Trial Information

Summary: A Phase II/III Pivotal trial evaluating the safety and pharmacokinetic properties of a plasma derived antithrombin III concentrate with administration for surgery, pregnancy and serious thromboembolic or thrombotic events

ATIII Deficiency is a blood disorder that results in a higher chance of forming clots in the blood vessels (thrombosis). ATIII is a protein in blood that limits the blood's ability to clot (coagulation). In people with Congenital ATIII Deficiency, there is a reduced amount of this substance in the blood due to a genetic abnormality from birth. This is known as thrombophilia.

This study uses an investigational product called ATIII-DAF/DI. ATIII-DAF/DI has been approved in several European countries but not yet by the United States Food and Drug Administration (FDA) and its use is experimental in this study. ATIII-DAF/DI is made from ATIII taken from human plasma.

Subject Inclusion Criteria
In order to qualify for entrance into the study, each subject must satisfy all criteria listed below.

• Congenital ATIII deficiency documented by determination of plasma levels of ATIII off all therapies. Specifically, the baseline levels of ATIII activity should be equal to or less than 60%.
• Age > 12 years with a body weight of no less than 30 kg.
• Have not participated in another investigational study for at least 30 days
• For Segment II,enrollment requires a pregnancy/delivery or a surgical procedure (it should be a major surgery although data from a minor surgery will also be collected).
• Documented personal history of major thromboembolic or thrombotic event.
• HIV, HBV, HCV, HAV and PARVO B19 status known prior to entry.
• The subject is willing to comply with all aspects of the protocol, including blood sampling, for the duration of the study.
• The subject has signed an informed consent form (if at least 18 years old), or the subject’s parent or legal guardian has signed the informed consent form.
  Subjects below the age of 18 years will also be asked to sign an assent form. All consent and assent forms must be approved in advance by the Institutional
  Review Board of the investigator’s institution.
• Patients with heparin-associated thrombocytopenia who require anticoagulation with non-heparin containing drugs will be eligible if they can be safely transitioned during the washout period for the Segment I PK study.
• If pregnant, a woman must be Parvo B19 IgG antibody positive.

Subject Exclusion Criteria
Patients will be excluded if any of the following exclusion criteria are met.

• Acquired deficiency of ATIII
• Receiving concomitant treatment for thrombophilic disorders other than ATIII deficiency
• History of anaphylactic reaction(s) to blood or blood components
• Allergies to excipients.
• Liver function tests = 2.5 X ULN
• Serum creatinine > 1.2 X ULN
• Urine = 2+ protein with urine dipstick test.
• The subject is known to have abused alcohol or illicit drugs within the past 12 months.
• The subject is unlikely to adhere to the protocol requirements of the study or is likely to be uncooperative or unable to provide a storage serum sample at the screening visit.
• Patients on heparin-treatment who, for clinical reasons, cannot safely be discontinued from heparin therapy during the PK segment .

If you would like to learn more about participating in this study, please send an e-mail message using the form below.