Clinical Trials on Creatine Deficiency, X-linked in China

Total 70 results

Children's Hospital of Chongqing Medical University

Recruiting

Gene Therapy for X Linked Severe Combined Immunodeficiency

Gene Therapy

China
Shenzhen Geno-Immune Medical Institute

Unknown

Gene Transfer for SCID-X1 Using a Self-inactivating Lentiviral Vector （TYF-IL-2Rg）

SCID, X Linked

China
West China Hospital

Recruiting

Safety and Efficacy of a Single Subretinal Injection of JWK002 Gene Therapy in Subjects With X-linked Retinoschisis(XLRS)

X Linked Retinoschisis

China
Shanghai General Hospital, Shanghai Jiao Tong University...
Innostellar Biotherapeutics

Recruiting

Safety and Efficacy Study of LX103 Treatment of X-Linked Retinoschisis (XLRS)

X-linked Retinoschisis

China
Zhongmou Therapeutics

Recruiting

Safety and Efficacy Study of Novel Gene Therapy ZM-01 for X-linked Retinoschisis Patients

X-linked Retinoschisis

China
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
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
Children's Hospital of Fudan University

Recruiting

Metformin in Children With Fragile X Syndrome

Fragile X Syndrome | Metformin

China
Children's Hospital of Fudan University

Recruiting

Esketamine for the Treatment of Rett Syndrome

Rett Syndrome

China
Beijing Tiantan Hospital

Recruiting

Registry of X-linked Adrenoleukodystrophy

X-linked Adrenoleukodystrophy

China
Shenzhen Geno-Immune Medical Institute

Unknown

Lentiviral Gene Therapy for X-ALD

X-linked Adrenoleukodystrophy

China
Shanghai Children's Medical Center

Unknown

Chronic Granulomatous Disease Study in China

Granulomatous Disease, Chronic

China
InnoVec Biotherapeutics Inc.
Peking Union Medical College Hospital

Recruiting

Safety and Efficacy Study of IVB102 Injection in Subjects With X-linked Retinoschisis

Eye Diseases | Retinal Degeneration | Retinal Disease | Retinoschisis

China
Children's Hospital of Fudan University

Terminated

Pioglitazone Therapy for Chronic Granulomatous Disease

Chronic Granulomatous Disease

China
Shenzhen Geno-Immune Medical Institute

Unknown

Lentiviral Gene Therapy for CGD

Chronic Granulomatous Disease

China
Frontera Therapeutics
Peking Union Medical College Hospital; Eye & ENT Hospital of Fudan University; Henan Provincial People's Hospital

Recruiting

Gene Therapy for Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa

X-Linked Retinitis Pigmentosa

China
Children's Hospital of Fudan University

Recruiting

Vitamin D3 For CGD Patients With BCGosis/Itis

BCG | Vitamin D3 | Chronic-granulomatous Disease

China
Novartis Pharmaceuticals

Recruiting

Study to Evaluate the Efficacy, Safety and Tolerability of MAS825 in Patients With Monogenic IL-18 Driven Autoinflammatory Diseases, Including NLRC4-GOF, XIAP Deficiency, or CDC42 Mutations (MASter-1)

NLRC4-GOF, AIFEC (Autoinflammation With Infantile Enterocolitis), XIAP Deficiency, CDC42 Mutations

Spain, France, United States, Canada, Japan, United Kingdom, Turkey, Czechia, Italy
Peking Union Medical College Hospital

Unknown

Using Different Doses of Active Vitamin D Combined With Neutral Phosphate in Children With X-linked Hypophosphatemia

X-linked Hypophosphatemia

China
Kunming Hope of Health Hospital

Terminated

Recombinant Adeno-associated Virus Administration for Patients With Menkes Syndrome

Menkes Syndrome

China
Tianjin Anding Hospital

Not yet recruiting

Exosome for Early Diagnosis of Bipolar Affective Disorder

Major Depressive Disorder | Bipolar Affective Disorder

China
Institute of Hematology & Blood Diseases Hospital

Not yet recruiting

A Study to Evaluate the Safety and Efficacy of ZS801 in Adult Hemophilia B Patients

Hemophilia B

China
Jiangsu Gensciences lnc.

Completed

A Phase I of SS109 in Hemophilia A or and B With Inhibitors

Factor VII Deficiency | Hemophilia A With Inhibitor | Hemophilia B With Inhibitor

China
Institute of Hematology & Blood Diseases Hospital

Not yet recruiting

The Efficacy and Safety of ZS801 in Chinese Hemophilia B Patients.

Hemophilia B

China
Shanghai Vitalgen BioPharma Co., Ltd.

Not yet recruiting

A Study to Evaluate the Safety and Efficacy of VGB-R04 in Adult Hemophilia B Patients

Hemophilia B

China
Ning Wang, MD., PhD.

Recruiting

A Registered Cohort Study on Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

China
Institute of Hematology & Blood Diseases Hospital

Recruiting

Safety and Tolerability of VGB-R04 in Patients With Haemophilia B

Hemophilia B

China
Pfizer

Completed

An Open-Label, Single Dose Pharmacokinetic Study of Benefix (Recombinant Factor IX) in Male Chinese Subjects With Hemophilia B

Hemophilia B

China
Shenzhen Beike Bio-Technology Co., Ltd.
The Second Affiliated Hospital of Kunming Medical University

Unknown

Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

China
West China Hospital

Recruiting

A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of Single Intravenous Infusion of JWK007 in Patients With Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy

China
Institute of Hematology & Blood Diseases Hospital
East China University of Science and Technology

Active, not recruiting

Gene Therapy for Chinese Hemophilia B

Hemophilia B

China
General Hospital of Chinese Armed Police Forces

Unknown

The Safety, Efficacy and Tolerability of Remote Ischemic Preconditioning as a Therapy to DMD

Duchenne Muscular Dystrophy (DMD)

China
Pfizer

Completed

A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

China
Suzhou Alphamab Co., Ltd.

Recruiting

Efficacy and Safety of KN057 Prophylaxis in Patients With Haemophilia A or B With Inhibitors

Hemophilia A With Inhibitor | Hemophilia B With Inhibitor

China
Chia Tai Tianqing Pharmaceutical Group Co., Ltd.

Unknown

Study of Recombinant Human Coagulation Factor VIIa for Injection (FⅦa) in Patients With Hemophilia.

Hemophilia A With Inhibitor | Hemophilia B With Inhibitor

China
Shenzhen Second People's Hospital
Guangzhou Women and Children's Medical Center; Shenzhen University

Recruiting

Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy

Adrenoleukodystrophy | Metachromatic Leukodystrophy

China
Wyeth is now a wholly owned subsidiary of Pfizer

Completed

Study Evaluating On-Demand Treatment With BeneFIX In Chinese Subjects

Hemophilia B

China
Peking Union Medical College Hospital
National Natural Science Foundation of China; Cancer Institute and Hospital...

Recruiting

Bisoprolol in DMD Early Cardiomyopathy

Cardiomyopathy, Dilated | Duchenne Muscular Dystrophy

China
Chia Tai Tianqing Pharmaceutical Group Co., Ltd.

Recruiting

Clinical Study of Recombinant Human Activated Coagulation Factor VII for Injection in Patients With Hemophilia With Inhibitor

Hemophilia A | Hemophilia B

China
Novo Nordisk A/S

Enrolling by invitation

Post-marketing Surveillance (Use Result Surveillance) With Refixia®

Haemophilia B

Japan, Denmark
NS Pharma, Inc.
Nippon Shinyaku Co., Ltd.

Completed

Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53)

Duchenne Muscular Dystrophy

Spain, United States, Russian Federation, Italy, China, Turkey
Shanghai Belief-Delivery BioMed Co., Ltd

Active, not recruiting

Evaluation of the Safety and Efficacy of Hemophilia B Gene Therapy Drug

Hemophilia B

China
Jiangsu Gensciences lnc.

Recruiting

SS109 and NovoSeven ® PK / PD Profile, and Preliminary Efficacy and Safety of SS109 on Demand Treatment

Hemophilia | Hemophilia A With Inhibitor | Male | Hemophilia B With Inhibitor

China
Shanghai Children's Hospital

Recruiting

Study of Hydroxychloroquine in Patients With X-linked Alport Syndrome in China (CHXLAS) (CHXLAS)

Alport Syndrome, X-Linked

China
Pfizer

Completed

Safety and Efficacy of Benefix in Patients With Hemophilia B in Usual Care Settings in China

HEMOPHILIA B

China
Novo Nordisk A/S

Active, not recruiting

A Research Study Investigating Nonacog Beta Pegol (N9-GP) for Treatment and Prevention of Bleedings in Chinese People With Haemophilia B (Paradigm9)

Haemophilia B

China
Children's Hospital of Fudan University

Not yet recruiting

Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease

Fabry Disease

China
NS Pharma, Inc.
Nippon Shinyaku Co., Ltd.

Active, not recruiting

Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X)

Duchenne Muscular Dystrophy

Korea, Republic of, Chile, Japan, China, United Kingdom, Netherlands, Spain, Norway, Russian Federation, Australia, Greece, Italy, New Zealand, Turkey, Mexico, Czechia, Canada
NS Pharma, Inc.
Nippon Shinyaku Co., Ltd.

Completed

Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)

Duchenne Muscular Dystrophy

Korea, Republic of, Chile, Taiwan, China, United States, United Kingdom, Netherlands, Spain, Australia, Norway, Russian Federation, Greece, Italy, Turkey, Mexico, Canada, Hong Kong, New Zealand, Ukraine
Genzyme, a Sanofi Company

Completed

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors (ATLAS-INH)

Hemophilia A | Hemophilia B

United States, Australia, Canada, China, France, Korea, Republic of, Malaysia, Turkey, Ukraine, United Kingdom, Germany, India, Italy, Japan, South Africa, Spain, Taiwan

Clinical Trials on Creatine Deficiency, X-linked in China

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