庞贝病临床试验招募信息（全球在招）
这里汇总了全球正在招募患者的 庞贝病（国际通用名 Pompe disease）新药临床试验，包含试验分期、招募状态、开展国家和研究团队联系方式，全部以中文呈现。数据实时来自全球最大的临床试验注册库 ClinicalTrials.gov。本页同时覆盖糖原贮积症 II 型等说法。
正在招募的庞贝病新药试验
Registry of Patients Diagnosed With Lysosomal Storage Diseases
不适用招募中美国NCT05619900Pompe Pregnancy Sub-Registry
不适用招募中美国澳大利亚BelgiumBrazilNCT00567073A Clinical Trial Evaluating the Safety and Efficacy of a New Light Combination Therapy Addressing Intermediate AMD
不适用招募中意大利NCT06557369Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology
不适用招募中BelgiumNCT07136844Phrenic Nerve and Diaphragm Electrophysiology in Pompe Disease
不适用招募中意大利NCT07664930Avalglucosidase Alfa Pregnancy Study
不适用招募中美国NCT05734521Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
不适用招募中美国NCT07478172A Global Prospective Observational Registry of Patients With Pompe Disease
不适用招募中美国AustriaBelgiumDenmarkNCT06121011A Long-term Study for the Outcome of Pompe Disease
不适用招募中中国台湾NCT02399748A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
III 期招募中美国德国意大利NetherlandsNCT04808505Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
不适用招募中美国NCT01665326Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases
不适用招募中法国NCT06833489
庞贝病试验不在中国大陆，还能参加吗？
可以尝试四条现实路径：查找国内同类试验、申请同情用药与扩展准入、赴境外研究中心入组、直接写信给申办方申请在华开展。本站提供中英文对照的咨询信模板，患者和家属填写基本情况即可生成。