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The objective of this study is to assess whether prophylactic therapy with an activated prothrombin complex concentrate (FEIBA)will result in a significant reduction in the number of bleeds in patients with hemophilia and persistent high responding inhibitors.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| On-demand first | Other | Patients receive 6 months of on-demand therapy with study drug followed by 6 months of prophylaxis therapy with study drug |
|
| Prophylaxis first | Other | Patients receive 6 months of prophylaxis therapy with study drug followed by 6 months on-demand therapy with study drug |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| activated prothrombin complex concentrate (FEIBA) | Drug | FEIBA for prophylaxis therapy dosed at 85 U/Kg +/- 15% on three non-consecutive days each week for 6 months FEIBA for on-demand therapy dosed at 85 U/Kg +/- 15% for bleeding episodes for 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Bleeds During 6 Month Treatment Period | 6 months |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Cindy A Leissinger | Tulane University School of Medicine | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Tulane University School of Medicine | New Orleans | Louisiana | 70112 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 23731246 | Derived | Gringeri A, Leissinger C, Cortesi PA, Jo H, Fusco F, Riva S, Antmen B, Berntorp E, Biasoli C, Carpenter S, Kavakli K, Morfini M, Negrier C, Rocino A, Schramm W, Windyga J, Zulfikar B, Mantovani LG. Health-related quality of life in patients with haemophilia and inhibitors on prophylaxis with anti-inhibitor complex concentrate: results from the Pro-FEIBA study. Haemophilia. 2013 Sep;19(5):736-43. doi: 10.1111/hae.12178. Epub 2013 Jun 4. | |
| 22047559 |
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Patients were randomized to one of two 6 month treatment periods. Following the initial treatment period each subject had a 3 month wash-out period during which time they used on-demand therapy to treat bleeding. Following the 3 month wash-out period each patient was crossed-over to the other 6 month treatment period.
The study was conducted at 16 hemophilia treatment centers in Europe and the United States.
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| ID | Title | Description |
|---|---|---|
| FG000 | On-demand First | Patients receive 6 months of on-demand therapy with study drug followed by 6 months of prophylaxis therapy with study drug |
| FG001 | Prophylaxis First | Patients receive 6 months of prophylaxis therapy with study drug followed by 6 months on-demand therapy with study drug |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Therapy 1 (Months 1-6) |
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| Washout Period (Months 7-9) |
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| Therapy 2 (Months 10-15) |
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| ID | Title | Description |
|---|---|---|
| BG000 | On-demand First | Patients receive 6 months of on-demand therapy with study drug followed by 6 months of prophylaxis therapy with study drug |
| BG001 | Prophylaxis First | Patients receive 6 months of prophylaxis therapy with study drug followed by 6 months on-demand therapy with study drug |
| 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 Bleeds During 6 Month Treatment Period | Posted | Mean | Standard Deviation | bleeds | 6 months |
|
Adverse events were collected from time of consent to the end of the study, which totaled 15 months.
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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 | On-demand Period | During the on-demand period, bleeding was treated with AICC at a target dose of 85 U per kilogram of body weight (±15%) (range, 72 to 98). For bleeding episodes that did not respond to the specified therapy, alternative treatment, including additional doses of AICC, rFVIIa, or factor VIII, was allowed at the discretion of the treating physician. There were 3 subjects that never received on-demand therapy, therefore 31 subjects were assessed for AEs during this period. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Chest Pain | Cardiac disorders | Non-systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anemia | Blood and lymphatic system disorders | Non-systematic Assessment |
One limitation was the relatively short duration; in addition, due to the small number of children enrolled, it is not possible to draw conclusions regarding the relationship of age and the benefits of prophylaxis.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Cindy Leissinger | Tulane University | 504-988-5433 | cleissi@tulane.edu |
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| ID | Term |
|---|---|
| C065655 | anti-inhibitor coagulant complex |
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| Derived |
| Leissinger C, Gringeri A, Antmen B, Berntorp E, Biasoli C, Carpenter S, Cortesi P, Jo H, Kavakli K, Lassila R, Morfini M, Negrier C, Rocino A, Schramm W, Serban M, Uscatescu MV, Windyga J, Zulfikar B, Mantovani L. Anti-inhibitor coagulant complex prophylaxis in hemophilia with inhibitors. N Engl J Med. 2011 Nov 3;365(18):1684-92. doi: 10.1056/NEJMoa1104435. |
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| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
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| 3 |
| 31 |
| 16 |
| 31 |
| EG001 | Prophylaxis Period | During the prophylaxis period, AICC was administered at a target dose of 85 U per kilogram (±15%) (range, 72 to 98) on 3 nonconsecutive days weekly. Bleeding episodes during the prophylaxis period were treated with AICC at a target dose of 85 U per kilogram of body weight (±15%) (range, 72 to 98). For bleeding episodes that did not respond to the specified therapy, alternative treatment, including additional doses of AICC, rFVIIa, or factor VIII, was allowed at the discretion of the treating physician. There were 3 subjects that never received prophylaxis therapy, therefore 31 subjects were assessed for AEs during this period. | 4 | 31 | 17 | 31 |
| EG002 | Washout Period | Bleeding episodes during the washout period were treated with AICC at a target dose of 85 U per kilogram of body weight (±15%) (range, 72 to 98). For bleeding episodes that did not respond to the specified therapy, alternative treatment, including additional doses of AICC, rFVIIa, or factor VIII, was allowed at the discretion of the treating physician. There were 5 subjects that never entered the washout period, therefore 31 subjects were assessed for AEs during this period. | 4 | 29 | 14 | 29 |
| Pain | Musculoskeletal and connective tissue disorders | Non-systematic Assessment |
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| Drug hypersensitivity | Product Issues | Non-systematic Assessment |
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| Phlebitis | Blood and lymphatic system disorders | Non-systematic Assessment |
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| Hospitalization | Endocrine disorders | Non-systematic Assessment |
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| Surgical Procedure | Surgical and medical procedures | Non-systematic Assessment |
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| Catheter-site Hemorrhage | Blood and lymphatic system disorders | Non-systematic Assessment |
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| Catheter-site Infection | Infections and infestations | Non-systematic Assessment |
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| Staphylococcal Infection | Infections and infestations | Non-systematic Assessment |
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| Cerebral Hemorrhage | Vascular disorders | Non-systematic Assessment |
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| Subdural Hematoma | Vascular disorders | Non-systematic Assessment |
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| Gastrointestinal Hemorrhage | Gastrointestinal disorders | Non-systematic Assessment |
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| Joint Swelling | Musculoskeletal and connective tissue disorders | Non-systematic Assessment |
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| Muscle Hemorrhage | Musculoskeletal and connective tissue disorders | Non-systematic Assessment |
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| Headache | Musculoskeletal and connective tissue disorders | Non-systematic Assessment |
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| Pain | Musculoskeletal and connective tissue disorders | Non-systematic Assessment |
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| Pyrexia | General disorders | Non-systematic Assessment |
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| Drug Hypersensitivity | Immune system disorders | Non-systematic Assessment |
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| Hypersensitivity | Immune system disorders | Non-systematic Assessment |
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| Ecchymosis | Blood and lymphatic system disorders | Non-systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
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| Influenza | Infections and infestations | Non-systematic Assessment |
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| Nasopharyngitis | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
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| Pharyngitis | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
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| Upper Abdominal Pain | Gastrointestinal disorders | Non-systematic Assessment |
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| Vomiting | Gastrointestinal disorders | Non-systematic Assessment |
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| Poor Venous Access | Surgical and medical procedures | Non-systematic Assessment |
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| Catheter-site Hemorrhage | Blood and lymphatic system disorders | Non-systematic Assessment |
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| Catheter-site Infection | Infections and infestations | Non-systematic Assessment |
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| Staphylococcal Infection | Infections and infestations | Non-systematic Assessment |
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| Tongue Hemorrhage | Blood and lymphatic system disorders | Non-systematic Assessment |
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