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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| OBI-1 | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| OBI-1 | Biological | intravenous infusion, up to every 2-3 hours for the first 24 hours of treatment |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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). | 24 hours after initiation of treatment |
| Measure | Description | Time Frame |
|---|---|---|
| Overall Proportion of Serious Bleeding Episodes Successfully Controlled With OBI-1 Therapy, as Assessed by the Investigator. | 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 |
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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:
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:
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Indiana Hemophilia and Thrombosis Center | Indianapolis | Indiana | 46260 | United States | ||
| Charlotte Maxeke Johannesburg Academic Hospital |
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| ID | Title | Description |
|---|---|---|
| FG000 | OBI-1 | OBI-1: intravenous infusion, up to every 2-3 hours for the first 24 hours of treatment |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
This study was terminated early and only enrolled one participant. Due to concerns that the participant would be at risk of being re-identified, 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 this study (Congenital Hemophilia A).
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| ID | Title | Description |
|---|---|---|
| BG000 | OBI-1 | OBI-1: intravenous infusion, up to every 2-3 hours for the first 24 hours of treatment |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | This study was terminated early and only enrolled one participant. Due to concerns that the participant would be at risk of being re-identified, 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 this study (Congenital Hemophilia A). |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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). | This study was terminated early and only enrolled one participant. Due to concerns that the participant would be at risk of being re-identified, 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 this study (Congenital Hemophilia A). | Posted | 24 hours after initiation of treatment |
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | OBI-1 | OBI-1: intravenous infusion, up to every 2-3 hours for the first 24 hours of treatment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Shire | +1 866 842 5335 | ClinicalTransparency@shire.com |
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| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| D001778 | Blood Coagulation Disorders |
| D006474 | Hemorrhagic Disorders |
| D020147 | Coagulation Protein Disorders |
| D006402 | Hematologic Diseases |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D006425 | Hemic and Lymphatic Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| Through 90 days ± 7days following final OBI-1 dose |
| Frequency of Infusions of OBI-1 Required to Successfully Control Qualifying Bleeding Episodes. | Through 90 days ± 7days following final OBI-1 dose |
| Total Dose of OBI-1 Required to Successfully Control Qualifying Bleeding Episodes. | Through 90 days ± 7days following final OBI-1 dose |
| Total Number of Infusions of OBI-1 Required to Successfully Control Qualifying Bleeding Episodes. | 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. | 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. | 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. | 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. | 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) | Through 90 days ± 7days following final OBI-1 dose |
| Anti-human Factor VIII Antibody Titer. | Through 90 days ± 7days following final OBI-1 dose |
| Anti-OBI-1 Antibody Titer. | Through 90 days ± 7days following final OBI-1 dose |
| Anti-host Cell Protein Baby Hamster Kidney (BHK) Antibody Titer. | Through 90 days ± 7days following final OBI-1 dose |
| Johannesburg |
| Gauteng |
| 2193 |
| South Africa |
| Great Ormond Street Hospital | London | England | WC1N 3JH | United Kingdom |
| years |
| Sex: Female, Male | This study was terminated early and only enrolled one participant. Due to concerns that the participant would be at risk of being re-identified, 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 this study (Congenital Hemophilia A). |
|
| Region of Enrollment | This study was terminated early and only enrolled one participant. Due to concerns that the participant would be at risk of being re-identified, 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 this study (Congenital Hemophilia A). |
|
|
| Secondary | Overall Proportion of Serious Bleeding Episodes Successfully Controlled With OBI-1 Therapy, as Assessed by the Investigator. | This study was terminated early and only enrolled one participant. Due to concerns that the participant would be at risk of being re-identified, 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 this study (Congenital Hemophilia A). | Posted | Through 90 days ± 7days following final OBI-1 dose |
|
|
| Secondary | Proportion of Bleeding Episodes Responsive to OBI-1 Therapy at Designated Assessment Time Points After the Initiation of Therapy, as Assessed by the Investigator | This study was terminated early and only enrolled one participant. Due to concerns that the participant would be at risk of being re-identified, 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 this study (Congenital Hemophilia A). | Posted | Through 90 days ± 7days following final OBI-1 dose |
|
|
| Secondary | Frequency of Infusions of OBI-1 Required to Successfully Control Qualifying Bleeding Episodes. | This study was terminated early and only enrolled one participant. Due to concerns that the participant would be at risk of being re-identified, 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 this study (Congenital Hemophilia A). | Posted | Through 90 days ± 7days following final OBI-1 dose |
|
|
| Secondary | Total Dose of OBI-1 Required to Successfully Control Qualifying Bleeding Episodes. | This study was terminated early and only enrolled one participant. Due to concerns that the participant would be at risk of being re-identified, 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 this study (Congenital Hemophilia A). | Posted | Through 90 days ± 7days following final OBI-1 dose |
|
|
| Secondary | Total Number of Infusions of OBI-1 Required to Successfully Control Qualifying Bleeding Episodes. | This study was terminated early and only enrolled one participant. Due to concerns that the participant would be at risk of being re-identified, 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 this study (Congenital Hemophilia A). | Posted | Through 90 days ± 7days following final OBI-1 dose |
|
|
| Secondary | Correlation Between Response to OBI-1 Therapy at Specified Time Points and Eventual Control of Serious Bleeding Episodes. | This study was terminated early and only enrolled one participant. Due to concerns that the participant would be at risk of being re-identified, 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 this study (Congenital Hemophilia A). | Posted | Through 90 days ± 7days following final OBI-1 dose |
|
|
| Secondary | 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. | This study was terminated early and only enrolled one participant. Due to concerns that the participant would be at risk of being re-identified, 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 this study (Congenital Hemophilia A). | Posted | Frame: Through 90 days ± 7days following final OBI-1 dose |
|
|
| Secondary | Correlation Between the Pre-infusion Anti-OBI-1 Antibody Titers and the Recovery of 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, 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 this study (Congenital Hemophilia A). | Posted | Through 90 days ± 7days following final OBI-1 dose |
|
|
| Secondary | Recovery and Elimination Rate Parameters of OBI-1 in Subjects With Inhibitors Treated With OBI-1 Therapy. | This study was terminated early and only enrolled one participant. Due to concerns that the participant would be at risk of being re-identified, 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 this study (Congenital Hemophilia A). | Posted | Through 90 days ± 7days following final OBI-1 dose |
|
|
| Secondary | Efficacy Assessment of OBI-1 in Participants With Anti-human Factor VIII Titers >30 Bethesda Units (BU) | This study was terminated early and only enrolled one participant. Due to concerns that the participant would be at risk of being re-identified, 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 this study (Congenital Hemophilia A). | Posted | Through 90 days ± 7days following final OBI-1 dose |
|
|
| Secondary | Anti-human Factor VIII Antibody Titer. | This study was terminated early and only enrolled one participant. Due to concerns that the participant would be at risk of being re-identified, 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 this study (Congenital Hemophilia A). | Posted | Through 90 days ± 7days following final OBI-1 dose |
|
|
| Secondary | Anti-OBI-1 Antibody Titer. | This study was terminated early and only enrolled one participant. Due to concerns that the participant would be at risk of being re-identified, 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 this study (Congenital Hemophilia A). | Posted | Through 90 days ± 7days following final OBI-1 dose |
|
|
| Secondary | Anti-host Cell Protein Baby Hamster Kidney (BHK) Antibody Titer. | This study was terminated early and only enrolled one participant. Due to concerns that the participant would be at risk of being re-identified, 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 this study (Congenital Hemophilia A). | Posted | Through 90 days ± 7days following final OBI-1 dose |
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| 0 |
| 1 |
| 0 |
| 1 |
Baxter's agreements with PIs may vary per requirements of individual PI, but contain common elements. For this study, PIs are restricted from independently publishing results until the earlier of the primary multicenter publication or 12 months after study completion. Baxter requires a review of results communications (e.g., for confidential information) ≥30 days prior to submission or communication. Baxter may request an additional delay of ≤30 days(e.g., for intellectual property protection)