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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Baxter Healthcare Corporation
ClinicalTrials.gov Identifier:
NCT01178294
First received: August 6, 2010
Last updated: February 28, 2014
Last verified: February 2014
  Purpose

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 acquired hemophilia A.


Condition Intervention Phase
Hemophilia A
Biological: OBI-1
Phase 2
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Efficacy and Safety of B-Domain Deleted Recombinant Porcine Factor VIII (OBI-1) in the Treatment of Acquired Hemophilia A Due to Factor VIII Inhibitory Auto-antibodies

Resource links provided by NLM:


Further study details as provided by Baxter Healthcare Corporation:

Primary Outcome Measures:
  • Proportion of serious bleeding episodes responsive to OBI-1 [ Time Frame: 24 hours after initiation of treatment ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Overall proportion of serious bleeding episodes successfully controlled with OBI-1 therapy, as assessed by the investigator. [ Time Frame: Through 90 days ± 7days following final OBI-1 dose ] [ Designated as safety issue: No ]
  • Proportion of bleeding episodes responsive to OBI-1 therapy at designated assessment time points after the initiation of therapy, as assessed by the investigator [ Time Frame: Through 90 days ± 7days following final OBI-1 dose ] [ Designated as safety issue: No ]
  • Frequency of infusions of OBI-1 required to successfully control qualifying bleeding episodes. [ Time Frame: Through 90 days ± 7days following final OBI-1 dose ] [ Designated as safety issue: No ]
  • Total dose of OBI-1 required to successfully control qualifying bleeding episodes. [ Time Frame: Through 90 days ± 7days following final OBI-1 dose ] [ Designated as safety issue: No ]
  • Total number of infusions of OBI-1 required to successfully control qualifying bleeding episodes [ Time Frame: Through 90 days ± 7days following final OBI-1 dose ] [ Designated as safety issue: No ]
  • Correlation between response to OBI-1 therapy at specified time points and eventual control of serious bleeding episodes. [ Time Frame: Through 90 days ± 7days following final OBI-1 dose ] [ Designated as safety issue: No ]
  • Correlation between the pre-infusion anti-OBI-1 antibody titers, the total dose of OBI-1, the outcome at 24 hours and the eventual control of the bleeding episode. [ Time Frame: Through 90 days ± 7days following final OBI-1 dose ] [ Designated as safety issue: No ]
  • Pharmacokinetics (PK) analysis- Plasma clearance [ Time Frame: Pre-infusion 15-20 minutes, Post-infusion 1, 3, 6, 12, 18, 24 hours ] [ Designated as safety issue: No ]
  • PK analysis- Volume of distribution (Vd) [ Time Frame: Pre-infusion 15-20 minutes, Post-infusion 1, 3, 6, 12, 18, 24 hours ] [ Designated as safety issue: No ]
  • PK analysis- Area under the concentration-time curve (AUC) [ Time Frame: Pre-infusion 15-20 minutes, Post-infusion 1, 3, 6, 12, 18, 24 hours ] [ Designated as safety issue: No ]
  • PK analysis- Incremental recovery [ Time Frame: Pre-infusion 15-20 minutes, Post-infusion 1, 3, 6, 12, 18, 24 hours ] [ Designated as safety issue: No ]
    Presented as maximum concentration/dose (Cmax/Dose)

  • Anti-human factor VIII antibody titer. [ Time Frame: Through 90 days ± 7days following final OBI-1 dose ] [ Designated as safety issue: Yes ]
  • Anti-OBI-1 antibody titer. [ Time Frame: Through 90 days ± 7days following final OBI-1 dose ] [ Designated as safety issue: Yes ]
  • Anti-host cell protein baby hamster kidney (BHK) antibody titer. [ Time Frame: Through 90 days ± 7days following final OBI-1 dose ] [ Designated as safety issue: Yes ]

Enrollment: 25
Study Start Date: November 2010
Study Completion Date: October 2013
Primary Completion Date: July 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: OBI-1 Biological: OBI-1
intravenous infusion, up to every 2-3 hours for the first 24 hours of treatment

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Main Inclusion Criteria:

  • Written informed consent from participant or their legal representative.
  • Participants with acquired hemophilia with autoimmune inhibitory antibodies to human factor VIII
  • Has a serious bleeding episode, as documented by the investigator.
  • Be willing and able to follow all instructions and attend all study visits.
  • Participants taking anti-thrombotics may be included depending on time elapsed since taking such medications
  • Life expectancy, before bleed, of at least 90 days.
  • Participants of reproductive age must use acceptable methods of contraception and if female, undergo pregnancy testing as part of the screening process.

Main Exclusion Criteria:

  • Hemodynamically unstable
  • Has an established reason for bleeding that is not correctable.
  • Bleeding episode assessed likely to resolve on its own if left untreated.
  • Anti-OBI-1 inhibitor that exceeds 20 Bethesda Units (BU)
  • Prior history of bleeding disorder other than acquired hemophilia.
  • Known major sensitivity to pig or hamster products.
  • Use of hemophilia medication within 3 to 6 (depending on medication) hours prior to OBI-1 administration
  • Participation in any other clinical study within 30 days of the first OBI-1 treatment.
  • Anticipated need for treatment or device during the study that may interfere with the evaluation
  • Is currently pregnant, breastfeeding, or planning to become pregnant or father a child during the study
  • Abnormal baseline findings
  • Inability or unwillingness to comply with the study design, protocol requirements, or the follow-up procedures.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01178294

  Show 23 Study Locations
Sponsors and Collaborators
Baxter Healthcare Corporation
Investigators
Study Director: Heinrich Farin, MD Baxter Healthcare Corporation
  More Information

No publications provided

Responsible Party: Baxter Healthcare Corporation
ClinicalTrials.gov Identifier: NCT01178294     History of Changes
Other Study ID Numbers: OBI-1-301
Study First Received: August 6, 2010
Last Updated: February 28, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Baxter Healthcare Corporation:
hemophilia A
haemophilia A
blood coagulation disorders
hemorrhagic disorders
coagulation protein disorder
hematologic diseases
Acquired Hemophilia A

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on August 25, 2014