Clinical Trials on Genetic Diseases, Inborn in China

Total 31698 results

Capital Medical University

Recruiting

Safety and Efficacy of Remote Ischemic Conditioning on Cerebral Amyloid Angiopathy. (RIC-CAA)

Cerebral Amyloid Angiopathy

China
Shanghai East Hospital
Shanghai Juvensis Therapeutics Biotechnology Company Limited

Recruiting

Myocardial Telomere Recapping Study for Dilated Cardiomyopathy (MERCURY-DCM)

Heart Failure | Dilated Cardiomyopathy

China
Chinese Academy of Medical Sciences, Fuwai Hospital

Recruiting

Cardiac Magnetic Resonance for Risk Stratification in Dilated Cardiomyopathy

Dilated Cardiomyopathy | Single-center Study

China
Sun Yat-sen University

Completed

Minocycline Treatment in Retinitis Pigmentosa

Retinitis Pigmentosa | Inherited Retinal Dystrophy | Retina Disorder

China
Peking Union Medical College Hospital

Completed

Interferon α2a Versus Cyclosporine for Refractory Behçet's Disease Uveitis

Uveitis | Behçet Disease

China
Huazhong University of Science and Technology
The First Affiliated Hospital with Nanjing Medical University; Wuhan University and other collaborators

Completed

Study of Qiliqiangxin Capsule to Treat Dilated Cardiomyopathy (QLQX-DCM)

Heart Failure | Dilated Cardiomyopathy

China
GeneScience Pharmaceuticals Co., Ltd.
Tongji Hospital; Shengjing Hospital; Shandong Provincial Hospital; Children's Hospital... and other collaborators

Recruiting

A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Achondroplasia

Achondroplasia

China
Wenjie Zheng

Terminated

Golimumab for the Treatment of Refractory Behcet's Uveitis

Behcet Syndrome | Uveitis

China
Wenjie Zheng

Terminated

Tocilizumab for the Treatment of Refractory Behcet's Uveitis

Behcet Syndrome | Uveitis

China
Shanghai BDgene Co., Ltd.
Ruijin Hospital

Not yet recruiting

Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent β-thalassemia

β-thalassemia

China
Shanghai Jiao Tong University School of Medicine

Completed

The Effect of Low Sodium Diet on Idiopathic Hyperaldosteronism

Primary Aldosteronism Due to Adrenal Hyperplasia (Bilateral)

China
Nanfang Hospital of Southern Medical University
Shenzhen Second People's Hospital; Shenzhen Children's Hospital; ZhuHai Hospital and other collaborators

Recruiting

Evaluation of Heart Status in Patients of Beta Thalassemia Using Echocardiogram

Beta Thalassemia

China
Children's Hospital of Fudan University
CorrectSequence Therapeutics Co., Ltd

Recruiting

Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With β-thalassemia Major

Beta-Thalassemia

China
CorrectSequence Therapeutics Co., Ltd
First Affiliated Hospital of Guangxi Medical University

Active, not recruiting

A Clinical Study Evaluating the Safety and Efficacy of CS-101 in Treating Subjects With β-thalassemia

Beta-Thalassemia

China
First Affiliated Hospital of Guangxi Medical University
Peking University People's Hospital; Ruijin Hospital; The Affiliated Hospital... and other collaborators

Recruiting

Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study

Thalassemia Major

China
Second Affiliated Hospital, School of Medicine,...

Not yet recruiting

Effectiveness of Ketogenic Diet in MELAS Syndrome

MELAS Syndrome | Ketogenic Dieting

China
Children's Hospital of Chongqing Medical University

Recruiting

Gene Therapy for X Linked Severe Combined Immunodeficiency

Gene Therapy

China
Wyeth is now a wholly owned subsidiary of Pfizer

Completed

Study Evaluating On-Demand Treatment With BeneFIX In Chinese Subjects

Hemophilia B

China
Nanfang Hospital of Southern Medical University
Xiangya Hospital of Central South University; Peking University People's Hospital and other collaborators

Unknown

Risk-stratified Therapy Based on Molecular Cytogenetic Aberration and Treatment Response in AML

MRD | Risk-directed Therapy | Cytogenetic Abnormality | Molecular Abnormality

China
Kyowa Kirin Co., Ltd.

Active, not recruiting

Study to Assess the Safety, Pharmacokinetics and Efficacy of KRN23 in Adult Chinese Patients With XLH

X-linked Hypophosphatemia (XLH)

China
Qingdao University

Unknown

Intramuscular Injection of Mesenchymal Stem Cell for Treatment of Children With Idiopathic Dilated Cardiomyopathy

Dilated Cardiomyopathy

China
Institute of Hematology & Blood Diseases Hospital...

Not yet recruiting

Safety and Efficacy Study of NGGT003 in Hemophilia A Patients

Hemophilia A

China
Suzhou Alphamab Co., Ltd.

Recruiting

KN057 Multiple Dose Study in Moderately Severe to Severe Hemophilia

Hemophilia

China
Staidson (Beijing) Biopharmaceuticals Co., Ltd

Unknown

A Safety Study of STSP-0601 in Adult Patients With Hemophilia A or B With Inhibitor

Hemophilia

China
Sinocelltech Ltd.
Cancer Institute and Hospital, Chinese Academy of Medical Sciences; Parexel; Q2...

Completed

Evaluation of SCT800 in Prophylaxis Therapy on Previous Treated Patients With Severe Hemophilia A

Hemophilia A

China
National Eye Institute (NEI)

Completed

Studies in Families With Corneal Dystrophy or Other Inherited Corneal Diseases

Corneal Dystrophies, Hereditary | Corneal Disease

United States, China, Italy
Kyowa Kirin Co., Ltd.

Active, not recruiting

Study to Assess the Safety, Pharmacokinetics and Efficacy of KRN23 in Pediatric Chinese Patients With XLH

X-linked Hypophosphatemia (XLH)

China
Affiliated Hospital of Jiangnan University

Completed

Effectiveness of Recombinant Human Growth Hormone Therapy for Children With PMS

Growth Hormone Treatment | Phelan-McDermid Syndrome

China
Reistone Biopharma Company Limited

Completed

A Phase II Study in Adult Patients With Moderate to Severe Atopic Dermatitis

Atopic Dermatitis

China
Institute of Hematology & Blood Diseases Hospital...
Shanghai Vitalgen BioPharma Co., Ltd.

Recruiting

Safety and Efficacy Evaluation of Autologous CRISPR-Cas12b Edited Hematopoietic Stem Cells

Thalassemia Major | Thalassemia, Beta

China
Shanghai BDgene Co., Ltd.
920th Hospital of Joint Logistics Support Force of People's Liberation Army...

Recruiting

A Study Evaluating the Safety and Efficacy of the BD211 Drug Product in β-Thalassemia Major Participants

Hematologic Diseases

China
Nanfang Hospital of Southern Medical University
Shenzhen Second People's Hospital; Shenzhen Children's Hospital; ZhuHai Hospital and other collaborators

Recruiting

Association of Hb F Level With Clinical Severity of Beta Thalassemia

Beta Thalassemia

China
Nanfang Hospital of Southern Medical University
303rd Hospital of the People's Liberation Army; Liuzhou Municipal Maternity... and other collaborators

Recruiting

Genetic Variants Affecting the Clinical Severity of Beta Thalassemia

Beta Thalassemia

China
First Affiliated Hospital of Guangxi Medical University

Unknown

Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major

Thalassemia Major

China
Nanfang Hospital of Southern Medical University

Unknown

Unrelated Umbilical Cord Blood Following HLA-haploidentical Hematopoietic Stem Cell Transplantation in Patients With β-thalassemia Major

Thalassemia Major

China
Tianjin Eye Hospital

Completed

The Characteristics of Backscattering With Depth in the Progression of Keratoconus

Elasticum, Incomplete Pseudoxanthoma

China
Essen Biotech

Not yet recruiting

Investigation Into the Use of BAH243 Lentiviral Vector for Gene Therapy in Treating Sickle Cell Disease (BAH243)

Sickle Cell Disease | Sickle-Cell Disease With Crisis

China
Bioray Laboratories
Xiangya Hospital of Central South University; The 923rd Hospital of Joint Logistics...

Completed

Safety and Efficacy Evaluation of γ-globin Reactivated Autologous Hematopoietic Stem Cells

β Thalassemia Major

China
Bioray Laboratories
PLA 923 Hospital

Terminated

β-globin Restored Autologous HSC in β-thalassemia Major Patients

β-thalassemia Major

China
Beijing Children's Hospital
Office for Rare Conditions Registries, University of Glasgow

Recruiting

The Efficacy and Safety of PEGylated GH for the Treatment of Short Stature in Chinese Children-GLOBE Reg

Growth Disorders | Growth Hormone Treatment

China
Guangdong Provincial People's Hospital

Recruiting

Observational Study for Patients With Dilated Cardiomyopathy

DCM - Dilated Cardiomyopathy

China
Peking Union Medical College Hospital

Unknown

Effect Observation Study of COX-2 Inhibitor to Treat Primary Hypertrophic Osteoarthropathy (EOSCITPHO)

Primary Hypertrophic Osteoarthropathy

China
Xue-chun Lu

Recruiting

Combination Regimen With Sodium Valproate for Severe Hemophilia: a Single-arm, Phase 1, Pilot Trial.

Hemophilia

China
Sinocelltech Ltd.

Recruiting

Safety and Efficacy of Long-term Treatment With SCT800 in Previously Treated Hemophilia A Patients.

Hemophilia A

China
Staidson (Beijing) Biopharmaceuticals Co., Ltd

Completed

A Safety and Efficacy Study of STSP-0601 in Adult Patients With Hemophilia A or B With Inhibitor

Hemophilia

China
Pfizer

Completed

Study of PF-05208756, Moroctocog Alfa (AF-CC), Xyntha For Male Chinese Subjects With Hemophilia A

Hemophilia A

China
Keymed Biosciences Co.Ltd

Completed

Dose Escalation Trial of CM310 in Patients With Moderate-to-Severe Atopic Dermatitis (AD)

Moderate-to-severe Atopic Dermatitis

China
Institute of Hematology & Blood Diseases Hospital

Not yet recruiting

The Efficacy and Safety of ZS802 in Chinese Hemophilia A Patients.

Hemophilia A

China
Hoffmann-La Roche

Completed

A Study to Investigate the Pharmacokinetics, Safety, and Tolerability of Emicizumab in Healthy Chinese Volunteers

Hemophilia A | Healthy Volunteers

China
Galderma R&D

Completed

Cetaphil Restoraderm Effect on Young Children With Atopic Dermatitis

Atopic Dermatitis

Philippines, China

Clinical Trials on Genetic Diseases, Inborn in China

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Singapore

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations