Study of Recombinant Porcine Factor VIII (FVIII) in Hemophilia and Inhibitors to FVIII

NCT00245245 | Completed Phase 2

• 2 other identifiers: OBI-1 - 201NIH Grant 2R44 HL064497-02
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Hemophilia A

Keywords

hemophilia A; inhibitor; treatment

Outcome Measures

Primary Outcomes (1)

• 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

Interventions

recombinant porcine coagulation factor VIII (OBI-1) DRUG

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Octagen Corporation: lead
• Biomeasure Inc, Ipsen Group: collaborator

Study Sites (1)

Octagen Corporation

Blue Bell, Pennsylvania, 19422, United States

Location

MeSH Terms

Conditions

Hemophilia A

Interventions

ZNF717 protein, human

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Josef N Mueksch, MD, MBA

  Octagen Corporation

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY

Study Record Dates

• First Submitted: October 25, 2005
• First Posted: October 27, 2005
• Study Start: May 1, 2005
• Study Completion: June 1, 2007
• Last Updated: October 4, 2007

Record last verified: 2007-10

Locations

US (1)