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Pompe Disease Registry Protocol

Recruitingn/aAges any - anyAllGenzyme, a Sanofi Company
NCT00231400 on ClinicalTrials.gov ↗
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In plain words

What the trial is, and whether it could suit you

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

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📋 Eligibility rules (as published)
Inclusion Criteria: All patients with a confirmed diagnosis of Pompe disease who have signed the informed consent and authorization form(s) are eligible for inclusion. Confirmed diagnosis is defined as documented GAA enzyme deficiency from blood, skin, or muscle tissue and/or documentation of 2 GAA gene mutations. Exclusion Criteria: There are no exclusion criteria in this Registry
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What it tests

  • Not listed
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Where

272 sites
University of Alabama at Birmingham- Site Number : 840106
Birmingham, United States
Barrow Neurological Institute- Site Number : 840087
Phoenix, United States
Phoenix Children's Hospital- Site Number : 840003
Phoenix, United States
University of Arizona- Site Number : 840015
Tucson, United States
Arkansas Children's Hospital- Site Number : 840109
Little Rock, United States
University of Arkansas for Medical Sciences- Site Number : 840113
Little Rock, United States
University of California at Irvine- Site Number : 840036
Irvine, United States
Loma Linda University- Genetics- Site Number : 840070
Loma Linda, United States

…and 264 more on ClinicalTrials.gov.

🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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