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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 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.

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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.

Conditions Studied

Pompe Disease Glycogen Storage Disease Type II (GSD-II) Acid Maltase Deficiency Disease

Interventions

  • BIOLOGICAL alglucosidase alfa

Study Locations (3)

Florida

  • - Gainesville

Georgia

  • - Decatur

Michigan

  • - Detroit

Trial Details

FieldValue
Enrollment Target 12 participants
Start Date 2008-08-26
Est. Completion 2021-11-23
Phase Phase 4

Sponsor

Genzyme, a Sanofi Company

196 total trials

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What the Registry Record Tells You About NCT00486889

The ClinicalTrials.gov registry entry for NCT00486889 describes a study currently listed as completed, categorized as Phase 4. The registered enrollment target is 12 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 232-participant average among 14 other Pompe Disease trials with a reported enrollment target (95% lower). The listed sponsor is Genzyme, a Sanofi Company, which has 196 total studies on file at ClinicalTrials.gov.

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 3 study locations spanning 3 distinct geographic areas - top geographies include 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.

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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.