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这是一项关于庞贝病的II 期研究，登记的药物或干预包括S-606001；Placebo，当前状态为招募中。登记地点显示中国Irvine、Gainesville、Atlanta、St Louis等城市有研究中心记录。是否仍有名额以及是否值得进一步询问，需要向研究中心确认。
药物或干预
S-606001；Placebo · II 期
中国哪些地方有记录
以下地点来自登记信息，不代表此刻仍有名额；请向研究中心确认最新情况。
- University of California - Irvine Medical Center · Irvine
- University of Florida (UF) - Gainesville · Gainesville
- Emory University Hospital · Atlanta
- Washington University in St. Louis · St Louis
- Duke University Medical Center · Durham
- Cincinnati Children's Hospital Medical Center · Cincinnati
- University of Pennsylvania · Philadelphia
- University of Pittsburgh School of Medicine · Pittsburgh
- Lysosomal and Rare Disorders Research and Treatment Center (LDRTC) · Fairfax
- UZ Leuven · Leuven
- Aarhus University Hospital · Aarhus
- HLC Hopital Pierre Wertheimer · Bron
- AP-HP Hopital Raymond Poincare · Garches
- Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone · Marseille
- CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires · Nice
- Universitaetsklinikum Halle (Saale) · Halle
- SphinCS GmbH · Höchheim
- Klinikum der Ludwig-Maximilians-Universitaet Muenchen · München
- A.O.U. Policlinico "G. Martino" · Messina
- AOU Citta della Salute e della Scienza di Torino - Ospedale le Molinette · Torino
- Erasmus MC · GE Rotterdam
- Hospital Universitario 12 de Octubre · Madrid
- Hospital Universitari i Politecnic La Fe · Valencia
- Queen Elizabeth Hospital Birmingham · Birmingham
- National Hospital for Neurology & Neurosurgery · London
- Royal Free London NHS Foundation Trust · London
- Royal Victoria Infirmary · Newcastle upon Tyne
- Salford Royal Hospital · Salford
公开的入选条件
- * Participant must be ≥18 years of age and ≥40 kilograms (kg) of body weight at the time of signing the informed consent.
- * Participant must have a diagnosis of LOPD based on documentation of 1 of the following:
- 1. Deficiency of acid alpha-glucosidase (GAA) enzyme
- 2. GAA genotype
- * Participant has a %FVC ≥30% and ≤80% in an upright position without mechanical ventilation at screening; or Participant has ≥10% %FVC drop from upright position to supine position and %FVC ≥20% in a supine position.
- * Participant performs the 6MWT at screening, as determined by the clinical evaluator, and meets all of the following criteria:
- 1. Screening values of 6-minute walk distance (6MWD) are ≥75 meters
- 2. Screening values of 6MWD are ≤90% of the predicted value for healthy adults
联系前需要确认
请继续确认中心是否启动、疾病分型、既往治疗、检查费用和知情同意安排。
查看登记信息
登记号：NCT07123155；登记信息更新于 2026-06-23。