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Pompe Disease Registry Protocol
NCT00231400 · View on ClinicalTrials.gov ↗
Study Summary
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.
Conditions Studied
Study Locations (20)
California
- University of California at Irvine- Site Number : 840036 — Irvine
- Loma Linda University- Genetics- Site Number : 840070 — Loma Linda
- Southern California Permanente Medical Group- Site Number : 840108 — Los Angeles
- USC Health Sciences Center Dept of Genetics- Site Number : 840082 — Los Angeles
- Children's Hospital Oakland- Site Number : 840029 — Oakland
- Children's Hospital of Orange County- Site Number : 840074 — Orange
- UC Davis MIND Institute- Site Number : 840010 — Sacramento
- University of California at San Diego- Site Number : 840007 — San Diego
- University of California at San Francisco- Site Number : 840051 — San Francisco
- Stanford University Pediatrics- Site Number : 840021 — Stanford
Arizona
- Barrow Neurological Institute- Site Number : 840087 — Phoenix
- Phoenix Children's Hospital- Site Number : 840003 — Phoenix
- University of Arizona- Site Number : 840015 — Tucson
Arkansas
- Arkansas Childrens Hospital- Site Number : 840109 — Little Rock
- University of Arkansas for Medical Sciences- Site Number : 840113 — Little Rock
Alabama
- University Alabama at Birmingham- Site Number : 840106 — Birmingham
Colorado
- Children's Hospital Colorado - Aurora- Site Number : 840069 — Aurora
Connecticut
- Yale - Site Number : 840047 — New Haven
District of Columbia
- Children's National Medical Center- Site Number : 840067 — Washington D.C.
Florida
- University of Florida College of Medicine- Site Number : 840012 — Gainesville
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 2,000 participants |
| Start Date | 2004-09-15 |
| Est. Completion | 2034-01-31 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00231400
The ClinicalTrials.gov registry entry for NCT00231400 describes a study currently listed as recruiting. It is categorized as an unspecified phase, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 2,000 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Genzyme, a Sanofi Company, which has 52 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 2 conditions, with Pompe Disease appearing as the primary indexed condition, and to 0 interventions. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT00231400 reports 20 study locations spanning 8 distinct geographic areas — top geographies include California, Arizona, Arkansas. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT00231400 about?
NCT00231400 is a clinical study titled "Pompe Disease Registry Protocol". 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 glob...
What is the current status of trial NCT00231400?
This trial is currently recruiting. The enrollment target is 2,000 participants. The study started on 2004-09-15. Estimated completion is 2034-01-31.
What conditions does trial NCT00231400 study?
This clinical trial studies the following conditions: Pompe Disease, Glycogen Storage Disease Type II. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
Who is sponsoring clinical trial NCT00231400?
This trial is sponsored by Genzyme, a Sanofi Company, which has 52 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT00231400 being conducted?
This trial has 20 study locations across Alabama, Arizona, Arkansas, California, Colorado. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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