Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT00231400 · ClinicalTrials.gov registry record
Pompe Disease Registry Protocol
A clinical trial of Pompe Disease and Glycogen Storage Disease Type II, sponsored by Genzyme, a Sanofi Company.
- Recruiting
- Registry status
- 2,000
- Enrollment target
- 20
- Study locations
NCT00231400 is a study of Pompe Disease and Glycogen Storage Disease Type II that is actively recruiting participants, run by Genzyme, a Sanofi Company. The registered enrollment target is 2,000 participants, above the 90-participant average among 14 other Pompe Disease trials with a reported enrollment target (2122% higher). The trial reports 20 study locations across 8 states.
The verdict
NCT00231400, a study of Pompe Disease and Glycogen Storage Disease Type II, is actively recruiting participants, sponsored by Genzyme, a Sanofi Company.
- RECRUITING
- Registry status
- 2,000 participants
- Enrollment target
- 20
- Study locations
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 |
Interested in This Trial?
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, categorized as an unspecified phase. The registered enrollment target is 2,000 participants, a figure that helps gauge the scale of data the investigators plan to collect, above the 90-participant average among 14 other Pompe Disease trials with a reported enrollment target (2122% higher). The listed sponsor is Genzyme, a Sanofi Company, which has 196 total studies on file at ClinicalTrials.gov.
The record links to 2 conditions, with Pompe Disease appearing as the primary indexed condition, and to 0 interventions.
NCT00231400 reports 20 study locations spanning 8 distinct geographic areas - top geographies include California, Arizona, Arkansas.
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.
Who is sponsoring clinical trial NCT00231400?
This trial is sponsored by Genzyme, a Sanofi Company, which has 196 total clinical trials registered on ClinicalTrials.gov.
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.
Learn More About Clinical Trials
Similar trials for Pompe Disease
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
-
Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
RECRUITING · Phase 2
-
Cognitive and Neurological Pathologies in Pompe Disease
RECRUITING
-
Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
RECRUITING
-
A Global Prospective Observational Registry of Patients With Pompe Disease
RECRUITING
-
Avalglucosidase Alfa Pregnancy Study
RECRUITING
-
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
ACTIVE NOT RECRUITING · Phase 1
Explore more on PlainTrial
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.