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NCT00455195 · ClinicalTrials.gov registry record · Phase 4

Late-Onset Treatment Study Extension Protocol

A Phase 4 study of Pompe Disease (Late-onset) and Glycogen Storage Disease Type II (GSD-II), sponsored by Genzyme, a Sanofi Company.

Completed
Registry status
Phase 4
Development phase
81
Enrollment target
20
Study locations

NCT00455195 is a Phase 4 study of Pompe Disease (Late-onset) and Glycogen Storage Disease Type II (GSD-II) that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 81 participants, above the 37-participant average among 8 other Pompe Disease (Late-onset) trials with a reported enrollment target (119% higher). The trial reports 20 study locations across 16 states.

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The verdict

NCT00455195, a Phase 4 study of Pompe Disease (Late-onset) and Glycogen Storage Disease Type II (GSD-II), has completed, sponsored by Genzyme, a Sanofi Company.

COMPLETED
Registry status
Phase 4
Development phase
81 participants
Enrollment target
20
Study locations

Study Summary

Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. The objective of this extension study is to assess the long-term safety and efficacy of alglucosidase alfa treatment in patients with Late-Onset Pompe Disease who were previously treated under the placebo-controlled, double-blind study AGLU02704 (NCT00158600).

Conditions Studied

Pompe Disease (Late-onset) Glycogen Storage Disease Type II (GSD-II) Glycogenesis Type II Acid Maltase Deficiency (AMD)

Interventions

  • BIOLOGICAL alglucosidase alfa

Study Locations (20)

Florida

  • - Coral Springs
  • - Gainesville
  • - Sarasota

California

  • - Beverly Hills
  • - San Diego

Ohio

  • - Cincinnati
  • - Toledo

Alaska

  • - Anchorage

Arizona

  • - Phoenix

District of Columbia

  • - Washington D.C.

Illinois

  • - Chicago

Kansas

  • - Kansas City

Trial Details

FieldValue
Enrollment Target 81 participants
Start Date 2007-03
Est. Completion 2008-11
Phase Phase 4

Sponsor

Genzyme, a Sanofi Company

196 total trials

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What the Registry Record Tells You About NCT00455195

The ClinicalTrials.gov registry entry for NCT00455195 describes a study currently listed as completed, categorized as Phase 4. The registered enrollment target is 81 participants, a figure that helps gauge the scale of data the investigators plan to collect, above the 37-participant average among 8 other Pompe Disease (Late-onset) trials with a reported enrollment target (119% higher). The listed sponsor is Genzyme, a Sanofi Company, which has 196 total studies on file at ClinicalTrials.gov.

The record links to 4 conditions, with Pompe Disease (Late-onset) appearing as the primary indexed condition, and to 1 intervention - of which alglucosidase alfa is the first listed.

NCT00455195 reports 20 study locations spanning 16 distinct geographic areas - top geographies include Florida, California, Ohio.

Frequently Asked Questions

What is clinical trial NCT00455195 about?

NCT00455195 is a clinical study titled "Late-Onset Treatment Study Extension Protocol". Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In ...

What is the current status of trial NCT00455195?

This trial is currently completed. It is a Phase 4 study. The enrollment target is 81 participants. The study started on 2007-03. Estimated completion is 2008-11.

What conditions does trial NCT00455195 study?

This clinical trial studies the following conditions: Pompe Disease (Late-onset), Glycogen Storage Disease Type II (GSD-II), Glycogenesis Type II, Acid Maltase Deficiency (AMD).

What interventions are being tested in trial NCT00455195?

The interventions under investigation include: alglucosidase alfa (BIOLOGICAL).

Who is sponsoring clinical trial NCT00455195?

This trial is sponsored by Genzyme, a Sanofi Company, which has 196 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT00455195 being conducted?

This trial has 20 study locations across Alaska, Arizona, California, District of Columbia, Florida. 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.