Condition: Glycogen Storage Disease Type II · Pompe Disease · Sponsor: Genzyme, a Sanofi Company
The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to track the disease's natural history and outcomes in patients, both treated and not. Data from the Registry are also used to fulfill various global regulatory commitments, to support product development/reimbursement, and for other research and non-research related purposes. The objectives of the Registry are: * To enhance understanding of the variability, progression, identification, and natural history of Pompe disease, with the ultimate goal of better guiding and assessing therapeutic intervention. * To assist the Pompe medical community with the development of recommendations for monitoring patients, and to provide reports on patient outcomes, to optimize patient care. * To characterize the Pompe disease population. * To evaluate the long-term effectiveness of alglucosidase alfa.
This description comes directly from the study's public registry record.
Trial Transparency email recommended (Toll free number for US & Canada) · 800-633-1610 · contact-us@sanofi.com
Pompe Registry HelpLine · 617-591-5500
Always discuss trial participation with your own doctor first.
| University of Alabama at Birmingham- Site Number : 840106 | Birmingham, Alabama, United States | Recruiting |
| Barrow Neurological Institute- Site Number : 840087 | Phoenix, Arizona, United States | Recruiting |
| Phoenix Children's Hospital- Site Number : 840003 | Phoenix, Arizona, United States | Recruiting |
| University of Arizona- Site Number : 840015 | Tucson, Arizona, United States | Recruiting |
| Arkansas Children's Hospital- Site Number : 840109 | Little Rock, Arkansas, United States | Recruiting |
| University of Arkansas for Medical Sciences- Site Number : 840113 | Little Rock, Arkansas, United States | Recruiting |
| University of California at Irvine- Site Number : 840036 | Irvine, California, United States | Recruiting |
| Loma Linda University- Genetics- Site Number : 840070 | Loma Linda, California, United States | Completed |
| Southern California Permanente Medical Group- Site Number : 840108 | Los Angeles, California, United States | Recruiting |
| USC Health Sciences Center Dept of Genetics- Site Number : 840082 | Los Angeles, California, United States | Completed |
| UCSF Benioff Children's Hospital - Oakland- Site Number : 840029 | Oakland, California, United States | Completed |
| Children's Hospital of Orange County- Site Number : 840074 | Orange, California, United States | Recruiting |
+ 260 more locations — full list on the registry record.
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This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.
Source record: clinicaltrials.gov/study/NCT00231400