NCT00231400

Pompe Disease Registry Protocol

RecruitingOBSERVATIONAL

Trial Details

Start: 2004-09-15Est. Completion: 2034-01-31

Enrollment target: 2,000 participants

Locations

University of Alabama at Birmingham- Site Number : 840106, Birmingham, Alabama, United States
Barrow Neurological Institute- Site Number : 840087, Phoenix, Arizona, United States
Phoenix Children's Hospital- Site Number : 840003, Phoenix, Arizona, United States
University of Arizona- Site Number : 840015, Tucson, Arizona, United States
Arkansas Children's Hospital- Site Number : 840109, Little Rock, Arkansas, United States
University of Arkansas for Medical Sciences- Site Number : 840113, Little Rock, Arkansas, United States
University of California at Irvine- Site Number : 840036, Irvine, California, United States
Loma Linda University- Genetics- Site Number : 840070, Loma Linda, California, United States
Southern California Permanente Medical Group- Site Number : 840108, Los Angeles, California, United States
USC Health Sciences Center Dept of Genetics- Site Number : 840082, Los Angeles, California, United States
UCSF Benioff Children's Hospital - Oakland- Site Number : 840029, Oakland, California, United States
Children's Hospital of Orange County- Site Number : 840074, Orange, California, United States

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The aim of this study is to assess the efficacy and safety of endovascular treatment versus medical management in patien…

Beijing, ChinaCompleted Feb 25, 2026

Pompe Disease (Late-onset)Pompe Disease Late-OnsetLOPD

This is a single-arm, open-label, dose-escalation study to evaluate the safety, tolerability and efficacy of a single in…

Phoenix, United StatesEst. completion Sep 2028

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