a systemic inflammatory response associated with increasing blood levels of endotoxin and causes damage to children's immature organ systems including the heart, lungs, kidneys and brain. The incidence of complications associated with pediatric OHS patients undergoing CPB ranges from 25-60 percent, with the inflammatory response to CPB causing a prolonged need for mechanical ventilation and heart medications, and additionally bleeding complications and multiple organ failure. All of these post-operative complications can impair patient recovery, resulting in extended hospital stays and increased medical expenses.
About NEUPREX (rBPI-21)
NEUPREX is an injectable formulation of rBPI-21, a modified recombinant fragment of BPI (bactericidal/permeability-increasing protein). BPI is a human host-defense protein made by neutrophils -- a type of white blood cell. NEUPREX has been tested in a number of disease indications, including in pediatric patients suffering from severe meningococcemia.
NEUPREX kills gram-negative bacteria, enhances the activity of antibiotics, neutralizes endotoxin (a poisonous molecule in the cell walls of gram-negative bacteria that can trigger local and systemic inflammatory reactions in the human host) and inhibits inflammation.
These characteristics underlie the rationale for testing rBPI-21 in multiple infectious and inflammatory disease indications, including a phase III study in severe pediatric meningococcemia (also known as meningococcal sepsis) patients.
XOMA's BPI-related patent portfolio presently includes more than 75 issued US patents. This portfolio also includes corresponding foreign patents and applications, and encompasses claims for novel compounds and compositions, manufacturing methods, formulations and therapeutic uses of BPI protein products, as well as a number of patents exclusively licensed from New York University and Incyte Pharmaceuticals, Inc. |
