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| ID | Type | Description | Link |
|---|---|---|---|
| 2012-003599-38 | EudraCT Number |
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To assess efficacy and safety, including immunogenicity of BAX 855 administered as prophylaxis and as on-demand therapy in adult and adolescent (12-65 years) previously treated patients (PTPs) with severe hemophilia A To determine the pharmacokinetic (PK) parameters of BAX 855.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Prophylaxis | Experimental |
| |
| On-demand | Experimental |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method | Biological | Pharmacokinetic (PK) evaluation of ADVATE |
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| Measure | Description | Time Frame |
|---|---|---|
| Annualized Bleeding Rate (ABR) | Comparisons between prophylactic and on-demand treatment were based on ABR estimates from a negative binomial regression model, taking into account the treatment regimen, target joints and age at screening, and duration of the observation period for efficacy. | 9 months |
| Measure | Description | Time Frame |
|---|---|---|
| Rate of Success of BAX 855 for Treatment of Bleeding Episodes | Success in the control of bleeding was defined as a rating of excellent or good using the Efficacy Rating Scale for Treatment of Bleeding Episodes measured 24 hours after initiation of treatment for the bleeding episode. EXCELLENT: Full relief of pain and cessation of objective signs of bleeding (eg, swelling, tenderness, and decreased range of motion in the case of musculoskeletal hemorrhage) after a single infusion. No additional infusion is required for the control of bleeding. Administration of further infusions to maintain hemostasis would not affect this scoring. GOOD: Definite pain relief and/or improvement in signs of bleeding after a single infusion. Possibly requires more than 1 infusion for complete resolution. FAIR: Probable and/or slight relief of pain and slight improvement in signs of bleeding after a single infusion. Required more than 1 infusion for complete resolution. NONE: No improvement or condition worsens. |
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Main Inclusion Criteria:
Main Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Colorado | Aurora | Colorado | 80045 | United States | ||
| University of Florida, College of Medicine |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 26157075 | Derived | Konkle BA, Stasyshyn O, Chowdary P, Bevan DH, Mant T, Shima M, Engl W, Dyck-Jones J, Fuerlinger M, Patrone L, Ewenstein B, Abbuehl B. Pegylated, full-length, recombinant factor VIII for prophylactic and on-demand treatment of severe hemophilia A. Blood. 2015 Aug 27;126(9):1078-85. doi: 10.1182/blood-2015-03-630897. Epub 2015 Jul 8. |
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Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
A total of 159 participants provided informed consent and were screened for study participation, of which there were 21 screen failures. 138 participants were assigned to the prophylactic arm or the on-demand treatment regimen.
Participants were enrolled (signed informed consent) at 72 sites.
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| ID | Title | Description |
|---|---|---|
| FG000 | Prophylaxis | Twice weekly at a dose of 45 ± 5 IU/kg |
| FG001 | On-demand | 10 to 60 ± 5 IU/kg |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| PEGylated Recombinant Factor VIII | Biological | Pharmacokinetic (PK) evaluation of BAX 855 |
|
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| PEGylated Recombinant Factor VIII | Biological | Prophylaxis treatment |
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| PEGylated Recombinant Factor VIII | Biological | On-demand treatment |
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| At least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm and 6 months (± 2 weeks) for the on-demand arm. |
| Average Number of BAX 855 Infusions Needed for the Treatment of Bleeding Episodes | From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm]. |
| Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes | Interval between Bleeds in months was calculated as: Observation period for efficacy (in days)/(number of bleeds)*(12/365.2425) | From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm]. |
| Weight-adjusted Consumption of BAX 855 - Per Prophylactic Infusion and Pharmacokinetic (PK) Infusion | Prophylactic Infusion: ≥50 exposure days or 6 months (±2 weeks), whichever occurs last. PK Infusion: PK #1 Pre-infusion within 30 minutes; Post-infusion 10 min, and 0.5, 1, 3, 6, 24, 32, 48, 56 hours (h). PK #2 also at Post-infusion 96h |
| Weight-adjusted Consumption of BAX 855 - Per Treatment of Bleeding Episode (BE) and Per BE for Maintenance of Hemostasis | Infusions per bleeding episode for maintenance of hemostasis only includes infusions following the resolution of a bleed to maintain hemostasis. | Treatment of Bleeding Episode (BE): Minor/Moderate BE every 12 to 24 hours until bleeding is resolved; Major BE every 8 to 12 hours until bleeding is resolved. Per BE for Maintenance of Hemostasis: within 48 hours after bleeding episode resolution. |
| Percentage of Participants With Adverse Events | Adverse Events (AEs) and Serious Adverse Events (SAEs) | From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm]. |
| Immunogenicity - Number of Participants With Positive Inhibitory Antibodies to FVIII, Binding Antibodies to FVIII, PEG-VIII, PEG and Anti-CHO Antibodies at Study Completion/Termination | Number of participants who received BAX855, with immunogenicity data from study completion/termination visit. FVIII = factor VIII; PEG-VIII = polyethylene glycol-factor VIII; Anti-CHO = Anti-Chinese hamster ovary | From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm]. |
| Patient Reported Outcomes: Haemo-SYM Questionnaire, Change in Score From Baseline to End of Study | The HAEMO-SYM has two subscales: pain and bleeds. HAEMO-SYM subscale scores are calculated by taking the mean of the items in each subscale and transforming them to a 0 (none or absent) to 100 (very severe) scale. Given that higher scores indicate more severe symptoms on the Haemo-SYM and that the change scores were calculated as the value at study completion minus the value at baseline, a negative change score indicates an improvement (reduction in symptoms). Conversely, a positive change score indicates worsening symptoms. | Baseline; and end of study visit [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm and 6 months (± 2 weeks) for the on-demand arm]. |
| Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study | Change from Baseline to End of Study for SF-36 Questionnaire is provided. Scores for individual SF-36 categories range from 0 to 100 with higher scores representing better health. Given that higher scores indicate better health-related quality of life (HRQoL) and that the change scores were calculated as the value at study completion minus the value at baseline, a negative change score indicates a worsening of HRQoL. | Baseline; and end of study visit [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm and 6 months (± 2 weeks) for the on-demand arm] |
| Pharmacokinetics (Pk) - Plasma Half-life (One-stage Clotting Assay) | Terminal half-life calculated as log_e2/λz where λz is the terminal elimination rate constant. Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3). | Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only). |
| Pharmacokinetics (Pk) - Mean Residence Time (One-stage Clotting Assay) | The mean residence time (MRT) w as calculated as total area under the moment curve divided by the total area under the curve starting from the begin of infusion (or the end of infusion if start time is not available). Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3). | Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only). |
| Pharmacokinetics (Pk) - Total Body Clearance (One-stage Clotting Assay) | Clearance in dL/(kg.h) will be calculated as the dose in IU/kg divided by the total area under the curve starting from the begin of infusion (or the end of infusion if start time is not available). Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3). | Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only). |
| Pharmacokinetics (Pk) - Incremental Recovery Over Time (One-stage Clotting Assay) | Incremental recovery (IR) in (IU/dL)/ (IU/kg) calculated as: IR = (Cmax- (C pre-infusion)) / (Dose/kg), where C =concentration. Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3). | Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only). |
| Pharmacokinetics (Pk) - Area Under the Concentration Versus Time Curve From 0 to Infinity (AUC0-∞) (One-stage Clotting Assay) | Calculated by WinNonlin NCA (Model 201, calculation method: Linear Trapezoidal Linear/Log Interpolation). Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3). | Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only). |
| Pharmacokinetics (Pk) - Apparent Volume of Distribution at Steady State (Vss) (One-stage Clotting Assay) | The apparent volume of distribution at steady state (Vss) will be calculated as: Vss = Clearance * Mean Residence Time. Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3). | Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only). |
| Pharmacokinetics (Pk) - Maximum Plasma Concentration (Cmax) (One-stage Clotting Assay) | Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3). | Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only). |
| Pharmacokinetics (Pk) -Time to Maximum Concentration in Plasma (Tmax) (One-stage Clotting Assay) | Tmax in hours will be defined as the time to reach Cmax. Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3). | Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only). |
| Change in Vital Signs From Screening - Temperature | Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination |
| Change in Vital Signs From Screening - Pulse Rate | Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination |
| Change in Vital Signs From Screening - Respiratory Rate | Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination |
| Changes in Vital Signs From Screening - Blood Pressure | Systolic Blood Pressure (SBP) Diastolic Blood Pressure (DBP) | Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination |
| Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein | Screening, week 2, week 4, month 3, study completion/termination |
| Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase | Alkaline Phosphatase (Alk Phos); Alanine Aminotransferase (Ala Amino); Aspartate Aminotransferase (Asp Amino) | Screening, week 2, week 4, month 3, study completion/termination |
| Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN) | Screening, week 2, week 4, month 3, study completion/termination |
| Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin | Screening, week 2, week 4, month 3, study completion/termination |
| Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes | Screening, week 2, week 4, month 3, study completion/termination |
| Changes in Hematology Laboratory Assessments From Screening - Hematocrit | Screening, week 2, week 4, month 3, study completion/termination |
| Changes in Hematology Laboratory Assessments From Screening - Hemoglobin | Screening, week 2, week 4, month 3, study completion/termination |
| Changes in Hematology Laboratory Assessments From Screening - Erythrocytes | Screening, week 2, week 4, month 3, study completion/termination |
| Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL) | Screening, week 2, week 4, month 3, study completion/termination |
| Gainesville |
| Florida |
| 32610 |
| United States |
| Children's Healthcare of Atlanta | Atlanta | Georgia | 30322 | United States |
| Bleeding and Clotting Disorders Institute | Peoria | Illinois | 61614 | United States |
| University of Kentucky Medical Center | Lexington | Kentucky | 40504 | United States |
| University of Louisville Hospital | Louisville | Kentucky | 40202 | United States |
| Tulane University Medical School | New Orleans | Louisiana | 70124 | United States |
| Children's Mercy Hospitals & Clinics | Kansas City | Missouri | 66211 | United States |
| Weill Cornell Medical College-New York Presbyterian Hospital | New York | New York | 10065 | United States |
| University of North Carolina Chapel Hill | Chapel Hill | North Carolina | 27599 | United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| The Cleveland Clinic Foundation | Cleveland | Ohio | 44195 | United States |
| Penn State Milton S. Hershey Medical Center | Hershey | Pennsylvania | 17033 | United States |
| Palmetto Health | Columbia | South Carolina | 29203 | United States |
| University of Utah Health Sciences Center | Salt Lake City | Utah | 84132 | United States |
| Puget Sound Blood Group | Seattle | Washington | 98104 | United States |
| Royal Adelaide Hospital | Adelaide | South Australia | 5000 | Australia |
| The Alfred Hospital | Clayton | Victoria | 3168 | Australia |
| Fremantle Hospital | Fremantle | Western Australia | 6160 | Australia |
| Hollywood Specialist Centre | Nedlands | Western Australia | 6009 | Australia |
| Landes-Frauen-und Kinderklinik Linz | Linz | 4020 | Austria |
| AKH - Medizinische Universität Wien | Vienna | 1090 | Austria |
| SHAT of Oncohaematology Diseases | Sofia | 1527 | Bulgaria |
| Fakultni nemocnice Brno | Brno | 61300 | Czechia |
| Fakultni nemocnice Olomouc | Olomouc | 775 20 | Czechia |
| Fakultni nemocnice v Motole | Prague | 150 06 | Czechia |
| Gerinnungszentrum Rhein-Ruhr | Duisburg | North Rhine-Westphalia | 47051 | Germany |
| Vivantes Klinikum im Friedrichshain - Landsberger Allee | Berlin | 10249 | Germany |
| Universitaetsklinikum Hamburg-Eppendorf | Hamburg | 20246 | Germany |
| Werlhof-Institut MVZ | Hanover | 30159 | Germany |
| Rambam Health Care Campus | Haifa | 3109601 | Israel |
| Chaim Sheba Medical Center | Tel Aviv | 64239 | Israel |
| Nagoya University Hospital | Nagoya | Aichi-ken | 466-8560 | Japan |
| University of Occupational and Environmental Health Hospital | Kitakyushu-shi | Fukuoka | 807-8556 | Japan |
| Hiroshima University Hospital | Hiroshima | Hiroshima | 734-8551 | Japan |
| Hyogo College of Medicine Hospital | Nishinomiya-shi | Hyōgo | 663-8501 | Japan |
| St. Marianna University School of Medicine Hospital | Kawasaki-shi | Kanagawa | 216-8511 | Japan |
| Nara Medical University Hospital | Kashihara-shi | Nara | 634-8522 | Japan |
| Tokyo Medical University Hospital | Shinjuku-ku | Tokyo-To | 160-0023 | Japan |
| Ogikubo Hospital | Suginami-ku | Tokyo-To | 167-8515 | Japan |
| Vilnius University Hospital Santariskiu Clinics, Public Institution | Vilnius | 08661 | Lithuania |
| Children's Hospital, Affiliate of Vilnius University Hospital Santariskiu Klinikos | Vilnius | LT-08406 | Lithuania |
| Hospital Pulau Pinang | George Town | Pulau Pinang | 10990 | Malaysia |
| Hospital Tengku Ampuan Rahimah | Klang | Selangor | 41200 | Malaysia |
| Pusat Darah Negara | Kuala Lumpur | 50400 | Malaysia |
| Academisch Medisch Centrum | Amsterdam | 1105 AZ | Netherlands |
| Uniwersyteckie Centrum Kliniczne | Gdansk | 80-952 | Poland |
| Wojewodzki Szpital Specjalistyczny im.M.Kopernika w Lodzi | Lodz | 93-510 | Poland |
| Sanador SRL | Bucharest | 011026 | Romania |
| Chonnam National University Hwasun Hospital | Hwasun | Jeollanam-do | 519-763 | South Korea |
| Pusan National University Hospital | Busan | 602-739 | South Korea |
| Eulji University Hospital | Daejeon | 302-120 | South Korea |
| Kyung hee University Hospital at Gangdong | Seoul | 134-727 | South Korea |
| Ulsan University Hospital | Ulsan | 682-714 | South Korea |
| Hospital Universitari Son Espases | Palma de Mallorca | Balearic Islands | 07010 | Spain |
| Complejo Hospitalario Universitario A Coruña | A Coruña | La Coruña | 15006 | Spain |
| Hospital Regional Universitario de Malaga | Málaga | Málaga | 29010 | Spain |
| Hospital Universitari i Politecnic La Fe | Valencia | 46026 | Spain |
| Skånes Universitetssjukhus, Malmö | Malmö | 20502 | Sweden |
| Karolinska Universitetssjukhuset, Solna | Stockholm | 17176 | Sweden |
| Universitatsspital Zurich | Zurich | 8091 | Switzerland |
| Tri-Service General Hospital | Taipei | 11490 | Taiwan |
| SI V.K.Gusak Emergency and Reconstructive Surgery Institute of NAMSU Center of IT | Donetsk | 83045 | Ukraine |
| SI Institute of Blood Pathology and Transfusion Medicine of NAMSU | Lviv | 79044 | Ukraine |
| Bristol Royal Hospital for Children | Bristol | Avon | BS2 8BJ | United Kingdom |
| Royal Free Hospital | London | Greater London | NW3 2QG | United Kingdom |
| St Thomas' Hospital | London | Greater London | SE1 7EH | United Kingdom |
| Manchester Royal Infirmary | Manchester | Greater Manchester | M13 9WL | United Kingdom |
| Royal Manchester Children's Hospital | Manchester | Greater Manchester | M13 9WL | United Kingdom |
| Leicester Royal Infirmary | Leicester | Leicestershire | LE1 5WW | United Kingdom |
| Churchill Hospital | Oxford | Oxfordshire | OX3 7LJ | United Kingdom |
| COMPLETED |
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| NOT COMPLETED |
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All Study Participants
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| ID | Title | Description |
|---|---|---|
| BG000 | Prophylaxis | |
| BG001 | On-demand | |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
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| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
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| Age, Continuous | Mean | Standard Deviation | Years |
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| Sex: Female, Male | Count of Participants | Participants |
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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 | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Primary | Annualized Bleeding Rate (ABR) | Comparisons between prophylactic and on-demand treatment were based on ABR estimates from a negative binomial regression model, taking into account the treatment regimen, target joints and age at screening, and duration of the observation period for efficacy. | Full Analysis Set | Posted | Least Squares Mean | 95% Confidence Interval | Bleeds per year | 9 months |
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| Secondary | Rate of Success of BAX 855 for Treatment of Bleeding Episodes | Success in the control of bleeding was defined as a rating of excellent or good using the Efficacy Rating Scale for Treatment of Bleeding Episodes measured 24 hours after initiation of treatment for the bleeding episode. EXCELLENT: Full relief of pain and cessation of objective signs of bleeding (eg, swelling, tenderness, and decreased range of motion in the case of musculoskeletal hemorrhage) after a single infusion. No additional infusion is required for the control of bleeding. Administration of further infusions to maintain hemostasis would not affect this scoring. GOOD: Definite pain relief and/or improvement in signs of bleeding after a single infusion. Possibly requires more than 1 infusion for complete resolution. FAIR: Probable and/or slight relief of pain and slight improvement in signs of bleeding after a single infusion. Required more than 1 infusion for complete resolution. NONE: No improvement or condition worsens. | Full Analysis Set - All bleeding episodes treated with BAX 855 in participants on on-demand and prophylaxis treatment regimens were analyzed as a single group. | Posted | Number | 95% Confidence Interval | Bleeding episodes | At least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm and 6 months (± 2 weeks) for the on-demand arm. | Bleeding episodes | Bleeding episodes |
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| Secondary | Average Number of BAX 855 Infusions Needed for the Treatment of Bleeding Episodes | Participants from the Full Analysis Set who experienced at least one bleeding episode. | Posted | Mean | Standard Deviation | Infusions | From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm]. |
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| Secondary | Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes | Interval between Bleeds in months was calculated as: Observation period for efficacy (in days)/(number of bleeds)*(12/365.2425) | Study participants from the Full Analysis Set (FAS) who received BAX855 during the study period. Note: one participant was assigned to the prophylactic arm (thus was included in the FAS) and received only ADVATE during the screening period. | Posted | Number | Participants | From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm]. |
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| Secondary | Weight-adjusted Consumption of BAX 855 - Per Prophylactic Infusion and Pharmacokinetic (PK) Infusion | Full Analysis Set (FAS) - Note: data analyzed by subsets of FAS (1) participants who received BAX855 prophylactic infusion or (2) BAX855 pharmacokinetic (PK) participants. - Subset of participants who received BAX855 prophylactic infusion: N= 120 - Subset of BAX855 pharmacokinetic (PK) participants: N=26 | Posted | Mean | Standard Deviation | IU/kg | Prophylactic Infusion: ≥50 exposure days or 6 months (±2 weeks), whichever occurs last. PK Infusion: PK #1 Pre-infusion within 30 minutes; Post-infusion 10 min, and 0.5, 1, 3, 6, 24, 32, 48, 56 hours (h). PK #2 also at Post-infusion 96h | Infusions | Infusions |
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| Secondary | Weight-adjusted Consumption of BAX 855 - Per Treatment of Bleeding Episode (BE) and Per BE for Maintenance of Hemostasis | Infusions per bleeding episode for maintenance of hemostasis only includes infusions following the resolution of a bleed to maintain hemostasis. | Full Analysis Set (FAS) - Note: data analyzed by subsets of FAS (1) participants who received BAX855 for treatment of BEs (2) BAX855 for Maintenance of Hemostasis. - Subset of participants who received BAX855 for treatment of BEs: N= 92 - Subset BAX855 for Maintenance of Hemostasis participants: N=16 | Posted | Mean | Standard Deviation | IU/kg | Treatment of Bleeding Episode (BE): Minor/Moderate BE every 12 to 24 hours until bleeding is resolved; Major BE every 8 to 12 hours until bleeding is resolved. Per BE for Maintenance of Hemostasis: within 48 hours after bleeding episode resolution. | Bleeds | Bleeds |
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| Secondary | Percentage of Participants With Adverse Events | Adverse Events (AEs) and Serious Adverse Events (SAEs) | Safety Analysis Set (SAS) - All participants treated with BAX 855 were analyzed as a single group (ie on-demand and prophylaxis treatment regimens were analyzed as a single group). | Posted | Number | percent of participants | From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm]. |
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| Secondary | Immunogenicity - Number of Participants With Positive Inhibitory Antibodies to FVIII, Binding Antibodies to FVIII, PEG-VIII, PEG and Anti-CHO Antibodies at Study Completion/Termination | Number of participants who received BAX855, with immunogenicity data from study completion/termination visit. FVIII = factor VIII; PEG-VIII = polyethylene glycol-factor VIII; Anti-CHO = Anti-Chinese hamster ovary | Safety Analysis Set (SAS) - who received BAX855 during the study period. Note: one participant was assigned to the prophylactic arm but did not receive BAX855 (only received ADVATE, during the screening period). | Posted | Number | Participants | From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm]. |
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| Secondary | Patient Reported Outcomes: Haemo-SYM Questionnaire, Change in Score From Baseline to End of Study | The HAEMO-SYM has two subscales: pain and bleeds. HAEMO-SYM subscale scores are calculated by taking the mean of the items in each subscale and transforming them to a 0 (none or absent) to 100 (very severe) scale. Given that higher scores indicate more severe symptoms on the Haemo-SYM and that the change scores were calculated as the value at study completion minus the value at baseline, a negative change score indicates an improvement (reduction in symptoms). Conversely, a positive change score indicates worsening symptoms. | Full Analysis Set - Subset of participants with both baseline and study completion HAEMO-SYM scores | Posted | Mean | Standard Deviation | Score on a scale | Baseline; and end of study visit [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm and 6 months (± 2 weeks) for the on-demand arm]. |
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| Secondary | Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study | Change from Baseline to End of Study for SF-36 Questionnaire is provided. Scores for individual SF-36 categories range from 0 to 100 with higher scores representing better health. Given that higher scores indicate better health-related quality of life (HRQoL) and that the change scores were calculated as the value at study completion minus the value at baseline, a negative change score indicates a worsening of HRQoL. | Full Analysis Set - Subset of participants with both baseline and study completion SF-36 scores | Posted | Mean | Standard Deviation | Score on a scale | Baseline; and end of study visit [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm and 6 months (± 2 weeks) for the on-demand arm] |
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| Secondary | Pharmacokinetics (Pk) - Plasma Half-life (One-stage Clotting Assay) | Terminal half-life calculated as log_e2/λz where λz is the terminal elimination rate constant. Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3). | Pharmacokinetic full analysis set (PKFAS) | Posted | Mean | Standard Deviation | hours | Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only). |
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| Secondary | Pharmacokinetics (Pk) - Mean Residence Time (One-stage Clotting Assay) | The mean residence time (MRT) w as calculated as total area under the moment curve divided by the total area under the curve starting from the begin of infusion (or the end of infusion if start time is not available). Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3). | Pharmacokinetic full analysis set (PKFAS) | Posted | Mean | Standard Deviation | hours | Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only). |
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| Secondary | Pharmacokinetics (Pk) - Total Body Clearance (One-stage Clotting Assay) | Clearance in dL/(kg.h) will be calculated as the dose in IU/kg divided by the total area under the curve starting from the begin of infusion (or the end of infusion if start time is not available). Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3). | Pharmacokinetic full analysis set (PKFAS) | Posted | Mean | Standard Deviation | dL/(kg*hours) | Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only). |
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| Secondary | Pharmacokinetics (Pk) - Incremental Recovery Over Time (One-stage Clotting Assay) | Incremental recovery (IR) in (IU/dL)/ (IU/kg) calculated as: IR = (Cmax- (C pre-infusion)) / (Dose/kg), where C =concentration. Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3). | Pharmacokinetic full analysis set (PKFAS) | Posted | Mean | Standard Deviation | (IU/dL)/(IU/kg) | Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only). |
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| Secondary | Pharmacokinetics (Pk) - Area Under the Concentration Versus Time Curve From 0 to Infinity (AUC0-∞) (One-stage Clotting Assay) | Calculated by WinNonlin NCA (Model 201, calculation method: Linear Trapezoidal Linear/Log Interpolation). Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3). | Pharmacokinetic full analysis set (PKFAS) | Posted | Mean | Standard Deviation | (IU*hours)/dL | Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only). |
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| Secondary | Pharmacokinetics (Pk) - Apparent Volume of Distribution at Steady State (Vss) (One-stage Clotting Assay) | The apparent volume of distribution at steady state (Vss) will be calculated as: Vss = Clearance * Mean Residence Time. Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3). | Pharmacokinetic full analysis set (PKFAS) | Posted | Mean | Standard Deviation | dL/kg | Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only). |
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| Secondary | Pharmacokinetics (Pk) - Maximum Plasma Concentration (Cmax) (One-stage Clotting Assay) | Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3). | Pharmacokinetic full analysis set (PKFAS) | Posted | Mean | Standard Deviation | IU/dL | Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only). |
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| Secondary | Pharmacokinetics (Pk) -Time to Maximum Concentration in Plasma (Tmax) (One-stage Clotting Assay) | Tmax in hours will be defined as the time to reach Cmax. Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3). | Pharmacokinetic full analysis set (PKFAS) | Posted | Mean | Standard Deviation | hours | Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only). |
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| Secondary | Change in Vital Signs From Screening - Temperature | Safety Analysis Set | Posted | Median | Inter-Quartile Range | Celsius | Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination |
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| Secondary | Change in Vital Signs From Screening - Pulse Rate | Safety Analysis Set | Posted | Median | Inter-Quartile Range | beats per minute | Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination |
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| Secondary | Change in Vital Signs From Screening - Respiratory Rate | Safety Analysis Set | Posted | Median | Inter-Quartile Range | breaths per minute | Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination |
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| Secondary | Changes in Vital Signs From Screening - Blood Pressure | Systolic Blood Pressure (SBP) Diastolic Blood Pressure (DBP) | Safety Analysis Set | Posted | Median | Inter-Quartile Range | mmHg | Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination |
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| Secondary | Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein | Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data. | Posted | Median | Inter-Quartile Range | g/L | Screening, week 2, week 4, month 3, study completion/termination |
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| Secondary | Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase | Alkaline Phosphatase (Alk Phos); Alanine Aminotransferase (Ala Amino); Aspartate Aminotransferase (Asp Amino) | Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data. | Posted | Median | Inter-Quartile Range | Units per Liter | Screening, week 2, week 4, month 3, study completion/termination |
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| Secondary | Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN) | Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data. | Posted | Median | Inter-Quartile Range | mmol/L | Screening, week 2, week 4, month 3, study completion/termination |
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| Secondary | Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin | Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data. | Posted | Median | Inter-Quartile Range | µmol/L | Screening, week 2, week 4, month 3, study completion/termination |
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| Secondary | Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes | Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data. | Posted | Median | Inter-Quartile Range | giga (10^9) cells per liter (Gi/L) | Screening, week 2, week 4, month 3, study completion/termination |
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| Secondary | Changes in Hematology Laboratory Assessments From Screening - Hematocrit | Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data. | Posted | Median | Inter-Quartile Range | Percentage red blood cells | Screening, week 2, week 4, month 3, study completion/termination |
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| Secondary | Changes in Hematology Laboratory Assessments From Screening - Hemoglobin | Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data. | Posted | Median | Inter-Quartile Range | g/L | Screening, week 2, week 4, month 3, study completion/termination |
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| Secondary | Changes in Hematology Laboratory Assessments From Screening - Erythrocytes | Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data. | Posted | Median | Inter-Quartile Range | TI/L | Screening, week 2, week 4, month 3, study completion/termination |
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| Secondary | Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL) | Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data. | Posted | Median | Inter-Quartile Range | mmol/L | Screening, week 2, week 4, month 3, study completion/termination |
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From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].
All study participants were analyzed in a single arm/group.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | All Study Participants | All study participants were analyzed in a single arm/group. | 5 | 137 | 26 | 137 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| HERPES ZOSTER INFECTION NEUROLOGICAL | Infections and infestations | MedDRA |
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| HUMERUS FRACTURE | Injury, poisoning and procedural complications | MedDRA |
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| MUSCLE HAEMORRHAGE | Musculoskeletal and connective tissue disorders | MedDRA |
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| OSTEOARTHRITIS | Musculoskeletal and connective tissue disorders | MedDRA |
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| NEUROENDOCRINE CARCINOMA | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nasopharyngitis | Infections and infestations | MedDRA |
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| Upper respiratory tract infection | Infections and infestations | MedDRA |
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| Headache | Nervous system disorders | MedDRA |
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Baxalta's agreements with PIs may vary per individual PI, but contain common elements. For this study, PIs may be restricted from independently publishing results without prior approval.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Shire | +1 866 842 5335 | ClinicalTransparency@shire.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 |
Not provided
Not provided
| ID | Term |
|---|---|
| D005169 | Factor VIII |
| C000609799 | BAX 855 |
| ID | Term |
|---|---|
| D001779 | Blood Coagulation Factors |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D011498 | Protein Precursors |
| D001685 | Biological Factors |
Not provided
Not provided
| Male |
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