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| ID | Type | Description | Link |
|---|---|---|---|
| 2005-002757-45 | EudraCT Number |
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The purpose of the study is to evaluate the effect of prophylactic treatment on the number of joint bleeds and quality of life in severe hemophilia A subjects compared to on-demand treatment in a one-group two-treatment schedule design.
In addition, the effect of prophylactic treatment on the joint function, the number of all bleeds, and on the quality of life compared to on-demand treatment and health-economic data will be assessed.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| rFVIII-FS (octocog-alfa), (Kogenate FS) | Experimental | On-demand treatment was to follow the same treatment pattern the subject was using before entering the study. While on prophylactic treatment, all subjects were to be treated at a dose of 20-40 IU/kg, 3 times per week at a stable dose. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Kogenate (BAY14-2222) | Drug | One group two treatment schedules, first on-demand then switch to prophylaxis |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Joint Bleeds | Number of joint bleeds during Months 8-13 compared to number of joint bleeds during Months 1-6 | Months 1-6 (on-demand treatment) and 8-13 (prophylactic treatment) |
| Measure | Description | Time Frame |
|---|---|---|
| Number of All Bleeds | Mean number of all bleeds during Months 8-13 (prophylactic) compared to mean number of all bleeds during Months 1-6 (on-demand) | Months 1-6 (on-demand treatment) and 8-13 (prophylactic treatment) |
| Physical Assessment Compared to On-demand Treatment as Determined by the Gilbert Score |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Bayer Study Director | Bayer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Aurora | Colorado | 80045 | United States | |||
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| Label | URL |
|---|---|
| Click here to find information about studies related to Bayer Healthcare products conducted in Europe | View source |
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All subjects who met eligibility criteria on screening were enrolled into the study and began the on-demand treatment period.
Patients were recruited at Haemophilia treatment centers
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| ID | Title | Description |
|---|---|---|
| FG000 | rFVIII-FS (Octocog-alfa), (Kogenate FS) | On-demand treatment was to follow the same treatment pattern the subject was using before entering the study. While on prophylactic treatment, all subjects were to be treated at a dose of 20-40 IU/kg, 3 times per week at a stable dose. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| On Demand Treatment (Months 1-6) |
|
| ||||||||||||||||||
| Prophylactic Treatment (Months 8-13) |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | rFVIII-FS (Octocog-alfa), (Kogenate FS) | On-demand treatment was to follow the same treatment pattern the subject was using before entering the study. While on prophylactic treatment, all subjects were to be treated at a dose of 20-40 IU/kg, 3 times per week at a stable dose. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| 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 | Number of Joint Bleeds | Number of joint bleeds during Months 8-13 compared to number of joint bleeds during Months 1-6 | intent-to-treat population | Posted | Mean | Standard Deviation | Number of joint bleeds | Months 1-6 (on-demand treatment) and 8-13 (prophylactic 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 | rFVIII-FS (Octocog-alfa), (Kogenate FS) | On-demand treatment was to follow the same treatment pattern the subject was using before entering the study. While on prophylactic treatment, all subjects were to be treated at a dose of 20-40 IU/kg, 3 times per week at a stable dose. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Carbon monoxide poisoning | Injury, poisoning and procedural complications | MedDRA 9.1 | Non-systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Lower respiratory tract infection | Infections and infestations | MedDRA 9.1 | Non-systematic Assessment |
The short observation period of 6 months may be responsible for the lack of statistical significance between treatments in the subject's overall assessment of disease-related quality of life; however, a positive trend was noted.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Therapeutic Area Head | BAYER | clinical-trials-contact@bayerhealthcare.com |
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| ID | Term |
|---|---|
| D006402 | Hematologic Diseases |
| D006467 | Hemophilia A |
| D020141 | Hemostatic Disorders |
| ID | Term |
|---|---|
| D006425 | Hemic and Lymphatic Diseases |
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D020147 | Coagulation Protein Disorders |
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| ID | Term |
|---|---|
| C078147 | F8 protein, human |
| C414350 | BAY 14-2222 |
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Total score with a range of 0-100, evaluating ankle, knee and elbow, 0 indicates normal function, higher values indicate joint damage |
| Month 13 (end of prophylactic treatment) and Month 6 (end of on-demand treatment) |
| Quality of Life Compared to On-demand Treatment as Measured by the Haemo-QoL A Questionnaire | Total transformed score with a range of 0-100, higher values indicate better outcome. 41 items in 6 domains: physical functioning; role functioning; worry; consequences; positive affect; treatment concern. | Month 13 (end of prophylactic treatment) and Month 6 (end of on-demand treatment) |
| Chapel Hill |
| North Carolina |
| 27599 |
| United States |
| Houston | Texas | 77030 | United States |
| Strasbourg | 67098 | France |
| Florence | 50134 | Italy |
| Pavia | 27100 | Italy |
| Roma | 00161 | Italy |
| Madrid | Madrid | 28046 | Spain |
| Santa Cruz de Tenerife | Santa Cruz de Tenerife | 38009 | Spain |
| Cardiff | South Glamorgan | CF14 4XW | United Kingdom |
| Sheffield | South Yorkshire | S10 2JF | United Kingdom |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Target joints presence | 16 (80%) subjects had at least 1 target joint; in most elbow(s) and/or ankles(s) were affected | Number | participants |
|
| Participants |
|
|
|
| Secondary | Number of All Bleeds | Mean number of all bleeds during Months 8-13 (prophylactic) compared to mean number of all bleeds during Months 1-6 (on-demand) | intention-to-treat | Posted | Mean | Standard Deviation | All bleeds | Months 1-6 (on-demand treatment) and 8-13 (prophylactic treatment) |
|
|
|
|
| Secondary | Physical Assessment Compared to On-demand Treatment as Determined by the Gilbert Score | Total score with a range of 0-100, evaluating ankle, knee and elbow, 0 indicates normal function, higher values indicate joint damage | intent-to-treat | Posted | Mean | Standard Deviation | Gilbert score (0-100) | Month 13 (end of prophylactic treatment) and Month 6 (end of on-demand treatment) |
|
|
|
|
| Secondary | Quality of Life Compared to On-demand Treatment as Measured by the Haemo-QoL A Questionnaire | Total transformed score with a range of 0-100, higher values indicate better outcome. 41 items in 6 domains: physical functioning; role functioning; worry; consequences; positive affect; treatment concern. | intent-to-treat-population | Posted | Mean | Standard Deviation | Transformed score | Month 13 (end of prophylactic treatment) and Month 6 (end of on-demand treatment) |
|
|
|
|
| 2 |
| 20 |
| 12 |
| 20 |
| Femur fracture | Injury, poisoning and procedural complications | MedDRA 9.1 | Non-systematic Assessment |
|
| Road traffic accident | Injury, poisoning and procedural complications | MedDRA 9.1 | Non-systematic Assessment |
|
| Dehydration | Metabolism and nutrition disorders | MedDRA 9.1 | Non-systematic Assessment |
|
| Alcohol use | Social circumstances | MedDRA 9.1 | Non-systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA 9.1 | Non-systematic Assessment |
|
| Bronchitis | Infections and infestations | MedDRA 9.1 | Non-systematic Assessment |
|
| Gastroenteritis | Infections and infestations | MedDRA 9.1 | Non-systematic Assessment |
|
| Gingival infection | Infections and infestations | MedDRA 9.1 | Non-systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA 9.1 | Non-systematic Assessment |
|
| Road traffic accident | Injury, poisoning and procedural complications | MedDRA 9.1 | Non-systematic Assessment |
|
| Traumatic haemorrhage | Injury, poisoning and procedural complications | MedDRA 9.1 | Non-systematic Assessment |
|
| Limb injury | Injury, poisoning and procedural complications | MedDRA 9.1 | Non-systematic Assessment |
|
| Meniscus lesion | Injury, poisoning and procedural complications | MedDRA 9.1 | Non-systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 9.1 | Non-systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 9.1 | Non-systematic Assessment |
|
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 9.1 | Non-systematic Assessment |
|
| Synovitis | Musculoskeletal and connective tissue disorders | MedDRA 9.1 | Non-systematic Assessment |
|
| Dysgeusia | Nervous system disorders | MedDRA 9.1 | Non-systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA 9.1 | Non-systematic Assessment |
|
| Sinus headache | Nervous system disorders | MedDRA 9.1 | Non-systematic Assessment |
|
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 9.1 | Non-systematic Assessment |
|
| Sinus disorder | Respiratory, thoracic and mediastinal disorders | MedDRA 9.1 | Non-systematic Assessment |
|
| Dermatitis | Skin and subcutaneous tissue disorders | MedDRA 9.1 | Non-systematic Assessment |
|
| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA 9.1 | Non-systematic Assessment |
|
| Ear discomfort | Ear and labyrinth disorders | MedDRA 9.1 | Non-systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 9.1 | Non-systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 9.1 | Non-systematic Assessment |
|
| Chest pain | General disorders | MedDRA 9.1 | Non-systematic Assessment |
|
| Anxiety | Psychiatric disorders | MedDRA 9.1 | Non-systematic Assessment |
|
| Renal colic | Renal and urinary disorders | MedDRA 9.1 | Non-systematic Assessment |
|
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| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |