Scientists map immune barriers for future pompe disease gene therapy

NCT ID NCT06150820

Ongoing Knowledge-focused Sponsor: Astellas Gene Therapies Source: ClinicalTrials.gov ↗

Summary

This study aims to gather information that could help develop future gene therapies for late-onset Pompe disease, a genetic condition that causes progressive muscle weakness. Researchers are measuring antibody levels against a common virus used in gene therapy (AAV8) and against the current enzyme replacement treatment in 119 participants. The study involves collecting blood and urine samples over two years to understand how these factors might affect potential future treatments, but no experimental treatments are being tested.

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Contacts and locations

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Locations

AU61001

Herston, Australia
AU61003

Adelaide, Australia
BR55001

São Paulo, Brazil
BR55002

Porto Alegre, Brazil
BR55003

Flamengo, Brazil
CA15001

Montreal, Canada
CN15003

Edmonton, Canada
Children's Hospitals and Clinics of Minnesota

Minneapolis, Minnesota, 55404, United States
DT49003

Höchheim, Germany
DT49004

Essen, Germany
DT49005

Bonn, Germany
DT49006

Münster, Germany
ES34001

Madrid, Spain
ES34002

Valencia, Spain
ES34003

Albacete, Spain
ES34004

Barcelona, Spain
ES34005

Madrid, Spain
ES34007

L'Hospitalet de Llobregat, Spain
ES34009

Donostia / San Sebastian, Spain
Emory Clinic

Atlanta, Georgia, 30322, United States
FR33001

Strasbourg, France
FR33002

Marseille, France
FR33003

Nantes, France
FR33004

Nice, France
FR33005

Lille, France
FR33006

Angers, France
FR33007

Limoges, France
FR33009

Garches, France
Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States
IT39002

Florence, Italy
IT39003

Udine, Italy
IT39004

Roma, Italy
IT39005

Gussago, Italy
IT39006

Pisa, Italy
IT39008

Pavia, Italy
IT39009

Milan, Italy
IT39011

Milan, Italy
IT39012

Messina, Italy
Lysosomal and Rare Diseases Research and Treatment Center, Inc.

Fairfax, Virginia, 22030, United States
National Center of Neurology and Psychiatry

Kodaira-Shi, Japan
TW88601

Taipei, Taiwan
TW88602

Taipei, Taiwan
TW88603

Taoyuan, Taiwan
Tokyo Women's Medical University Hospital

Shinjuku-Ku, Japan
UK44001

Newcastle upon Tyne, United Kingdom
UK44003

Cambridge, United Kingdom
UK44004

Salford, United Kingdom
University of Cincinnati

Cincinnati, Ohio, 45221, United States
University of Kansas Medical Center

Kansas City, Kansas, 66160, United States
University of Michigan

Ann Arbor, Michigan, 48109, United States
University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States
University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15213, United States
University of UTAH - PPDS

Salt Lake City, Utah, 84132, United States

Conditions

Explore the condition pages connected to this study.

POMPE DISEASE (LATE-ONSET)