Health & Fitness
Health and Fitness
Health and Fitness
Fri, January 23, 2009
Thu, January 22, 2009 Gentium Announces Completion of Enrollment for the Phase II/III European Pediatric Prevention Trial of Defibrotide
VILLA GUARDIA (COMO), Italy--([ BUSINESS WIRE ])--Gentium S.p.A. (Nasdaq: GENT) announced today the completion of patient enrollment in a Phase II/III European, multi-center, open label, randomized trial to evaluate the prophylactic use of Defibrotide in pediatric patients undergoing stem cell transplantation who are at high risk for hepatic veno-occlusive disease (VOD).
In the Phase II/III trial, patients are randomly assigned to one of two treatment arms. Those allocated to the Defibrotide prophylaxis arm receive 25mg/kg/day in four divided doses beginning at the time of conditioning and finishing 30 days after stem cell transplantation (SCT) or upon discharge from inpatient care. Patients allocated to the control arm do not receive VOD prophylactic measures. The primary efficacy endpoint for the trial is the incidence of VOD within 30 days after SCT. The secondary safety endpoints include the occurrence of multi-system organ failure and survival at 100 days after SCT. Additional information on this trial can be found at [ www.clinicaltrials.gov ].
Gentium plans to release initial results of this trial at the Annual Meeting of the European Group for Blood and Marrow Transplantation, March 29 to April 1, 2009 in Göteborg, Sweden. As was previously announced, following discussions with the European Medicines Agency, Gentium has been notified of the potential for an accelerated review of Defibrotide in the pediatric prevention indication.
"We are excited to have completed enrollment in our European Phase II/III prophylaxis trial for Defibrotide in pediatric patients," stated Dr. Laura Ferro, CEO of Gentium S.p.A. "Prior investigator sponsored trials for Defibrotide in the prevention setting have shown promising results, and we are hopeful that this trial will fall in line with what we have previously seen."
About VOD
Veno-occlusive disease is a potentially life-threatening condition, which typically occurs as an important complication of stem cell transplantation. Certain high-dose chemo-radiation therapy regimens used as part of SCT can damage the lining cells of hepatic blood vessels and so result in VOD, a blockage of the small veins of the liver that leads to liver failure and can result in significant dysfunction in other organs such as the kidneys and lungs (so-called severe VOD). SCT is a frequently used treatment modality following high-dose chemotherapy and radiation therapy for hematologic cancers and other conditions in both adults and children. There is currently no approved agent for the treatment or prevention of VOD in the U.S. or the EU.
About Gentium
Gentium S.p.A. is a biopharmaceutical company focused on the research, discovery and development of drugs derived from DNA extracted from natural sources, and drugs that are synthetic derivatives, to treat and prevent a variety of vascular diseases and conditions related to cancer and cancer treatments. Defibrotide, the Company's lead product candidate, is an investigational drug that has been granted Orphan Drug status by the U.S. Food and Drug Administration and EMEA to prevent and to treat VOD and Fast Track designation by the U.S. FDA for the treatment of severe VOD in recipients of stem cell transplants.
Cautionary Note Regarding Forward-Looking Statements
This press release contains "forward-looking statements." In some cases, you can identify these statements by forward-looking words such as "may," "might," "will," "should," "expect," "plan," "anticipate," "believe," "estimate," "predict," "potential" or "continue," the negative of these terms and other comparable terminology. These statements are not historical facts but instead represent the Company's belief regarding future results, many of which, by their nature, are inherently uncertain and outside the Company's control. It is possible that actual results, including clinical trial results and regulatory reviews, may differ materially from those anticipated in these forward-looking statements. For a discussion of some of the risks and important factors that could affect future results, see the discussion in our Form 20F filed with the Securities and Exchange Commission under the caption "Risk Factors."
