Clinical Trials on Genetic Disease in China

Total 31744 results

Bioray Laboratories
First Affiliated Hospital of Guangxi Medical University

Suspended

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

Thalassemia Major

China
Hangzhou Zede Pharma-Tech Co., Ltd.

Completed

Short-term Clinical Study of CN128 in Thalassemia Patients

Thalassemia

China
Hangzhou Zede Pharma-Tech Co., Ltd.

Completed

Clinical Study of CN128 in Thalassemia Patients

Thalassemia

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
The First Affiliated Hospital, Guangzhou University...
National Natural Science Foundation of China

Unknown

Study on the Mechanism of Colla Corri Asini in the Treatment of Thalassemia Patients With Pregnancy Anemia

Pregnancy | Thalassemia

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
Innovent Biologics (Suzhou) Co. Ltd.

Recruiting

A Multicenter, Open-label Extension Study Evaluating the Efficacy and Safety of IBI311 in Subjects With Thyroid Eye Disease

Thyroid Eye Disease, TED

China
Wuhan Union Hospital, China

Unknown

A Clinical Study of Immunoadsorption Therapy for Dilated Cardiomyopathy

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
Beijing Tiantan Hospital

Recruiting

Registry of X-linked Adrenoleukodystrophy

X-linked Adrenoleukodystrophy

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
Xinhua Hospital, Shanghai Jiao Tong University...

Unknown

Whole Genome Sequencing in the Detection of Rare Undiagnosed Genetic Diseases in Children in China

Intellectual Disability | Rare Diseases | Multiple Congenital Anomaly

China
China Cardiovascular Association
Amgen

Unknown

a Prospective Pilot Study of Screening Out Rate and Clinical Management of Familial Hypercholesterolemia

Familial Hypercholesterolemia

China
Peking University People's Hospital

Active, not recruiting

A Study of IBI311 in Subjects With Steroid-resistant, Thyroid Associated Ophthalmopathy

Thyroid Associated Ophthalmopathy

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
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 Xi'an Jiaotong University
Nanjing First Hospital, Nanjing Medical University; Beijing Tongren Hospital; People's Hospital of Xinjiang Uygur Autonomous Region and other collaborators

Not yet recruiting

Rapamycin Plus Methylprednisolone Versus Methylprednisolone Alone in Active, Moderate-to-severe Graves' Orbitopathy.

Graves Ophthalmopathy

China
Peking Union Medical College Hospital

Recruiting

68Ga-NY104 PET/CT in Von Hippel-Lindau Disease

Von Hippel-Lindau Disease

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
Jiangsu HengRui Medicine Co., Ltd.

Active, not recruiting

A Multicentre, Randomized, Double-blind, Allopurinol Controlled Study to Evaluate the Efficacy and Safety of SHR4640 in Subjects With Gout

Gout

China
Genzyme, a Sanofi Company

Active, not recruiting

Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen (APOLLO-LOPD)

Pompe's Disease

China
Jiangsu HengRui Medicine Co., Ltd.

Completed

A Study of Evaluating the Efficacy and Safety of SHR4640 in Subjects With Gout

Gout

China
Shanghai 10th People's Hospital

Unknown

Treatment of Refractory Gout With ACTH or Methylprednisolone

Gout

China
Sun Yat-sen University

Completed

Efficacy of Subantimicrobial Dose Doxycycline for Moderate to Severe and Active Graves' Orbitopathy

Immune System Diseases | Autoimmune Diseases | Eye Diseases | Endocrine System Diseases | Thyroid Diseases | Eye Diseases, Hereditary | Graves Ophthalmopathy | Graves Disease | Hyperthyroidism

China
Lei Li

Unknown

MSI in Circulatory DNA of Endometrial Cancer

Lynch Syndrome | Endometrial Cancer | Microsatellite Instability | Circulating Tumor Cell

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
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
Qilu Hospital of Shandong University

Recruiting

Screening Patients With Fabry Disease in Patients With Hypertrophic Cardiomyopathy or Left Ventricular Hypertrophy (SEARCH)

Fabry Disease | Hypertrophic Cardiomyopathy | Left Ventricular Hypertrophy

China
Sun Yat-sen University

Completed

Minocycline Treatment in Retinitis Pigmentosa

Retinitis Pigmentosa | Inherited Retinal Dystrophy | Retina Disorder

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
Peking Union Medical College Hospital

Unknown

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

Primary Hypertrophic Osteoarthropathy

China
Peking Union Medical College Hospital

Recruiting

A National Registry on Chinese Patients With Cystic Fibrosis

Pulmonary Function

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
Kunming Hope of Health Hospital

Terminated

Recombinant Adeno-associated Virus Administration for Patients With Menkes Syndrome

Menkes Syndrome

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
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
Shanghai Belief-Delivery BioMed Co., Ltd
Shanghai MYGT Biopharmaceutical LLC

Recruiting

Evaluation of the Safety and Efficacy of Hemophilia A Gene Therapy Drugs

Hemophilia A

China
Beijing Children's Hospital

Unknown

Pharmacokinetic (PK)Research on Chinese Children of Hemophilia (PK)

Pharmacokinetics

China
Beijing Children's Hospital

Unknown

A Clinical Phenotype Based Individualized Prophylaxis in Chinese Hemophilia A Children (CHIPS)

Haemophilia

China
The First Affiliated Hospital of Zhengzhou University

Recruiting

Survival Significance of Molecular Pathology and Genetic Variation in Brain Gliomas

Glioma | Molecular Sequence Variation | Genetic Change

China

Clinical Trials on Genetic Disease in China

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Gambia

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Rare Diseases

Drug Interventions

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