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| ID | Type | Description | Link |
|---|---|---|---|
| 2022-001246-38 | EudraCT Number |
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The BMN 270 clinical development program consists of multiple interventional studies designed to assess the safety and efficacy of a single infusion of BMN 270 for at least 5 years post-infusion. This long-term follow-up study is needed to help further understand the long-term safety of BMN 270 beyond 5 years and to assess the durability of efficacy.
Study 270-401 will collect additional follow-up data in a single study for approximately 10 years among all subjects who consent to participate and have completed their primary treatment study (ie, for any study in which they received BMN 270).
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| Measure | Description | Time Frame |
|---|---|---|
| Evaluate the long-term safety of BMN 270 | Occurrence of adverse drug reactions, serious adverse events, and events of special interest. | Duration of study (10 years) |
| Measure | Description | Time Frame |
|---|---|---|
| Evaluate the long-term effects of BMN 270 in subjects with hemophilia A previously treated in a BioMarin clinical trial | Changes in annualized bleeding rate (ABR) (treated bleeds and all bleeds) and FVIII activity measured over time (CSA and OSA) | Duration of study (10 years) |
| Evaluate the use of hemostatic agents (ie, emicizumab) |
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Inclusion Criteria:
Exclusion Criteria:
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Subjects must have completed their primary treatment study in which they were dosed BMN 270. Subjects may enroll in 270-401 even if they have restarted FVIII prophylaxis or other hemophilia A treatment.
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director, MD | BioMarin Pharmaceutical | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Orthopedic Institute for Children, Orthopedic Hemophilia Treatment Center | Los Angeles | California | 90007 | United States | ||
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Annualized use of concomitant hemostatic medications (annualized FVIII utilization and annualized FVIII infusion rate) |
| Duration of study (10 years) |
| Evaluate the long-term impact of BMN 270 on HRQoL | Changes in Haemo-QOL-A | Duration of study (10 years) |
| UC Davis Hemophilia Treatment Center |
| Sacramento |
| California |
| 95817 |
| United States |
| University of California San Diego, Hemophilia & Thrombosis Treatment Center | San Diego | California | 92121 | United States |
| University of California San Francisco - Moffit Hospital, Outpatient Hematology Clinic | San Francisco | California | 94143 | United States |
| UCN Hemophilia and Thrombosis Center | Shandon | California | 27517 | United States |
| Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical Campus | Aurora | Colorado | 80045 | United States |
| St Joseph's Children's Hospital, Center for Bleeding and Clotting Disorders | Tampa | Florida | 33614 | United States |
| University Medical Center, Inc,; DBA University of Louisville Hospital, James Graham Brown Cancer Center | Louisville | Kentucky | 40202-3229 | United States |
| University of Michigan, Pediatric Hematology and Oncology | Ann Arbor | Michigan | 48109-5718 | United States |
| Children's Hospital of Michigan, Hemophilia and Thrombosis Center | Detroit | Michigan | 48201 | United States |
| Washington University School of Medicine, Department of Pediatrics, Division of Hematology/Oncology | St Louis | Missouri | 63110-1093 | United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
| Hemophilia Center of Western Pennsylvania | Pittsburgh | Pennsylvania | 15213 | United States |
| Royal Prince Alfred Hospital, Institute of Haematology | Sydney | New South Wales | 2050 | Australia |
| Royal Brisbane and Women's Hospital | Herston | Queensland | 4029 | Australia |
| Royal Adelaide Hospital | Adelaide | South Australia | 5000 | Australia |
| The Alfred Hospital | Melbourne | Victoria | 3004 | Australia |
| Fiona Stanley Hospital | Murdoch | Western Australia | 6150 | Australia |
| University Hospitals Leuven, Campus Gasthuisberg, Department of Heart and Vascular Disease | Leuven | 3000 | Belgium |
| Holy Spirit Hematology and Hemotherapy Center | Vitória | Espírito Santo | 29040-090 | Brazil |
| Arthur De Siqueira Cavalcanti Hematology State Institute / Coordinating Hemocenter | Rio de Janeiro | Rio de Janeiro | 24000-000 | Brazil |
| Campinas University Clinical Hospital, Hematology and Hemotherapy Center (Hemocentro UNICAMP) | Campinas | São Paulo | 13083-878 | Brazil |
| Sao Paulo University Clinical Hospital | São Paulo | São Paulo | 05403 000 | Brazil |
| CHRU Lille Hospital Center Heart-Lung Institute | Lille | 59000 | France |
| CHU La Timone - La Timone Children's Hospital | Marseille | 13005 | France |
| University Hospital Bonn, Institute of Experimental Hematology and Transfusion Medicine, Hemophilia Care Center | Bonn | North Rhine-Westphalia | 53127 | Germany |
| Vivantes Hospital in Friedrichshain | Berlin | State of Berlin | 10249 | Germany |
| Chaim Sheba Medical Center, National Institute of Hemophilia and Coagulation | Ramat Gan | 5266202 | Israel |
| Maggiore Polyclinic Hospital, Foundation IRCCS Ca' Granda, Center for Hemophilia and Thrombosis "Angelo Bianchi Bonomi" | Milan | 20122 | Italy |
| Charlotte Maxeke Johannesburg Academic Hospital, Hemophilia Comprehensive Care Center | Johannesburg | 2193 | South Africa |
| Kyung Hee University Hospital at Gangdong | Seoul | 05278 | South Korea |
| University Hospital Virgen del Rocio (HUVR), Hematology Pediatrics | Seville | Andalusia | 41013 | Spain |
| Hospital Teresa Herrera, Department of Hematology and Hemotherapy, Unit of Hemostasis and Thrombosis | A Coruña | Galicia | 15006 | Spain |
| Addenbrooke's Hospital, Hemophilia and Thrombophilia Center | Cambridge | United Kingdom |
| Glasgow Royal Infirmary, Department of Hematology | Glasgow | G4 0SF | United Kingdom |
| Hammersmith Hospital, Clinical Research Facility, Centre for Translational and Experimental Medicine | London | United Kingdom |
| Queen Elizabeth Hospital Birmingham. Adult Hemophilia Centre, West Midlands Adult Comprehensive Care Hemophilia Centre | London | United Kingdom |
| Royal London Hospital, Barts and the London Hemophilia Center | London | United Kingdom |
| St Thomas' Hospital, Centre for Hemophilia, Hemostasis and Thrombosis | London | United Kingdom |
| Nuffield Orthopaedic Centre, Hemophilia & Thrombosis Center | Oxford | OX3 7LD | United Kingdom |
| University Hospital Southampton NHS Foundation Trust | Southampton | United Kingdom |
| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| D020141 | Hemostatic Disorders |
| D006402 | Hematologic Diseases |
| D001778 | Blood Coagulation Disorders |
| D025861 | Blood Coagulation Disorders, Inherited |
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| ID | Term |
|---|---|
| D006425 | Hemic and Lymphatic Diseases |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
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