Not yet recruiting

N/A

3

 

Curtin University, Rocky Bay Limited

Rare Disease

 

 

Not yet recruiting

N/A

249

 

Monash University, Monash University, F. Hoffmann-La Roche Ltd

Multiple Sclerosis, Neuromyelitis Optica Spectrum Disorder

 

 

N/A

25

 

John Hunter Hospital of the Hunter New England Local Health District, Merck Healthcare Pty. Ltd

Pregnancy, Multiple Sclerosis, sub-fertility, Neuromyelitis Optica Spectrum Disorders

 

 

Not yet recruiting

N/A

135

 

emicizumab ;FVIII prophylaxis

Beijing Children's Hospital, Capital Medical University; Beijing Children's Hospital, Capital Medical University, NO

Hemophilia

 

 

Completed

N/A

48

 

anal distention ;Resection of the left colon

Pediatric surgery of Tongji Hospital, Wuhan; Tongji Hospital, Hospital fund of Tongji hospital of Huazhong university of science and technology

Hirschsprung disease

 

 

Completed

N/A

60

 

tranexamic acid to control bleeding after ultrasonic scaling ;concentrate factor VIII to control bleeding after ultrasonic scaling

Peking Union Medical College Hospital (PUMCH) China Academy of Medical Science (CAMS); Peking Union Medical College Hospital (PUMCH) China Academy of Medical Science (CAMS)& Peking Union Medical College (PUMC), Bayer Hemophilia Awards

Hemophilia

 

 

Completed

N/A

90

 

Progressive full-thickness myotomy ;Full-thickness myotomy in combination with indwelling anti-reflux belt ;Circular muscle myotomy in combination with balloon shaping

Digestive Endoscopic Centre, Department of Gastroenterology and Hepatology. The Chinese PLA General Hospital.; Digestive Endoscopic Centre, Department of Gastroenterology and Hepatology. The Chinese PLA General Hospital., Self-funded

Achalasia

 

 

Completed

N/A

101

 

modified stent 2 days ;modified stent 3 days ;balloon dilatation ;classic stent

Renji Hospital, Shanghai Jiao Tong University School of Medicine; Renji Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai Municipal Health Bureau Scientific Research Project（20114321）

cardia achalasia

 

 

Recruiting

N/A

100

 

POEM ;PBD

Research Institute of Digestive Diseases, Southwest Hospital, Third Military Medical University; Level of the institution：, self-funded

Achalasia

 

 

Unknown status

N/A

 

Shenzhen Children's Hospital ultrasound department; Blood Diseases Hospital, Chinese Academy of Medical Sciences ultrasound department; Shenzhen children's Hospital, self-funded

hemophilia

 

 

Recruiting

N/A

185

 

Nil

Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College; Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, self-financing

Acquired Hemophilia

 

 

Recruiting

N/A

80

 

ERAS strategy ;Traditional approach

Zhongshan hospital of Fudan University; Zhongshan hospital of Fudan University, research funding

esophageal achalasia

 

 

Recruiting

N/A

20

 

no

Department of Clinical Pharmacology, Xiangya Hospital; Department of Clinical Pharmacology, Xiangya Hospital, Health and Family Planning Commission of Hunan Province and The national development and reform commission of hunan province funding suport

Familial Idiopathic Basal Ganglia Calcification

 

 

Completed

N/A

1000

 

EULAR+Chinese medicine ;EULAR treatment guidline

Huadong Hospital; Huadong Hospital, Hospital development center of Shanghai

Behcet’s Disease

 

 

Recruiting

N/A

50

 

DA chemotherapy regimen plus G-CSF

Tianjin Medical University General Hospital; Tianjin Medical University General Hospital, self-financing

paroxymal nocturnal hemoglobinuria

 

 

Recruiting

N/A

1000

US

Clinical care and quality improvement

Epilepsy Foundation of America

Epilepsy, Seizure Disorder, Neurologic Disorder, Rare Diseases

02/99

02/99

Recruiting

N/A

100

 

Oxycodone ;Morphine

Department of Anesthesiology of the First Affiliated Hospital, College of Medicine, Zhejiang University; the First Affiliated Hospital, College of Medicine, Zhejiang University, self-raised

Achalasia of cardia and Upper gastrointestinal tumor

 

 

Recruiting

N/A

164

 

Five in one therapy ;Using FVIII / FIX

Shaanxi Yida Hemophilia Institute affiliated Xi’an Xincheng Yida Hospital; Shaanxi Yida Hemophilia Institute affiliated Xi'an Xincheng Yida Hospital, Shaanxi Yida Hemophilia Institute fund

Haemophilic arthritis

 

 

Recruiting

N/A

132

 

Patients with achalasia underwent PD ;Patients with achalasia underwent POEM

Tianjin Medical University General Hospital; Tianjin Medical University General Hospital, none

Achalasia

 

 

Recruiting

N/A

300

Europe

Presence of polymorphisms

Augusto Vaglio

Erdheim-Chester Disease

07/24

10/24

Recruiting

N/A

100

 

Liu-POEM ;conventional POEM

the second affiliated hospital of Harbin Medical University; the second affiliated hospital of Harbin Medical University, self-funding

Achalasia

 

 

Recruiting

N/A

60

 

double-tunnel POEM ;single-tunnel POEM

Jiangsu Province Hospital; Jiangsu Province Hospital, National Natural Science Foundation 81770561

Achalasia

 

 

Not yet recruiting

N/A

6000

 

None

Zhongda Hospital affiliated of Southeast University; Zhongda Hospital Affiliated of Southeast University, None

Calciphylaxis

 

 

Recruiting

N/A

13

 

None

Guangzhou University of Traditional Chinese Medicine First Affiliated Hospital; Guangzhou University of Traditional Chinese Medicine First Affiliated Hospital, self-funding

hemophilia arthropathy

 

 

Not yet recruiting

N/A

600000000

 

Nil

Department of Epidemiology and Statistics, School of Public Health, Peking University; Department of Epidemiology and Statistics, School of Public Health, Peking University, self-sponsored

rare disease

 

 

Completed

N/A

1

RoW

LDH/albumin ratio

Ataturk University

Behcet's Disease, Albumin, Lactate Dehydrogenase, Disease Severity

04/23

04/23

Not yet recruiting

N/A

70

 

perform POEM operation and achieve esophageal muscle specimens ;upper gastrointestinal contrast

Tianjin Medical University General Hospital; Tianjin Medical University General Hospital, no

Achalasia

 

 

Not yet recruiting

N/A

50

 

Joint replacement

The First Affiliated Hospital of Guangzhou University of Traditional Chinese Medicine; 广州中医药大学第一附属医院, Department of Orthopedics, the First Affiliated Hospital of Guangzhou University of Chinese Medicine

Hemophilia arthritis

 

 

Active, not recruiting

N/A

33

US

In-home body weight support harness system, The Portable Mobility Aid for Children (PUMA)

Nationwide Children's Hospital, Novartis

Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II

06/20

12/23

Not yet recruiting

N/A

 

Institute of Hematology and Blood Diseases Hospital, CAMS & PUMC; Institute of Hematology and Blood Diseases Hospital, CAMS & PUMC, Self-raised funds

Hemophilia A

 

 

Completed

N/A

114

RoW

Per-oral endoscopic myotomy

Asian Institute of Gastroenterology, India

GERD

07/20

10/20

Recruiting

N/A

70

Europe

Investigation of BRAF mosaicism

Meyer Children's Hospital IRCCS

Erdheim-Chester Disease

09/24

12/24

No Longer Available

N/A

US

rivogenlecleucel, BPX-501 T cells, rimiducid, AP1903, Rimiducid for Injection, AP1903 for Injection

Bellicum Pharmaceuticals

Hurler Syndrome, Inherited Metabolic Disorder, Lysosomal Storage Disorder, Metachromatic Leukodystrophy, Inborn Errors of Metabolism

 

 

Not yet recruiting

N/A

90

 

Nil

Children's Hospital of Fudan University; Children's Hospital of Fudan University, Biogen Biotechnology (Shanghai) Co., Ltd support

Spinal Muscular Atrophy

 

 

Recruiting

N/A

750

US

Cystinosis Research Foundation

Cystinosis, Nephropathic Cystinosis, Renal Fanconi Syndrome

12/20

12/22

Enrolling by invitation

N/A

30

RoW

PEMF

Delta University for Science and Technology

Hemophilia A

12/20

12/20

Recruiting

N/A

120

 

Peroral endoscopic anterior myotomy ;Peroral endoscopic right-posterior myotomy

the Second XiangYa Hospital of Central South University; the Second XiangYa Hospital of Central South University, the Development and Reform Commission of Hunan Province (XFGTZ2014713).

achalasia

 

 

Recruiting

N/A

2000

 

Nil

West China Hospital, Sichuan University; West China Hospital, Sichuan University, 1.The National Key Research Development Program of China 2.Self-financing

Primary dystonia

 

 

Not yet recruiting

N/A

124

 

open peroral endoscopic myotomy (O-POEM) ;traditional tunneling peroral endoscopic myotomy (POEM)

West China Hospital, Sichuan University; West China Hospital, Sichuan University, None

Achalasia

 

 

Withdrawn

N/A

1000

Europe, RoW

CENTOGENE GmbH Rostock

Lysosomal Storage Disease, Lung Diseases, Obstructive Sleep Apnoea, Macroglossia, Eye Abnormalities

02/21

02/21

Completed

N/A

100

 

Nil

Affiliated Hospital of Guizhou Medical University; Affiliated Hospital of Guizhou Medical University, Guizhou Provincial Children's Hematological Tumor Clinical Research Center (Guizhou Provincial Department of Science and Technology)

hemophilia A

 

 

No Longer Available

N/A

RoW

Nusinersen, ISIS 396443, BIIB058, Spinraza

Biogen

Infantile-onset Spinal Muscular Atrophy

 

 

No Longer Available

N/A

US

OBI-1, B-Domain Deleted Recombinant Porcine Factor VIII

Baxalta now part of Shire

Acquired Hemophilia A

 

 

Completed

N/A

10

Europe

Phenylalanine-free protein substitute in tablet form (XPhe minis)

metaX Institut fuer Diatetik GmbH, Birmingham Children's Hospital

Phenylketonurias, Hyperphenylalaninaemia, Tetrahydrobiopterin Deficiency

05/21

05/21

Not yet recruiting

N/A

60

 

;Thalidomide

The First Affiliated Hospital of Heilongjiang Chinese Medicine Colledge; The First Affiliated Hospital of Heilongjiang University of Traditional Chinese Medicine, Self sponsored and department sponsored

Behcet's syndrome

 

 

Recruiting

N/A

669

 

No

Division of Nephrology, Department of Medicine, Sichuan University West China Hospital; West China Hosptial, administration

acute kidney injury

 

 

Available

N/A

NA

Pegcetacoplan

Apellis Pharmaceuticals, Inc.

C3G, IC-MPGN, C3 Glomerulopathy, C3 Glomerulonephritis, Complement 3 Glomerulopathy, Complement 3 Glomerulopathy (C3G), Complement 3 Glomerulonephritis, Dense Deposit Disease, DDD, Membranoproliferative Glomerulonephritis, Membranoproliferative Glomerulonephritis (MPGN), Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN)

 

 

Recruiting

N/A

72

 

different treatments

Nanfang Hospital, Southern Medical University, Guangzhou, China; Nanfang Hospital, Southern Medical University, scientific research funds on high-level university construction

hemophilia A

 

 

Recruiting

N/A

124

 

none

Department of Rheumatology and Immunology, West China Hospital, Sichuan University; West China Hospital, Sichuan University, None

Behcet's disease

 

 

Not yet recruiting

N/A

 

non

Ruijin Hospital Shanghai Jiaotong University School of Medicine; Ruijin Hospital, research funding

Bleeding disease