Pompe disease study aims to clear path for gene therapy
NCT ID NCT06150820
First seen Nov 01, 2025 · Last updated May 14, 2026 · Updated 27 times
Summary
This study looks at people with late-onset Pompe disease to measure antibodies that might interfere with future gene therapy. Participants are either new to enzyme replacement therapy or have been on it for at least six months. No treatment is given; instead, blood and urine samples are collected over two years to track antibodies and disease markers.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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AU61001
Herston, Australia
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AU61003
Adelaide, Australia
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BR55001
São Paulo, Brazil
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BR55002
Porto Alegre, Brazil
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BR55003
Flamengo, Brazil
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CA15001
Montreal, Canada
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CN15003
Edmonton, Canada
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Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, 55404, United States
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DT49003
Höchheim, Germany
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DT49004
Essen, Germany
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DT49005
Bonn, Germany
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DT49006
Münster, Germany
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ES34001
Madrid, Spain
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ES34002
Valencia, Spain
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ES34003
Albacete, Spain
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ES34004
Barcelona, Spain
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ES34005
Madrid, Spain
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ES34007
L'Hospitalet de Llobregat, Spain
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ES34009
Donostia / San Sebastian, Spain
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Emory Clinic
Atlanta, Georgia, 30322, United States
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FR33001
Strasbourg, France
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FR33002
Marseille, France
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FR33003
Nantes, France
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FR33004
Nice, France
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FR33005
Lille, France
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FR33006
Angers, France
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FR33007
Limoges, France
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FR33009
Garches, France
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Hackensack University Medical Center
Hackensack, New Jersey, 07601, United States
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IT39002
Florence, Italy
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IT39003
Udine, Italy
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IT39004
Roma, Italy
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IT39005
Gussago, Italy
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IT39006
Pisa, Italy
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IT39008
Pavia, Italy
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IT39009
Milan, Italy
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IT39011
Milan, Italy
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IT39012
Messina, Italy
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Lysosomal and Rare Diseases Research and Treatment Center, Inc.
Fairfax, Virginia, 22030, United States
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National Center of Neurology and Psychiatry
Kodaira-Shi, Japan
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TW88601
Taipei, Taiwan
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TW88602
Taipei, Taiwan
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TW88603
Taoyuan City, Taiwan
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Tokyo Women's Medical University Hospital
Shinjuku-Ku, Japan
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UK44001
Newcastle upon Tyne, United Kingdom
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UK44003
Cambridge, United Kingdom
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UK44004
Salford, United Kingdom
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University of Cincinnati
Cincinnati, Ohio, 45221, United States
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University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
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University of Michigan
Ann Arbor, Michigan, 48109, United States
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University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
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University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15213, United States
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University of UTAH - PPDS
Salt Lake City, Utah, 84132, United States
Conditions
Explore the condition pages connected to this study.