- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01434511
Study of Modified Recombinant Factor VIII (OBI-1) in Subjects With Congenital Hemophilia A
April 17, 2021 updated by: Baxalta now part of Shire
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
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 Overview
Study Type
Interventional
Enrollment (Actual)
1
Phase
- Phase 3
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Gauteng
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Johannesburg, Gauteng, South Africa, 2193
- Charlotte Maxeke Johannesburg Academic Hospital
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England
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London, England, United Kingdom, WC1N 3JH
- Great Ormond Street Hospital
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Indiana
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Indianapolis, Indiana, United States, 46260
- Indiana Hemophilia and Thrombosis Center
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
6 years and older (ADULT, OLDER_ADULT, CHILD)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: NA
- Interventional Model: SINGLE_GROUP
- Masking: NONE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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EXPERIMENTAL: OBI-1
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intravenous infusion, up to every 2-3 hours for the first 24 hours of treatment
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Proportion of Serious Bleeding Episodes Responsive to OBI-1
Time Frame: 24 hours after initiation of treatment
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This study was terminated early and only enrolled one participant.
Due to concerns that the participant would be at risk of being re-identified, the study results are not posted.
The decision to terminate this study was not related to any safety and/or efficacy concern of OBI-1 in the indication described within the OBI-1-302 study (Congenital Hemophilia A).
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24 hours after initiation of treatment
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Secondary Outcome Measures
Outcome Measure |
Time Frame |
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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
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Through 90 days ± 7days following final OBI-1 dose
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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
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Through 90 days ± 7days following final OBI-1 dose
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Frequency of Infusions of OBI-1 Required to Successfully Control Qualifying Bleeding Episodes.
Time Frame: Through 90 days ± 7days following final OBI-1 dose
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Through 90 days ± 7days following final OBI-1 dose
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Total Dose of OBI-1 Required to Successfully Control Qualifying Bleeding Episodes.
Time Frame: Through 90 days ± 7days following final OBI-1 dose
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Through 90 days ± 7days following final OBI-1 dose
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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
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Through 90 days ± 7days following final OBI-1 dose
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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
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Through 90 days ± 7days following final OBI-1 dose
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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: Frame: Through 90 days ± 7days following final OBI-1 dose
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Frame: Through 90 days ± 7days following final OBI-1 dose
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Correlation Between the Pre-infusion Anti-OBI-1 Antibody Titers and the Recovery of OBI-1.
Time Frame: Through 90 days ± 7days following final OBI-1 dose
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Through 90 days ± 7days following final OBI-1 dose
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Recovery and Elimination Rate Parameters of OBI-1 in Subjects With Inhibitors Treated With OBI-1 Therapy.
Time Frame: Through 90 days ± 7days following final OBI-1 dose
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Through 90 days ± 7days following final OBI-1 dose
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Efficacy Assessment of OBI-1 in Participants With Anti-human Factor VIII Titers >30 Bethesda Units (BU)
Time Frame: Through 90 days ± 7days following final OBI-1 dose
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Through 90 days ± 7days following final OBI-1 dose
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Anti-human Factor VIII Antibody Titer.
Time Frame: Through 90 days ± 7days following final OBI-1 dose
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Through 90 days ± 7days following final OBI-1 dose
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Anti-OBI-1 Antibody Titer.
Time Frame: Through 90 days ± 7days following final OBI-1 dose
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Through 90 days ± 7days following final OBI-1 dose
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Anti-host Cell Protein Baby Hamster Kidney (BHK) Antibody Titer.
Time Frame: Through 90 days ± 7days following final OBI-1 dose
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Through 90 days ± 7days following final OBI-1 dose
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (ACTUAL)
October 3, 2011
Primary Completion (ACTUAL)
July 29, 2013
Study Completion (ACTUAL)
July 29, 2013
Study Registration Dates
First Submitted
September 13, 2011
First Submitted That Met QC Criteria
September 13, 2011
First Posted (ESTIMATE)
September 15, 2011
Study Record Updates
Last Update Posted (ACTUAL)
May 14, 2021
Last Update Submitted That Met QC Criteria
April 17, 2021
Last Verified
April 1, 2021
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- OBI-1-302
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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Clinical Trials on OBI-1
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OBI Pharma, IncTerminatedPancreatic Adenocarcinoma | Solid TumorUnited States
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OBI Pharma, IncTerminatedLocally Advanced Solid TumorUnited States
-
Octagen CorporationBiomeasure Inc, Ipsen GroupCompletedHemophilia AUnited States
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Baxalta now part of ShireNo longer available
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OBI Pharma, IncTerminatedLocally Advanced Solid Tumors | Metastatic Solid TumorsUnited States, Taiwan