- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00245245
Study of Recombinant Porcine Factor VIII (FVIII) in Hemophilia and Inhibitors to FVIII
An Open-Label Study of the Hemostatic Activity, Pharmacokinetics and Safety of OBI-1 (B-Domain Deleted Recombinant Porcine FVIII), When Administered by Intravenous Injection, to Control Non-Life and Non-Limb Threatening Bleeding Episodes in Congenital Hemophilia A Patients With an Inhibitor to Human FVIII
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
The primary objective of the study is to evaluate the hemostatic activity of OBI-1 to control a bleeding episode in hemophilia A patients with inhibitors who are experiencing a non-life and/or non-limb threatening bleeding episode.
The secondary objectives of this study will be to assess the:
- safety of OBI-1,
- serial anti-OBI-1 and anti-human factor VIII (fVIII) inhibitor antibody responses following therapeutic administration of OBI-1, and
- pharmacokinetics of OBI-1 administered to control a bleeding episode.
After qualifying for the study at a Screening visit, patients will come to the investigator for treatment of a qualifying bleeding episode. After baseline blood samples are taken for inhibitor titer, a Loading Dose of OBI-1, calculated to inactivate the inhibitor, will be administered intravenously. After a blood sample is drawn to measure inhibitor titer and fVIII level, a Treatment Dose of 50 U/kg is administered. Serial blood samples will be drawn for fVIII levels for pharmacokinetics. At 6-hour intervals, additional Treatment doses, at increasing dose levels up to 150 U/kg, may be given if needed. Follow-up safety evaluations, beginning at Day 14, are scheduled; inhibitor titers against human fVIII and OBI-1 will be measured to assess continuing eligibility.
A second qualifying bleeding episode will be treated as the first, but without pharmacokinetic samples. If the patient's inhibitor titer against OBI-1 rises to > 20 Bu, further treatments with OBI-1 will be suspended until the titer decreases to 20 Bu or lower. Third and subsequent treatment episodes may be self-administered by the patient, under strict supervision of the investigator, in home care.
The study will continue until at least 12 patients have received at least 24 treatments, unless the Data Safety Monitoring Committee (DSMC) recommends a change in dosing calculations sooner.
Study Type
Enrollment (Anticipated)
Phase
- Phase 2
Contacts and Locations
Study Locations
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Pennsylvania
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Blue Bell, Pennsylvania, United States, 19422
- Octagen Corporation
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
Patients must meet at least the following criteria to participate in the trial:
- Age at least 12 years.
- Clinical diagnosis of congenital hemophilia A with current inhibitor to human fVIII OR the patient is known to have developed an anti-human fVIII inhibitor antibody anamnestic response to human fVIII in the past.
- OBI-1 inhibitor antibody titer < 20 Bethesda Units at screening.
- Uncomplicated joint or soft tissue bleed, or other non-life threatening or non-limb threatening bleeding episode.
Exclusion Criteria:
Patients will be ineligible to participate if any of the following are present:
- Current treatment plan for any acute bleeding episode incorporates the use of human fVIII (recombinant or plasma-derived).
- Presence of any life- or limb-threatening bleeding episode (defined)
- Patient has received any human fVIII or prothrombin complex concentrate (PCC), within 7 days prior to Screening, OR received any PCC within 7 days prior to treatment with OBI-1.
- Patient has received recombinant human fVIIa (rVIIa) within 3 days prior to Screening OR within 3 days prior to treatment with OBI-1.
- Significant liver disease or renal disease
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
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The percentage of successful treatment episodes, defined as having achieved control of the bleeding episode within One Loading Dose and 8 or fewer Treatment Doses, with a dose limit of 1000 U/kg in 24 hours
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Secondary Outcome Measures
Outcome Measure |
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Adverse events and serious adverse events observed throughout course of study
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Pattern of inhibitory antibody response to OBI-1 following treatments
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The percentage of patients who continue to qualify because their anti-OBI-1 titer remains at 20 Bu or less
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Pharmacokinetics of OBI-1 when it is administered for treatment of a qualifying bleeding episode, in the absence of an inhibitory antibody to OBI-1
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Collaborators and Investigators
Sponsor
Collaborators
Investigators
- Principal Investigator: Josef N Mueksch, MD, MBA, Octagen Corporation
Study record dates
Study Major Dates
Study Start
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- OBI-1 - 201
- NIH Grant 2R44 HL064497-02
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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