Study of Modified Recombinant Factor VIII (OBI-1) in Subjects With Congenital Hemophilia A

Brief Title

Study of Modified Recombinant Factor VIII (OBI-1) in Subjects With Congenital Hemophilia A

Official Title

Efficacy and Safety of B-Domain Deleted Recombinant Porcine Factor VIII (OBI-1) in the Treatment of Patients With Congenital Hemophilia A With Factor VIII Inhibitors

Brief Summary

      This study is to test whether the study drug (OBI-1) is safe and effective for the treatment
      of serious bleeding episodes in people with congenital hemophilia A.

Study Phase

Phase 3

Study Type

Interventional

Primary Outcome

Proportion of Serious Bleeding Episodes Responsive to OBI-1

Secondary Outcome

 Overall Proportion of Serious Bleeding Episodes Successfully Controlled With OBI-1 Therapy, as Assessed by the Investigator.

Condition

Hemophilia A

Intervention

OBI-1

Study Arms / Comparison Groups

 OBI-1

Description:

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status

Biological

Estimated Enrollment

Start Date

August 2011

Completion Date

July 2013

Primary Completion Date

July 2013

Eligibility Criteria

        Inclusion Criteria:

          -  Written informed consent/assent from participant and/or participant's parent or legal
             representative.

          -  Participants with congenital hemophilia A with human factor VIII inhibitor ≤30 BU
             assessed within 90 days prior to study entry.

          -  Has previously or is currently demonstrating suboptimal hemostatic response to
             bypassing agents for treatment of bleeding episodes; suboptimal response is determined
             by the investigator , but minimally includes no or minimal evidence of response after
             at least two doses of bypassing agents, either for the current or a historic bleeding
             episode.

          -  Has an anti-OBI-1 titer ≤ 10 BU

          -  Has any serious or life-threatening bleeding episode; or requires a surgical procedure
             that could lead to a serious bleeding episode if not well controlled.

          -  Is willing and able to follow all instructions and attend all study visits.

          -  Has no other significant hemostatic abnormality and:

               -  Platelets ≥100,000/mm-cubed

               -  Prothrombin time < 15 seconds

               -  INR < 1.3

          -  Participants taking anti-thrombotics (such as clopidogrel, heparin or heparin
             analogue) may be included provided three half-lives of the agent have elapsed since
             the last dose of the agent.

        Exclusion Criteria:

          -  Hemodynamically unstable after blood transfusion, fluid resuscitation and
             pharmacologic or volume replacement pressor therapy. This hemodynamic instability is
             characterized by symptomatic hypotension resulting in vital organ dysfunction, such as
             cardiac ischemia, oliguria (urine volume <0.5 mL/kg in the previous six hours),
             central nervous system hypoperfusion manifested by mental status change such as
             confusion (unless head injury or intracranial hemorrhage is present), pulmonary
             compromise, and/or acidosis (manifested by pH and lactate levels).

          -  Bleeding episode assessed likely to resolve on its own if left untreated.

          -  Use of hemophilia medication: recombinant factor VIIa within 3 hours prior to OBI-1
             administration or activated prothrombin complex concentrate (aPCC) treatment within 6
             hours prior to OBI-1 administration

          -  Prior history of bleeding disorder other than congenital hemophilia A

          -  Known major sensitivity (anaphylactoid reactions) to porcine or hamster products.
             Examples include therapeutics of porcine origin (e.g. previously marketed porcine
             factor VIII, Hyate-C®) and recombinant therapeutics prepared from hamster cells (e.g.
             Humira®, Advate® and Enbrel®).

          -  Received any other investigational treatment within 30 days of the first OBI-1
             treatment.

          -  Anticipated need for treatment or device during the study that may interfere with the
             evaluation of the safety or efficacy of OBI-1, or whose safety or efficacy may be
             affected by OBI-1.

          -  Is planning to father a child during the study

          -  Has abnormal baseline findings, any other medical condition(s) or laboratory findings
             that, in the opinion of the investigator, might jeopardize the participant's safety or
             decrease the chance of obtaining satisfactory data needed to achieve the objectives of
             the study.

          -  Inability or unwillingness to comply with the study design, protocol requirements, or
             the follow-up procedures.

Gender

All

Ages

6 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Heinrich Farin, MD, ,

Location Countries

South Africa

Location Countries

South Africa

Administrative Informations

NCT ID

NCT01434511

Organization ID

OBI-1-302

Responsible Party

Sponsor

Study Sponsor

Baxalta now part of Shire

Study Sponsor

Heinrich Farin, MD, Study Director, Baxter Healthcare Corporation

Verification Date

December 2014