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| ID | Type | Description | Link |
|---|---|---|---|
| 2007-003718-32 | EudraCT Number |
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A study to assess treatment with a new formulation of recombinant factor VIII reconstituted with liposomes (BAY79-4980) to evaluate whether a once-a-week treatment is safe and can prevent bleeds in subjects with severe haemophilia A.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| rFVIII-FS/pegylated liposomes (BAY79-4980) | Experimental | 35 IU/kg body weight of BAY79-4980 1x/week plus 2 dummy injections/week (dummy = rFVIII (recombinant factor VIII)-FS (formulated with sucrose) excipient reconstituted in WFI (sterile water for injection)) |
|
| rFVIII-FS/WFI (BAY14-2222) | Active Comparator | 25 IU/kg body weight of rFVIII-FS 3x/week (employing 1 percent POPC (1-palmitoyl-2-oleoyl-sn-glycero-3-phosphocholine)-alone liposome (rFVIII-FS-POPC) as blinding agent used for first weekly injection and rFVIII-FS in WFI for 2nd and 3rd injection) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| rFVIII-FS/pegylated liposomes (BAY79-4980) | Biological | 35 IU/kg body weight intravenous 1x/week for 52 weeks This arm will be stopped by 30.04.10 the subjects will be offered to change to the active comparator arm |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Less Than 9 Total Bleeds Per Year | Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event. | up to one year |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Less Than 5 Joint Bleeds Per Year | Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event. | up to one year |
| Number of Joint Bleeds Per Participant Per Year in Responders |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Bleeds Per Year | Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event. Number of bleeds 3 weeks after the first infusion per 12 months | up to one year |
| Percentage of Bleeds Treated by Various Numbers of Injections |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Bayer Study Director | Bayer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Orange | California | 92868 | United States | |||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 20059562 | Result | Di Minno G, Cerbone AM, Coppola A, Cimino E, Di Capua M, Pamparana F, Tufano A, Di Minno MN. Longer-acting factor VIII to overcome limitations in haemophilia management: the PEGylated liposomes formulation issue. Haemophilia. 2010 Jan;16 Suppl 1:2-6. doi: 10.1111/j.1365-2516.2009.02155.x. | |
| 23014711 | Result | Powell J, Martinowitz U, Windyga J, Di Minno G, Hellmann A, Pabinger I, Maas Enriquez M, Schwartz L, Ingerslev J; LipLong Study Investigators. Efficacy and safety of prophylaxis with once-weekly BAY 79-4980 compared with thrice-weekly rFVIII-FS in haemophilia A patients. A randomised, active-controlled, double-blind study. Thromb Haemost. 2012 Nov;108(5):913-22. doi: 10.1160/TH12-03-0188. Epub 2012 Sep 26. |
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Of 168 enrolled participants, 25 failed screening.
Previously treated participants with severe hemophilia A (<1% FVIII (Factor VIII)), who were currently on on-demand or secondary prophylaxis treatment with any FVIII for ≥150 exposure days (ED) with documented bleeds/injections during the last 6 months prior to study entry could participate in the study.
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| ID | Title | Description |
|---|---|---|
| FG000 | rFVIII-FS/Pegylated Liposomes (BAY79-4980) | 35 IU/kg body weight of BAY79-4980 1x/week plus 2 dummy injections/week (dummy = rFVIII (recombinant factor VIII)-FS (formulated with sucrose) excipient reconstituted in WFI (sterile water for injection)) |
| FG001 | rFVIII-FS/WFI (BAY14-2222) |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Double Blind (DB) |
|
Not provided
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| rFVIII-FS/WFI (BAY14-2222) | Biological | 25 IU/kg body weight intravenous 3x/week for 52 weeks |
|
Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event. Responders were the subjects with less than 9 total bleeds per year |
| up to one year |
Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event. |
| up to one year |
| Total rFVIII Consumption Per Year | Total number of units per kg of study medication (rFVIII) administered to participant for one year. rFVIII is recombinant factor VIII, factor VIII is functional coagulation factor | up to one year |
| Percentage of Participants With Less Than 9 Total Bleeds Per Year in the Open Label Extension Period | Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event. | 6 months after start of open label extension period |
| Sacramento |
| California |
| 95817 |
| United States |
| San Francisco | California | 94143-0324 | United States |
| Aurora | Colorado | 80045 | United States |
| New Orleans | Louisiana | 70112-2699 | United States |
| Boston | Massachusetts | 02115 | United States |
| Detroit | Michigan | 48202 | United States |
| East Lansing | Michigan | 48823 | United States |
| Rochester | Minnesota | 55905 | United States |
| Kansas City | Missouri | 64108-9898 | United States |
| Las Vegas | Nevada | 89109-2803 | United States |
| Albuquerque | New Mexico | 87131-0001 | United States |
| Buffalo | New York | 14215 | United States |
| Rochester | New York | 14621 | United States |
| Winston-Salem | North Carolina | 27157-1009 | United States |
| Cincinnati | Ohio | 45229 | United States |
| Philadelphia | Pennsylvania | 19104-0506 | United States |
| Pittsburgh | Pennsylvania | 15213 | United States |
| Houston | Texas | 77030 | United States |
| Milwaukee | Wisconsin | 53226 | United States |
| Buenos Aires | Ciudad Auton. de Buenos Aires | C1221ADC | Argentina |
| Buenos Aires | Ciudad Auton. de Buenos Aires | C1425AUM | Argentina |
| Rosario | Santa Fe Province | S2000CKF | Argentina |
| Perth | Western Australia | 6000 | Australia |
| Vienna | State of Vienna | 1090 | Austria |
| Graz | 8036 | Austria |
| Bruxelles - Brussel | 1020 | Belgium |
| Bruxelles - Brussel | 1200 | Belgium |
| Leuven | 3000 | Belgium |
| Calgary | Alberta | T2N 2T9 | Canada |
| Edmonton | Alberta | T6G 2C8 | Canada |
| Halifax | Nova Scotia | B3H 2Y9 | Canada |
| Hamilton | Ontario | L8S 4J9 | Canada |
| Ottawa | Ontario | K1H 8L6 | Canada |
| Toronto | Ontario | M5B 1W8 | Canada |
| Toronto | Ontario | M5G 1X8 | Canada |
| Santiago | Chile |
| Zagreb | 10000 | Croatia |
| Ã…rhus N | 8200 | Denmark |
| Tallinn | 13419 | Estonia |
| Besançon | 25030 | France |
| Le Kremlin-Bicêtre | 94275 | France |
| Lyon | 69437 | France |
| Marseille | 13005 | France |
| Montpellier | 34059 | France |
| Paris | 75015 | France |
| Paris | 75674 | France |
| Rennes | 35033 | France |
| Strasbourg | 67091 | France |
| Bonn | North Rhine-Westphalia | 53105 | Germany |
| Münster | North Rhine-Westphalia | 48143 | Germany |
| Berlin | State of Berlin | 10249 | Germany |
| Tel Litwinsky | 52621 | Israel |
| Catania | 95124 | Italy |
| Florence | 50134 | Italy |
| Milan | 20122 | Italy |
| Naples | 80131 | Italy |
| Naples | 80144 | Italy |
| Torino | 10126 | Italy |
| KlaipÄ—da | 50809 | Lithuania |
| Vilnius | 08661 | Lithuania |
| Groningen | 9713 GZ | Netherlands |
| Nijmegen | 6525 GA | Netherlands |
| Utrecht | 3508 GA | Netherlands |
| Auckland | 1023 | New Zealand |
| Christchurch | 8001 | New Zealand |
| Oslo | 0027 | Norway |
| Gdansk | 80-952 | Poland |
| Krakow | 31-501 | Poland |
| Lodz | 93-510 | Poland |
| Poznan | 61-833 | Poland |
| Warsaw | 02-776 | Poland |
| Timișoara | Timiș County | 300011 | Romania |
| Brasov | 50035 | Romania |
| Bucharest | 022328 | Romania |
| Johannesburg | Gauteng | 2132 | South Africa |
| Seville | Andalusia | 41013 | Spain |
| Barcelona | Barcelona | 08035 | Spain |
| Madrid | Madrid | 28046 | Spain |
| Oviedo | Principality of Asturias | 33006 | Spain |
| Santa Cruz de Tenerife | Santa Cruz de Tenerife | 38009 | Spain |
| Valencia | Valencia | 46026 | Spain |
| Bern | 3010 | Switzerland |
| Changhua | 500 | Taiwan |
| Taipei | 10002 | Taiwan |
| Taipei | 11217 | Taiwan |
| Adana | 01-330 | Turkey (Türkiye) |
| Antalya | 07059 | Turkey (Türkiye) |
| Izmir | 35-100 | Turkey (Türkiye) |
| Cambridge | Cambridgeshire | CB2 0QQ | United Kingdom |
| London | London | SE1 7EH | United Kingdom |
| Manchester | Manchester | M13 9WL | United Kingdom |
| Cardiff | South Glamorgan | CF14 4XW | United Kingdom |
| Sheffield | South Yorkshire | S10 2JF | United Kingdom |
| Birmingham | B15 2TH | United Kingdom |
25 IU/kg body weight of rFVIII-FS 3x/week (employing 1 percent POPC (1-palmitoyl-2-oleoyl-sn-glycero-3-phosphocholine)-alone liposome (rFVIII-FS-POPC) as blinding agent used for first weekly injection and rFVIII-FS in WFI for 2nd and 3rd injection) |
| Participants Received Treatment |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
| Open Label Follow-up |
|
|
| Open Label Extension Period |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | rFVIII-FS/Pegylated Liposomes (BAY79-4980) | 35 IU/kg body weight of BAY79-4980 1x/week plus 2 dummy injections/week (dummy = rFVIII (recombinant factor VIII)-FS (formulated with sucrose) excipient reconstituted in WFI (sterile water for injection)) |
| BG001 | rFVIII-FS/WFI (BAY14-2222) | 25 IU/kg body weight of rFVIII-FS 3x/week (employing 1 percent POPC (1-palmitoyl-2-oleoyl-sn-glycero-3-phosphocholine)-alone liposome (rFVIII-FS-POPC) as blinding agent used for first weekly injection and rFVIII-FS in WFI for 2nd and 3rd injection) |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| target joint | The presence or absence of target joints (defined as joints with at least 3 bleeds into the same joint within 6 months) was evaluated. | Number | participants |
| |||||||||||||||
| Previous treatment | Previous treatment was categorized as prophylaxis versus on demand | Number | 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 | Percentage of Participants With Less Than 9 Total Bleeds Per Year | Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event. | Per protocol (PP) population | Posted | Number | Percentage of participants | up to one year |
|
|
| |||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Less Than 5 Joint Bleeds Per Year | Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event. | PP population | Posted | Number | Percentage of participants | up to one year |
|
| ||||||||||||||||||||||||||||||
| Secondary | Number of Joint Bleeds Per Participant Per Year in Responders | Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event. Responders were the subjects with less than 9 total bleeds per year | PP population in responders | Posted | Median | Full Range | Joint bleeds per year | up to one year |
|
| |||||||||||||||||||||||||||||
| Other Pre-specified | Number of Bleeds Per Year | Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event. Number of bleeds 3 weeks after the first infusion per 12 months | PP population | Posted | Median | Full Range | bleeds per year | up to one year |
|
| |||||||||||||||||||||||||||||
| Other Pre-specified | Percentage of Bleeds Treated by Various Numbers of Injections | Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event. | PP Population | Posted | Number | percentage of bleeds | up to one year |
|
| ||||||||||||||||||||||||||||||
| Other Pre-specified | Total rFVIII Consumption Per Year | Total number of units per kg of study medication (rFVIII) administered to participant for one year. rFVIII is recombinant factor VIII, factor VIII is functional coagulation factor | ITT population | Posted | Median | Full Range | IU per kg | up to one year |
|
| |||||||||||||||||||||||||||||
| Other Pre-specified | Percentage of Participants With Less Than 9 Total Bleeds Per Year in the Open Label Extension Period | Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event. | Participants who completed open label extension period | Posted | Number | Percentage of participants | 6 months after start of open label extension period |
|
|
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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/Pegylated Liposomes (BAY79-4980) - Double Blind | Reporting Group 1 (RG1): Double Blind Study, 35 IU/kg body weight of BAY79-4980 1x/week plus 2 dummy injections/week (dummy = rFVIII-FS excipient reconstituted in WFI) | 7 | 67 | 27 | 67 | ||
| EG001 | rFVIII-FS/WFI (BAY14-2222) - Double Blind | Reporting group 2 (RG2): Double Blind Study, 25 IU/kg body weight of rFVIII-FS 3x/week (employing 1 percent POPC-alone liposome (rFVIII-FS-POPC) as blinding agent used for first weekly injection and rFVIII-FS in WFI for 2nd and 3rd injection) | 1 | 72 | 27 | 72 | ||
| EG002 | rFVIII-FS/WFI (BAY14-2222) - Follow-up | Reporting group 3 (RG3): Open Label Follow-up, 25 IU/kg body weight of rFVIII-FS 3x/week (employing 1 percent POPC-alone liposome (rFVIII-FS-POPC) as blinding agent used for first weekly injection and rFVIII-FS in WFI for 2nd and 3rd injection) | 0 | 26 | 2 | 26 | ||
| EG003 | rFVIII-FS/Pegylated Liposomes (BAY79-4980) - Extension | Reporting group 4 (RG4): Open Label Extension, 35 IU/kg body weight of BAY79-4980 1x/week plus 2 dummy injections/week (dummy = rFVIII-FS excipient reconstituted in WFI) | 1 | 20 | 10 | 20 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Glaucoma | Eye disorders | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Melaena | Gastrointestinal disorders | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Rectal haemorrhage | Gastrointestinal disorders | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Oedema | General disorders | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Hypersensitivity | Immune system disorders | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Device related infection | Infections and infestations | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Tinea pedis | Infections and infestations | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Varicella | Infections and infestations | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Abnormal weight gain | Metabolism and nutrition disorders | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Compartment syndrome | Musculoskeletal and connective tissue disorders | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Urinary retention | Renal and urinary disorders | MedDRA v. 13.0 | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Lymphadenopathy | Blood and lymphatic system disorders | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Localised infection | Infections and infestations | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Varicella | Infections and infestations | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Limb injury | Injury, poisoning and procedural complications | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Blood amylase increased | Investigations | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Lipase increased | Investigations | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Haemophilic arthropathy | Musculoskeletal and connective tissue disorders | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Dysuria | Renal and urinary disorders | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA v. 13.0 | Non-systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA v. 13.0 | Non-systematic Assessment |
|
The recruitment and double-blind study phase were prematurely terminated after a scheduled interim analysis confirmed overt failure regarding the primary endpoint as judged by the independent Data and Safety Monitoring Board.
Bayer will have up to 30/45 days to review publications, and may request an additional publication delay of up to 60 days to allow for filing a Patent Application (if applicable).
No publication of single center data should be done prior of publication if multi -center data.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Therapeutic Area Head | BAYER | clinical-trials-contact@bayerhealthcare.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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| ID | Term |
|---|---|
| C414350 | BAY 14-2222 |
Not provided
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| Participant convenience |
|
| Male |
|
| No |
|
| Prophylaxis |
|
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|
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