Hemophilia APhase 2NCT00245245
Study of Recombinant Porcine Factor VIII (FVIII) in Hemophilia and Inhibitors to FVIII
A Phase 2 study testing recombinant porcine coagulation factor VIII (OBI-1) in people with Hemophilia A, run by Octagen Corporation at 1 site in United States.
Trial summary
The ability of a new recombinant porcine coagulation factor VIII, B-domain deleted (called "OBI-1"), to control the non-life- or limb-threatening bleeding episodes patients with hemophilia A commonly develop is being evaluated. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Hemophilia A
- What is being tested?
- recombinant porcine coagulation factor VIII (OBI-1)
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- May 2005to Jun 2007
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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
The ability of a new recombinant porcine coagulation factor VIII, B-domain deleted (called "OBI-1"), to control the non-life- or limb-threatening bleeding episodes patients with hemophilia A commonly develop is being evaluated. Patients with congenital hemophilia A and a low-titer (<20 Bethesda units [Bu]) inhibitory antibody to OBI-1, who meet the inclusion/exclusion criteria, will receive OBI-1 to treat their soft tissue or joint bleeding episode. At least the first two treatment episodes will be performed in the controlled setting of the hemophilia center/clinic/office, where any side effects can be observed. If the patient continues to meet the inclusion/exclusion criteria, has had no serious or severe adverse reactions to OBI-1, and has been in a home care program, the investigator may permit the patient to self-administer OBI-1 at home to treat subsequent bleeding episodes. The study will continue at least until 12 or more patients have received at least 24 treatment episodes in the aggregate.
Read the 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.
- Conditions
- Hemophilia A
- Being tested
- recombinant porcine coagulation factor VIII (OBI-1)
- Keywords
- hemophilia A, inhibitor, treatment
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
recombinant porcine coagulation factor VIII (OBI-1)Drug
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Secondary outcomes (4)
Adverse events and serious adverse events observed throughout course of study
Pattern of inhibitory antibody response to OBI-1 following treatments
The percentage of patients who continue to qualify because their anti-OBI-1 titer remains at 20 Bu or less
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
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Oct 2005 (estimated)
- Study startMay 2005
- Study completionJun 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Octagen Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Biomeasure Inc, Ipsen Group
- Investigators
- Josef N Mueksch, MD, MBA, Principal investigator, Octagen Corporation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00245245
- Sponsor's protocol ID
- OBI-1 - 201
- Other IDs
- NIH Grant 2R44 HL064497-02
- Status last verified
- Oct 2007
- First submitted
- 25 Oct 2005
- Last updated
- 4 Oct 2007
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Oct 2007. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT00245245
- Acronym / sponsor ID
- OBI-1 - 201
- Conditions
- Hemophilia A
- Keywords
- hemophilia A, inhibitor, treatment
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Octagen CorporationIndustry (pharma, biotech, devices)
- Collaborators
- Biomeasure Inc, Ipsen Group
- Study start
- May 2005
- Study completion
- Jun 2007 (actual)
- First posted
- 27 Oct 2005 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The ability of a new recombinant porcine coagulation factor VIII, B-domain deleted (called "OBI-1"), to control the non-life- or limb-threatening bleeding episodes patients with hemophilia A commonly develop is being evaluated. Patients with congenital hemophilia A and a low-titer (<20 Bethesda units [Bu]) inhibitory antibody to OBI-1, who meet the inclusion/exclusion criteria, will receive OBI-1 to treat their soft tissue or joint bleeding episode. At least the first two treatment episodes will be performed in the controlled setting of the hemophilia center/clinic/office, where any side effects can be observed. If the patient continues to meet the inclusion/exclusion criteria, has had no serious or severe adverse reactions to OBI-1, and has been in a home care program, the investigator may permit the patient to self-administer OBI-1 at home to treat subsequent bleeding episodes. The study will continue at least until 12 or more patients have received at least 24 treatment episodes in the aggregate.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (6 listed)
- 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
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 |
|---|---|---|
| recombinant porcine coagulation factor VIII (OBI-1) | Drug | – |
Primary outcome
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
4 secondary outcomes
Adverse events and serious adverse events observed throughout course of study
Pattern of inhibitory antibody response to OBI-1 following treatments
The percentage of patients who continue to qualify because their anti-OBI-1 titer remains at 20 Bu or less
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Oct 2007.
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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.
Josef N Mueksch, MD, MBA principal investigator
Octagen Corporation
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing recombinant porcine coagulation factor VIII (OBI-1) in people with Hemophilia A, run by Octagen Corporation at 1 site in United States.
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 5 inclusion and 6 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: finished, as of the sponsor's last update on 4 Oct 2007. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
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. 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 4 Oct 2007.