主要目的 -通过与旁路制剂预防性治疗期间的出血频率进行比较,表征患者接受 fitusiran 治疗期间的出血频率。
次要目的 1)通过与 BPA 预防性治疗进行比较,表征患者接受 fitusiran 治疗期间的以下参数: -自发性出血频率 -关节出血频率 -患者的健康相关生活质量 (HRQOL) 2)表征患者在治疗的初始阶段的出血频率 3)表征 fitusiran 的安全性和耐受性
序号 |
机构名称 |
主要研究者 |
国家 |
省(州) |
城市 |
1 |
首都医科大学附属北京儿童医院 |
吴润晖 |
中国 |
北京市 |
北京市 |
2 |
Hospital Sultanah Aminah |
See Guan Toh |
马来西亚 |
NA |
Johor Bahru |
3 |
Nara Medical University Hospital |
Keiji Nogami |
日本 |
Nara |
Kashihara |
4 |
China Medical Univ Hospital |
Te-Fu Weng |
中国台湾 |
台湾 |
Taichung |
5 |
Severance Hospital Yonsei University Health System |
Seung Min Hahn |
韩国 |
NA |
首尔 |
6 |
State Institution Institute of Blood Pathology and Transfusion Medicine of NAMS of Ukraine |
Oleksandra Stasyshyn |
乌克兰 |
NA |
Lviv |
7 |
Kirov Research Institute of Haematology and Blood Transfusion |
Margarita Timofeeva |
俄罗斯 |
NA |
Kirov |
8 |
Groupement Hospitalier EST |
Claude Negrier |
法国 |
NA |
Lyon |
9 |
St. Petersburg City Outpatient Clinic #37 |
Tatyana Andreeva |
俄罗斯 |
NA |
St. Petersburg |
10 |
Tokyo Medical University Hospital |
Kagehiro Amano |
日本 |
Tokyo |
Tokyo |
11 |
Nagoya University Hospital |
Tadashi Matsushita |
日本 |
Aiti |
Nagoya |
12 |
Hospital of the University of Occupational and Environmental Health, Japan |
Koichi Oshida |
日本 |
NA |
Kita Kyushu-shi |
13 |
Ogikubo Hospital |
Takashi Suzuki |
日本 |
Tokyo |
Suginami |
14 |
Children's Hospital Los Angeles |
Guy Young |
美国 |
California |
Los Angeles |
15 |
Akdeniz University Medical Faculty |
Alphan Kupesiz |
土耳其 |
Antalya |
Antalya |
16 |
Gaziantep University Medical Faculty Sahinbey Research and Practice Hospital |
Vahap Okan |
土耳其 |
Gaziantep |
Gaziantep |
17 |
Queen Elizabeth Hospital |
Lily Wong |
马来西亚 |
Sabah |
Kota Kinabalu |
18 |
Taichung Veterans General Hospital |
Jiaan-Der Wang |
中国台湾 |
台湾 |
Taichung |
19 |
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico |
Elena Santagostino |
意大利 |
Lombardia |
Milano |
20 |
Royal Free Hospital |
Pratima Chowdary |
英国 |
NA |
London |
21 |
Royal Prince Alfred Hospital |
Teh-Liane Khoo |
澳大利亚 |
New South Wales |
Camperdown |
22 |
Hacettepe University Medical Faculty |
Salih Aksu |
土耳其 |
NA |
Ankara |
23 |
Hospital Universitario La Paz |
Victor Jimenez Yuste |
西班牙 |
NA |
Madrid |
24 |
Arkansas Children's Hospital |
Kimo Stine |
美国 |
Arkansas |
Little Rock |
25 |
Glasgow Royal Infirmary - PPDS |
Catherine Bagot |
英国 |
NA |
Glasgow |
26 |
Werlhof Institut |
Mario von Depka Prondzinski |
德国 |
NA |
Hannover |
27 |
Universitatsklinikum Frankfurt |
Wolfgang Miesbach |
德国 |
NA |
Frankfurt |
28 |
CIUSSS-de-l’Est-de-l’ le-de-Montreal |
Jean St.Louis |
加拿大 |
Quebec |
Montreal |
29 |
Hospital Ampang |
Jameela Sathar |
马来西亚 |
Selangor |
Ampang |
30 |
Kyiv City Clinical Hospital #9 |
Ievgenii Averianov |
乌拉圭 |
NA |
Kyiv |
31 |
Vivantes Klinikum Im Friedrichshain |
Robert Klamroth |
德国 |
Berlin |
Berlin |
32 |
Eul Ji University Hospital |
Chur-Woo You |
韩国 |
NA |
Daejeon |
33 |
Pusan National University Hospital |
Ho-Jin Shin |
韩国 |
韩国 |
Busan |
34 |
Chaim Sheba Medical Center |
Gili Kenet |
以色列 |
NA |
Ramat Gan |
35 |
Haemophilia Comprehensive Care Centre |
Johnny Mahlangu |
南非 |
Gauteng |
Parktown |
36 |
National Children's Specialized Hospital OHMATDYT of MoH of Ukraine |
Kateryna Vilchevska |
乌克兰 |
NA |
Kyiv |
37 |
Istanbul University Oncology Institute |
Bulent Zulfikar |
土耳其 |
NA |
Istanbul |
38 |
Alliance for Childhood Diseases |
Joseph Lasky |
美国 |
Nevada |
Las Vegas |
39 |
Acibadem Adana Hospital |
Ali Bulent Antmen |
土耳其 |
NA |
Adana |
40 |
Loma Linda University Children's Hospital |
Akshat Jain |
美国 |
California |
San Bernardino |
41 |
Kyung Hee University Hospital at Gangdong |
Young Shil Park |
韩国 |
NA |
Seoul |
42 |
Erciyes University Medical Faculty |
Turkan Patiroglu |
土耳其 |
NA |
Kayseri |
43 |
Changhua Christian Hospital |
Ming-Ching Shen |
中国台湾 |
NA |
Changhua |
44 |
Saitama Children's Medical Center |
Katsuyoshi Koh |
日本 |
Saitama |
Saitama |
45 |
Righospitalet Copenhagen University Hospital |
Peter Kampmann |
丹麦 |
NA |
Copenhagen |
46 |
Hyogo College of Medicine |
Satoshi Higasa |
日本 |
Hyogo |
Nishinomiya |
47 |
Our Lady's Children's Hospital - PIN |
Beatrice Nolan |
爱尔兰 |
NA |
Crumlin |
48 |
The Royal London Hospital |
John Pasi |
英国 |
NA |
London |
49 |
Guy's and St Thomas' NHS Foundation Trust - Guy's Hospital |
Steve Austin |
英国 |
NA |
London |
50 |
University of Texas Health Science Center at Houston |
Miguel Escobar |
英国 |
Texas |
Houston |
51 |
Taipei Medical University Hospital |
Chia-Yau Chang |
中国台湾 |
台湾 |
Taipei |
52 |
The Alfred Hospital |
Huyen Tran |
澳大利亚 |
Victoria |
Prahan |
53 |
Ondokuz Mayis Universitesi Tip Fakultesi Hastanesi |
Canan Albayrak |
土耳其 |
NA |
Samsun |
54 |
Ege Universitesi Tip Fakultesi Hastanesi |
Kaan Kavakli |
土耳其 |
NA |
Izmir |
55 |
Taipei Veterans General Hospital |
Liang-Tsai Hsiao |
中国台湾 |
台湾 |
Taipei |