Phase
Condition
Pompe Disease
Treatment
No Intervention
Clinical Study ID
Ages 16-69 All Genders
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
Participant has a documented clinical diagnosis of LOPD.
Participant is enzyme replacement therapy ERT-naïve (ERT-N) or if the participant iscurrently taking an approved ERT treatment or is participating in anERT-interventional study, the ERT must have been received for at least 6 months ormore (ERT-experienced [ERT-E]).
Participant is willing and able to comply with study visits and procedures.
Participant agrees to not start participating in any other clinical study involvingan investigational study treatment, including ERT, while participating in thisstudy.
Exclusion
Exclusion Criteria:
Participant previously received an AAV-related product (any serotype).
Participant is currently participating in a Pompe-related interventional study (other than ERT-interventional studies) or has received gene or cell therapy.
Participant requires any invasive or noninvasive ventilation support while awake andupright (non-invasive support while sleeping with either continuous positive airwaypressure (CPAP) or bilevel positive airway pressure (BiPAP) is acceptable foreligibility).
Participant is unable to ambulate (assistive devices [e.g., cane or walker] areacceptable for eligibility).
Participants who have received any ERT for less than 6 months as of the Baselinevisit are not eligible.
Study Design
Study Description
Connect with a study center
AU61003
Adelaide,
AustraliaActive - Recruiting
AU61001
Herston,
AustraliaActive - Recruiting
BR55002
Porto Alegre,
BrazilSite Not Available
CN15003
Edmonton,
CanadaActive - Recruiting
CA15001
Montreal,
CanadaActive - Recruiting
FR33006
Angers,
FranceActive - Recruiting
FR33009
Garches,
FranceActive - Recruiting
FR33005
Lille,
FranceActive - Recruiting
FR33007
Limoges,
FranceActive - Recruiting
FR33002
Marseille,
FranceActive - Recruiting
FR33003
Nantes,
FranceActive - Recruiting
FR33004
Nice Cedex 3,
FranceActive - Recruiting
FR33001
Strasbourg,
FranceActive - Recruiting
FR33007
Strasbourg,
FranceActive - Recruiting
DT49005
Bonn,
GermanyActive - Recruiting
DT49004
Essen,
GermanyActive - Recruiting
DT49003
Hochheim,
GermanyActive - Recruiting
DT49006
Münster,
GermanyActive - Recruiting
IT39002
Firenze,
ItalySite Not Available
IT39005
Gussago,
ItalyActive - Recruiting
IT39012
Messina,
ItalyActive - Recruiting
IT39009
Milano,
ItalyActive - Recruiting
IT39011
Milano,
ItalyActive - Recruiting
IT39008
Pavia,
ItalyActive - Recruiting
IT39006
Pisa,
ItalyActive - Recruiting
IT39004
Roma,
ItalyActive - Recruiting
National Center of Neurology and Psychiatry
Kodaira-Shi,
JapanActive - Recruiting
Tokyo Women's Medical University Hospital
Shinjuku-Ku,
JapanActive - Recruiting
ES34003
Albacete,
SpainActive - Recruiting
ES34004
Barcelona,
SpainSite Not Available
ES34006
Barcelona,
SpainSite Not Available
ES34007
L'hospitalet de Llobregat,
SpainActive - Recruiting
ES34001
Madrid,
SpainActive - Recruiting
ES34005
Madrid,
SpainActive - Recruiting
Hospital Universitario 12 de Octubre
Madrid,
SpainActive - Recruiting
ES34009
San Sebastian,
SpainSite Not Available
ES34002
Valencia,
SpainActive - Recruiting
Hospital Universitari i Politecnic La Fe de Valencia
Valencia,
SpainActive - Recruiting
National Taiwan University Hospital
Taipei, Province Of China
TaiwanActive - Recruiting
TW88601
Taipei,
TaiwanActive - Recruiting
TW88602
Taipei,
TaiwanActive - Recruiting
TW88603
Taoyuan City,
TaiwanSite Not Available
UK44003
Cambridge,
United KingdomActive - Recruiting
UK44001
Newcastle upon Tyne,
United KingdomActive - Recruiting
UK44004
Salford,
United KingdomActive - Recruiting
University of California Irvine
Irvine, California 92868
United StatesActive - Recruiting
Emory Clinic
Atlanta, Georgia 30322
United StatesActive - Recruiting
University of Kansas Medical Center
Kansas City, Kansas 66160
United StatesActive - Recruiting
University of Michigan
Ann Arbor, Michigan 48109
United StatesActive - Recruiting
Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota 55404
United StatesSite Not Available
Hackensack University Medical Center
Hackensack, New Jersey 07601
United StatesActive - Recruiting
University of Cincinnati
Cincinnati, Ohio 45221
United StatesActive - Recruiting
University of Pennsylvania
Philadelphia, Pennsylvania 19104
United StatesActive - Recruiting
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania 15213
United StatesActive - Recruiting
Lysosomal and Rare Diseases Research and Treatment Center, Inc.
Fairfax, Virginia 22030
United StatesActive - Recruiting
Not the study for you?
Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.