Trial record 1 of 1 for:    NCT00628498
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Defibrotide for Patients With Hepatic Veno-occlusive Disease: A Treatment IND Study

This study is currently recruiting participants. (see Contacts and Locations)
Verified January 2014 by Gentium SpA
Sponsor:
Information provided by (Responsible Party):
Gentium SpA
ClinicalTrials.gov Identifier:
NCT00628498
First received: February 25, 2008
Last updated: January 13, 2014
Last verified: January 2014

February 25, 2008
January 13, 2014
December 2007
December 2014   (final data collection date for primary outcome measure)
Complete response of VOD [ Time Frame: D+100 from SCT or 100 days from start of chemotherapy ] [ Designated as safety issue: No ]
Complete response of VOD [ Time Frame: D+100 from SCT ] [ Designated as safety issue: No ]
Complete list of historical versions of study NCT00628498 on ClinicalTrials.gov Archive Site
Survival [ Time Frame: D+100 post SCT or 100 days from start of chemotherapy ] [ Designated as safety issue: No ]
Survival [ Time Frame: D+100 ]
Number of Participants with Adverse Events [ Time Frame: From date study critera were met through 30 days from last Defibrotide dose ] [ Designated as safety issue: Yes ]
Not Provided
 
Defibrotide for Patients With Hepatic Veno-occlusive Disease: A Treatment IND Study
Defibrotide for Patients With Hepatic Veno-occlusive Disease: A Treatment IND Study (Under CFR 312.34)

Single arm, open-label study to provide Defibrotide to patients diagnosed with VOD. Defibrotide is no longer available though the Emergency Use IND mechanism (also known as compassionate use, or single patient named use). This protocol is the only mechanism by which Defibrotide can be made available to patients in the U.S.

Not Provided
Interventional
Phase 3
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Hepatic Veno-Occlusive Disease
  • Drug: Defibrotide

    Defibrotide is made up of a complex mixture of single stranded oligodeoxyribonucleotides having variable base sequences, chain lengths and molecular weights which form unfolded, folded or combined conformations. Defibrotide has a complex mechanism of action with antithrombotic, anti-ischemic, anti-inflammatory, anti-adhesive and thrombolytic properties but no significant systemic anti-coagulant effects.

    Defibrotide is dose intravenously as a 2-hour infusion every 6 hours at a dose of 25 mg/kg/day. Recommended duration of therapy is 21 days.

  • Drug: Defibrotide
Experimental: Defibrotide
Defibrotide 25 mg/kg day given in 4 divided doses approximately every 6 hours
Interventions:
  • Drug: Defibrotide
  • Drug: Defibrotide
Richardson PG, Murakami C, Jin Z, Warren D, Momtaz P, Hoppensteadt D, Elias AD, Antin JH, Soiffer R, Spitzer T, Avigan D, Bearman SI, Martin PL, Kurtzberg J, Vredenburgh J, Chen AR, Arai S, Vogelsang G, McDonald GB, Guinan EC. Multi-institutional use of defibrotide in 88 patients after stem cell transplantation with severe veno-occlusive disease and multisystem organ failure: response without significant toxicity in a high-risk population and factors predictive of outcome. Blood. 2002 Dec 15;100(13):4337-43. Epub 2002 Aug 1.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
600
Not Provided
December 2014   (final data collection date for primary outcome measure)

Inclusion Criteria:

Entry criteria include the following:

  1. Clinical diagnosis of VOD, made by Baltimore Criteria, Modified Seattle Criteria, or biopsy proven:

    1.1 Baltimore Criteria- Bilirubin ≥2 mg/dL and at least 2 of the following clinical findings:

    • Ascites (radiographic or physical exam)
    • Weight gain of ≥5% compared to the day of conditioning-- if this value is not available, the weight on the date of admission to the SCT unit may be used)
    • Hepatomegaly; increased over baseline.

    1.2 Modified Seattle Criteria: At least two of the following

    • Bilirubin ≥2 mg/dL
    • Ascites (radiographic or physical exam) and/or weight gain ≥5% above baseline weight (defined as weight on the first day of conditioning- if this value is not available, the weight on the date of admission to the SCT unit may be used)
    • hepatomegaly increased over baseline

    1.3 Patients that do not meet the Baltimore Criteria or Modified Seattle Criteria and have biopsy proven VOD are eligible.

  2. Patient must also provide written informed consent.

Exclusion Criteria:

  • Use of any medication which increases the risk of hemorrhage is disallowed. Use of heparin or other anticoagulants is disallowed within 12 hours unless being used for routine central venous line management, fibrinolytic instillation for central venous line occlusion, intermittent dialysis or ultrafiltration of CVVH.
  • Clinically significant uncontrolled acute bleeding, defined as hemorrhage requiring > 15 cc/kg of packed red blood cells (e.g., a pediatric patient weighing 20 kg and requiring > 300cc of packed red blood cells/24 hours, or an adult patient weighing 70 kg and requiring >3 units of packed red blood cells/24 hours) to replace blood loss, OR bleeding from a site which in the Investigator's opinion constitutes a potential life-threatening source (e.g. pulmonary hemorrhage or CNS bleeding), irrespective of amount of blood loss, at any point from the date of SCT through the date of severe VOD diagnosis.
  • Hemodynamic instability as defined by a requirement for multiple pressors, or inability to maintain mean arterial pressure (for children: to maintain mean arterial pressure within 1 standard deviation of age-adjusted levels) with single pressor support.
  • Woman who are pregnant.
Both
Not Provided
No
Contact: Lam Calderon 1.312.706.6240 0265-002Gentium@iconplc.com
Contact: William E Gannon, Jr.,, M.D. 1.202.548.4930 medicalmonitor@gentium.it
United States
 
NCT00628498
P2006-05
No
Gentium SpA
Gentium SpA
Not Provided
Study Director: Carin Heringa, M.D. Gentium, S.p.A.
Principal Investigator: Paul Richardson, M.D. Dana-Farber Cancer Institute
Gentium SpA
January 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP