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To compare the number of breakthrough bleeds under tailored prophylaxis with Human cell line recombinant factor FVIII (Human-cl rhFVIII) with the historical bleeding rate from patients who received Human-cl rhFVIII as on demand treatment.
There were 3 phases in this study: (1) An initial pharmacokinetic (PK) assessment in which participants received a single infusion of 60±5 IU/kg of Human-cl rhFVIII; blood samples were collected for 72 hours following the infusion. (2) Prophylactic Treatment-Phase I during which participants received infusions of 30-40 IU/kg of human-cl rhFVIII every other day or 3x/week for 1-3 months. (3) Prophylactic Treatment-Phase II during which the dose and dosing interval were determined individually from data gathered in the initial PK assessment. The maximum dosing interval with a dose of ≤ 60-80 IU/kg that maintains a trough level of ≥ 0.01 IU/mL was determined. Participants were treated for 6 months.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Human-cl rhFVIII | Experimental | Up to 60-80 IU/kg of intravenous Human-cl rhFVIII was administered at an individually determined dose and dose interval. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Human-cl rhFVIII | Biological | Human-cl rhFVIII was provided as a freeze-dried concentrate to be reconstituted in water for injection. |
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| Measure | Description | Time Frame |
|---|---|---|
| Annualized Number of Bleeding Episodes (BE) in Phase II | The annualized number of total BEs was calculated for each participant as follows: d*y/t, where y = the number of BEs documented in Phase II, t = the number of treatment periods in days, and d = 365.25, the number of days per year. A bleeding episode (BE) was defined as any BE whether treated or not during Phase II of the study; BEs related to surgery were not included. This study was considered as showing efficacy if the annualized number of BEs was reduced by 50% compared to the number of BEs observed in study GENA-01 where patient where severe Hemophilia A patients were treated on-demand (NCT00989196). | Beginning to the end of Phase II (6 months) |
| Measure | Description | Time Frame |
|---|---|---|
| Annualized Number of Spontaneous Bleeding Episodes (BE) in Phase II | The annualized number of spontaneous BEs was calculated for each participant as follows: d*y/t, where y = the number of spontaneous BEs documented in Phase II, t = the number of treatment periods in days, and d = 365.25, the number of days per year. A spontaneous bleeding episode (BE) was defined as a BE whether treated or not during Phase II of the study. BEs related to surgery and BEs due to trauma or due to other causes were not included. |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Medical University Vienna | Vienna | Austria | ||||
| University Multiprofile Hospital for Active Treatment |
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| ID | Title | Description |
|---|---|---|
| FG000 | Human-cl rhFVIII | Up to 60-80 IU/kg of intravenous human-cl rhFVIII (human cell line recombinant Factor VIII) was administered at an individually determined dose and dose interval. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Pharmacokinetic Assessment |
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| Beginning to the end of Phase II (6 months) |
| Annualized Number of Bleeding Episodes (BE) in Phase II in Participants With ≤ 2 Treatments/Week | The annualized number of BEs was calculated for each participant as follows: d*y/t, where y = the number of BEs documented in Phase II, t = the number of treatment periods in days, and d = 365.25, the number of days per year. A bleeding episode (BE) was defined as a BE whether treated or not during Phase II of the study. BEs related to surgery were not included. | Beginning to the end of Phase II (6 months) |
| Median Dosing Interval During Individually Tailored Prophylaxis | The median time between 2 prophylactic doses of Human-cl rhFVIII in the prophylactic treatment phase II were determined per patient | Beginning to the end of Phase II (6 months) |
| Dosage Per Week in Phase II | The mean dosage per week during Phase II of the study are reported. | Beginning to the end of Phase II (6 months) |
| Plovdiv |
| Bulgaria |
| Specialized Hospital for Active Treatment | Sofia | Bulgaria |
| Multiprofile Hospital for Active Treatment | Varna | Bulgaria |
| Vivantes Hospital in Friedrichshain | Berlin | Germany |
| SRH Kurpfalzklinik Heidelberg GMBH | Heidelberg | Germany |
| Hungarian National Healthcare Center | Budapest | Hungary |
| University of Debrecen, Medical and Health Science Center | Debrecen | Hungary |
| University Teaching Hospital in Bialystok, Teaching Department of Hematology with a Subdepartment of Vascular Diseases | Bialystok | Poland |
| University Clinical Center, Teaching Department of Hematology and Transplantology | Gdansk | Poland |
| Nicolaus Copernicus Municipal Specialist Hospital, Department of Hematology | Torun | Poland |
| Institute of Hematology and Transfusion Medicine, Depart. of Hemostatic Disorders and Internal Diseases | Warsaw | Poland |
| Sanador SRL | Bucharest | Romania |
| Louis Turcanu Emergency Clinical Children's Hospital | Timișoara | Romania |
| University Hospital Saint Cyril and Metod | Bratislava | Slovakia |
| University Hospital Martin, Department of Hematology and Transfusiology | Martin | Slovakia |
| Basingstoke and North Hampshire Hospital, Hemophilia, Hemostasis and Thrombosis Center | Basingstoke | United Kingdom |
| Royal London Hospital, Barts and the London Hemophilia Center | London | United Kingdom |
| Manchester Royal Infirmary, Department of Clinical Hematology | Manchester | United Kingdom |
| Royal Hallamshire Hospital | Sheffield | United Kingdom |
| COMPLETED |
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| NOT COMPLETED |
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| Prophylactic Treatment-Phase I |
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| Prophylactic Treatment-Phase II |
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Intent-to-treat/prophylactic/safety population: All participants who received at least 1 dose of Human-cl rhFVIII in Phase II and had any data collected after treatment with Human-cl rhFVIII.
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| ID | Title | Description |
|---|---|---|
| BG000 | Human-cl rhFVIII | Up to 60-80 IU/kg of intravenous human-cl rhFVIII (human cell line recombinant Factor VIII) was administered at an individually determined dose and dose interval. |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
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| Age | Mean | Standard Deviation | years |
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| Weight | Mean | Standard Deviation | kg |
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| BMI | Mean | Standard Deviation | kg/m^2 |
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| Hemophilia Joint Health Score (HJHS) | The HJHS is determined through a physical examination of the 6 joints most affected by hemophilia; the elbows, knees, and ankles. Knees and ankles are assessed on 11 items: Swelling, duration of swelling, muscle atrophy, axial alignment, crepitus on motion, flexion loss, extension loss, instability, joint pain, strength, and gait. Elbows are assessed on 9 of the 11 items (axial alignment and gait are not assessed). Each item is assessed on a scale that ranges from 0-1 to 0-4. The total score can range from 0 to 148, with a higher score indicating a bigger effect of hemophilia. | Mean | Standard Deviation | Units on a scale |
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| 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 | Annualized Number of Bleeding Episodes (BE) in Phase II | The annualized number of total BEs was calculated for each participant as follows: d*y/t, where y = the number of BEs documented in Phase II, t = the number of treatment periods in days, and d = 365.25, the number of days per year. A bleeding episode (BE) was defined as any BE whether treated or not during Phase II of the study; BEs related to surgery were not included. This study was considered as showing efficacy if the annualized number of BEs was reduced by 50% compared to the number of BEs observed in study GENA-01 where patient where severe Hemophilia A patients were treated on-demand (NCT00989196). | Prophylactic treatment population: All participants who received at least 1 dose of Human-cl rhFVIII in Phase II and had any data collected after treatment with Human-cl rhFVIII. | Posted | Mean | Standard Deviation | Annualized number of bleeding episodes | Beginning to the end of Phase II (6 months) |
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| Secondary | Annualized Number of Spontaneous Bleeding Episodes (BE) in Phase II | The annualized number of spontaneous BEs was calculated for each participant as follows: d*y/t, where y = the number of spontaneous BEs documented in Phase II, t = the number of treatment periods in days, and d = 365.25, the number of days per year. A spontaneous bleeding episode (BE) was defined as a BE whether treated or not during Phase II of the study. BEs related to surgery and BEs due to trauma or due to other causes were not included. | Prophylactic treatment population: All participants who received at least 1 dose of Human-cl rhFVIII in Phase II and had any data collected after treatment with Human-cl rhFVIII. | Posted | Mean | Standard Deviation | Annualized number of bleeding episodes | Beginning to the end of Phase II (6 months) |
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| Secondary | Annualized Number of Bleeding Episodes (BE) in Phase II in Participants With ≤ 2 Treatments/Week | The annualized number of BEs was calculated for each participant as follows: d*y/t, where y = the number of BEs documented in Phase II, t = the number of treatment periods in days, and d = 365.25, the number of days per year. A bleeding episode (BE) was defined as a BE whether treated or not during Phase II of the study. BEs related to surgery were not included. | Prophylactic treatment population: All participants who received at least 1 dose of Human-cl rhFVIII in Phase II and had any data collected after treatment with Human-cl rhFVIII. Only participants who received ≤ 2 treatments/week were included in the analysis. | Posted | Mean | Standard Deviation | Annualized number of bleeding episodes | Beginning to the end of Phase II (6 months) |
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| Secondary | Median Dosing Interval During Individually Tailored Prophylaxis | The median time between 2 prophylactic doses of Human-cl rhFVIII in the prophylactic treatment phase II were determined per patient | Prophylactic treatment population: All participants who received at least 1 dose of Human-cl rhFVIII in Phase II and had any data collected after treatment with Human-cl rhFVIII. | Posted | Median | Inter-Quartile Range | Hours | Beginning to the end of Phase II (6 months) |
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| Secondary | Dosage Per Week in Phase II | The mean dosage per week during Phase II of the study are reported. | Prophylactic treatment population: All participants who received at least 1 dose of Human-cl rhFVIII in Phase II and had any data collected after treatment with Human-cl rhFVIII. | Posted | Mean | Standard Deviation | IU/kg | Beginning to the end of Phase II (6 months) |
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Safety population: All participants who received at least 1 dose of Human-cl rhFVIII.
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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 | Human-cl rhFVIII | All subject who received at least one dose of intravenous Human-cl rhFVIII (human cell line recombinant Factor VIII). | 5 | 66 | 24 | 66 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Haematemesis | Gastrointestinal disorders | MedDRA (17.1) | Systematic Assessment |
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| Fracture of Lower Leg | Injury, poisoning and procedural complications | MedDRA (17.1) | Systematic Assessment |
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| Tenotomy of muculus gastrocnemius medialis | Surgical and medical procedures | MedDRA (17.1) | Systematic Assessment |
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| Appendicitis Acuta | Infections and infestations | MedDRA (17.1) | Systematic Assessment |
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| wound inflammation due to surgery | Infections and infestations | MedDRA (17.1) | Systematic Assessment |
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| Pain of lumbar spine | Musculoskeletal and connective tissue disorders | MedDRA (17.1) | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nasopharyngitis | Infections and infestations | MedDRA (17.1) | Systematic Assessment |
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| Tonsillitis | Infections and infestations | MedDRA (17.1) | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (17.1) | Systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (17.1) | Non-systematic Assessment |
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| Headache | Nervous system disorders | MedDRA (17.1) | Systematic Assessment |
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| Toothache | Gastrointestinal disorders | MedDRA (17.1) | Systematic Assessment |
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| Malaise | General disorders | MedDRA (17.1) | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA (17.1) | Systematic Assessment |
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Octapharma agreements may vary with individual investigators, but will not prohibit any investigator from publishing. Octapharma supports the publication of results from all centers of a multi-center trial but requests that reports based on single-site data not precede the primary publication of the entire clinical trial. Octapharma also reserves the right to review data prior to publishing and provide comments/changes within a certain time period.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Sylvia Werner | Octapharma | 415 260-9577 | sylvia.werner@octapharma.com |
| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| Hungary |
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| Poland |
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| United Kingdom |
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| Slovakia |
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| Bulgaria |
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| Germany |
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