Hemophilia APhase 3NCT01434511
Study of Modified Recombinant Factor VIII (OBI-1) in Subjects With Congenital Hemophilia A
A Phase 3 study testing OBI-1 in people with Hemophilia A, run by Baxalta now part of Shire at 3 sites in 3 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Hemophilia A
- What is being tested?
- OBI-1
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 3 sitesin 3 countries
- Study dates
- 3 Oct 2011to 29 Jul 2013
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Show all 11Show fewer
- 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.
Who cannot join
- 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.
Show all 10Show fewer
- 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.
Trial locations and contacts
3 sites in 3 countries.
3 sites in 3 countries
United States1 site
Indiana Hemophilia and Thrombosis Center
Indianapolis, Indiana, 46260
South Africa1 site
Charlotte Maxeke Johannesburg Academic Hospital
Johannesburg, Gauteng, 2193
United Kingdom1 site
Great Ormond Street Hospital
London, England, WC1N 3JH
About this study
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
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.
- Conditions
- Hemophilia A
- Being tested
- OBI-1
- Keywords
- hemophilia A, haemophilia A, blood coagulation disorders, hemorrhagic disorders, coagulation protein disorder, hematologic diseases, congenital hemophilia A
- Medical subject headings
- Hemophilia A, Blood Coagulation Disorders, Hemorrhagic Disorders, Coagulation Protein Disorders, Hematologic Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OBI-1Biological
intravenous infusion, up to every 2-3 hours for the first 24 hours of treatment
| Study group | Role | What they receive |
|---|---|---|
| OBI-1 | Experimental | Not described Receives: Biological: OBI-1 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of Serious Bleeding Episodes Responsive to OBI-1
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).
Measured: 24 hours after initiation of treatment
Secondary outcomes (13)
Overall Proportion of Serious Bleeding Episodes Successfully Controlled With OBI-1 Therapy, as Assessed by the Investigator.
Measured: Through 90 days ± 7days following final OBI-1 dose
Proportion of Bleeding Episodes Responsive to OBI-1 Therapy at Designated Assessment Time Points After the Initiation of Therapy, as Assessed by the Investigator
Measured: Through 90 days ± 7days following final OBI-1 dose
Frequency of Infusions of OBI-1 Required to Successfully Control Qualifying Bleeding Episodes.
Measured: Through 90 days ± 7days following final OBI-1 dose
Total Dose of OBI-1 Required to Successfully Control Qualifying Bleeding Episodes.
Measured: Through 90 days ± 7days following final OBI-1 dose
Total Number of Infusions of OBI-1 Required to Successfully Control Qualifying Bleeding Episodes.
Measured: Through 90 days ± 7days following final OBI-1 dose
Correlation Between Response to OBI-1 Therapy at Specified Time Points and Eventual Control of Serious Bleeding Episodes.
Measured: Through 90 days ± 7days following final OBI-1 dose
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.
Measured: Frame: Through 90 days ± 7days following final OBI-1 dose
Correlation Between the Pre-infusion Anti-OBI-1 Antibody Titers and the Recovery of OBI-1.
Measured: Through 90 days ± 7days following final OBI-1 dose
Recovery and Elimination Rate Parameters of OBI-1 in Subjects With Inhibitors Treated With OBI-1 Therapy.
Measured: Through 90 days ± 7days following final OBI-1 dose
Efficacy Assessment of OBI-1 in Participants With Anti-human Factor VIII Titers >30 Bethesda Units (BU)
Measured: Through 90 days ± 7days following final OBI-1 dose
Anti-human Factor VIII Antibody Titer.
Measured: Through 90 days ± 7days following final OBI-1 dose
Anti-OBI-1 Antibody Titer.
Measured: Through 90 days ± 7days following final OBI-1 dose
Anti-host Cell Protein Baby Hamster Kidney (BHK) Antibody Titer.
Measured: Through 90 days ± 7days following final OBI-1 dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Sep 2011 (estimated)
- Study start3 Oct 2011
- Primary completion29 Jul 2013
- Study completion29 Jul 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baxalta now part of Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01434511
- Sponsor's protocol ID
- OBI-1-302
- Status last verified
- Apr 2021
- First submitted
- 13 Sep 2011
- Last updated
- 14 May 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 14 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01434511
- Acronym / sponsor ID
- OBI-1-302
- Conditions
- Hemophilia A
- Keywords
- hemophilia A, haemophilia A, blood coagulation disorders, hemorrhagic disorders, coagulation protein disorder, hematologic diseases, congenital hemophilia A
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Baxalta now part of ShireIndustry (pharma, biotech, devices)
- Study start
- 3 Oct 2011 (actual)
- Primary completion
- 29 Jul 2013 (actual)
- Study completion
- 29 Jul 2013 (actual)
- First posted
- 15 Sep 2011 (estimated)
- Results
- Posted 17 Dec 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| OBI-1 | Experimental | Biological: OBI-1 |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (11 listed)
- 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.
Key exclusion criteria (10 listed)
- 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®).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| OBI-1intravenous infusion, up to every 2-3 hours for the first 24 hours of treatment | Biological | OBI-1 |
Primary outcome
Proportion of Serious Bleeding Episodes Responsive to OBI-1
Time frame: 24 hours after initiation of treatment
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).
13 secondary outcomes
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
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
Frequency of Infusions of OBI-1 Required to Successfully Control Qualifying Bleeding Episodes.
Time frame: Through 90 days ± 7days following final OBI-1 dose
Total Dose of OBI-1 Required to Successfully Control Qualifying Bleeding Episodes.
Time frame: Through 90 days ± 7days following final OBI-1 dose
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
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
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
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
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
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
Anti-human Factor VIII Antibody Titer.
Time frame: Through 90 days ± 7days following final OBI-1 dose
Anti-OBI-1 Antibody Titer.
Time frame: Through 90 days ± 7days following final OBI-1 dose
Anti-host Cell Protein Baby Hamster Kidney (BHK) Antibody Titer.
Time frame: Through 90 days ± 7days following final OBI-1 dose
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 May 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Hemophilia A
RecruitingObservational study
The CACHE Study: Coronary Artery Care in HaEmophilia
Haemophilia, Cardiovascular Diseases
Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
RecruitingPhase 1/2
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Hemophilia A
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
Takeda
3 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing OBI-1 in people with Hemophilia A, run by Baxalta now part of Shire at 3 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 14 May 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States, South Africa, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 17 Dec 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of Serious Bleeding Episodes Responsive to OBI-1 (time frame: 24 hours after initiation of treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 May 2021.