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NCT00486889 · ClinicalTrials.gov registry record · Phase 4
Growth and Development Study of Alglucosidase Alfa
A Phase 4 study of Pompe Disease and Glycogen Storage Disease Type II (GSD-II), sponsored by Genzyme, a Sanofi Company.
- Completed
- Registry status
- Phase 4
- Development phase
- 12
- Enrollment target
- 3
- Study locations
NCT00486889: Completed Phase 4 study of Pompe Disease and Glycogen Storage Disease Type II (GSD-II), sponsored by Genzyme, a Sanofi Company.
NCT00486889 is a Phase 4 study of Pompe Disease and Glycogen Storage Disease Type II (GSD-II) that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 12 participants, below the 232-participant average among 14 other Pompe Disease trials with a reported enrollment target (95% lower). The trial reports 3 study locations across 3 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00486889, a Phase 4 study of Pompe Disease and Glycogen Storage Disease Type II (GSD-II), has completed, sponsored by Genzyme, a Sanofi Company.
- COMPLETED
- Registry status
- Phase 4
- Development phase
- 12 participants
- Enrollment target
- 3
- Study locations
Study Summary
Pompe disease (also known as glycogen storage disease Type II) is a rare autosomal recessive metabolic muscle disease caused by the deficiency of acid α glucosidase (GAA), an enzyme that degrades lysosomal glycogen. As opposed to the exclusively cytoplasmic accumulation of glycogen that occurs in other glycogen storage disorders, Pompe disease is characterized by organelle bound (lysosomal) and extra-lysosomal accumulation of glycogen in many body tissues, ultimately leading to multisystemic pathology. The overall objective of this study was to evaluate the long-term growth and development of participants with infantile-onset Pompe disease with alglucosidase alfa before 1 year of age. Participants were to be followed for a 10-year period.
Primary Outcome
Height/Length of Participants was measured in centimeters. Week is denoted as Wk in time frame section.
Conditions Studied
Interventions
- BIOLOGICAL alglucosidase alfa
Study Locations (3)
Florida
- - Gainesville
Georgia
- - Decatur
Michigan
- - Detroit
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2008-08-26 |
| Est. Completion | 2021-11-23 |
| Phase | Phase 4 |
What the finished NCT00486889 record still lists
NCT00486889 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 12 participants, a relatively small participant target, below the 232-participant average among 14 other Pompe Disease trials with a reported enrollment target (95% lower).
The record links to 3 conditions, with Pompe Disease appearing as the primary indexed condition, and to 1 intervention - of which alglucosidase alfa is the first listed.
NCT00486889 reports a single indexed study location in Florida, Georgia, Michigan.
Frequently Asked Questions
What is clinical trial NCT00486889 about?
NCT00486889 is a clinical study titled "Growth and Development Study of Alglucosidase Alfa". Pompe disease (also known as glycogen storage disease Type II) is a rare autosomal recessive metabolic muscle disease caused by the deficiency of acid α glucosidase (GAA), an enzyme that degrades lysosomal glycogen. As opposed to the exclusively cytoplasmic accumulation of glycogen that occurs in ot...
What is the current status of trial NCT00486889?
This trial is currently completed. It is a Phase 4 study. The enrollment target is 12 participants. The study started on 2008-08-26. Estimated completion is 2021-11-23.
What conditions does trial NCT00486889 study?
This clinical trial studies the following conditions: Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease.
What interventions are being tested in trial NCT00486889?
The interventions under investigation include: alglucosidase alfa (BIOLOGICAL).
Who is sponsoring clinical trial NCT00486889?
This trial is sponsored by Genzyme, a Sanofi Company, which has 196 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT00486889 being conducted?
This trial has 3 study locations across Florida, Georgia, Michigan. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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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.
Where NCT00486889's enrollment target sits among peer trials
12 11th of 14 higher than 4 of 14 other Pompe Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Pompe Disease trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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