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已按「庞贝病」检索,对应国际通用词 Pompe disease

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共找到 19 项正在招募的试验(在欧美日韩等发达国家设有中心)

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正在招募I 期 / II 期NCT07282847

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)

United States

年龄 18 Years – 65 Years

AskBio Inc

正在招募不适用中国有中心NCT00231400

Pompe Disease Registry Protocol

United States、Argentina、Australia、Belgium、Brazil 等 12 国

年龄 不限 – 不限

Genzyme, a Sanofi Company

正在招募I 期NCT07354724

A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

United States

年龄 18 Years – 75 Years

Denali Therapeutics Inc.

正在招募不适用NCT06557369

A Clinical Trial Evaluating the Safety and Efficacy of a New Light Combination Therapy Addressing Intermediate AMD

Italy

年龄 50 Years – 不限

Oculox Technologies SA

正在招募不适用NCT01665326

Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

United States

年龄 不限 – 不限

Duke University

正在招募不适用NCT00567073

Pompe Pregnancy Sub-Registry

United States、Australia、Belgium、Brazil、Croatia 等 9 国

年龄 不限 – 不限

Genzyme, a Sanofi Company

正在招募不适用NCT04639336

Cognitive and Neurological Pathologies in Pompe Disease

United States

年龄 0 Years – 18 Years

Duke University

正在招募不适用NCT05989620

Long-Term Development of Muscular Dystrophy Outcome Assessments

United States

年龄 6 Years – 50 Years

Virginia Commonwealth University

正在招募不适用NCT07136844

Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology

Belgium

年龄 18 Years – 不限

Centre Hospitalier Universitaire de Liege

正在招募III 期中国有中心NCT04808505

A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

United States、Germany、Italy、Netherlands、Taiwan 等 6 国

年龄 不限 – 17 Years

Amicus Therapeutics

正在招募不适用NCT05734521

Avalglucosidase Alfa Pregnancy Study

United States

年龄 不限 – 不限

Sanofi

正在招募不适用NCT07664930

Phrenic Nerve and Diaphragm Electrophysiology in Pompe Disease

Italy

年龄 18 Years – 不限

IRCCS National Neurological Institute "C. Mondino" Foundation