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NCT00478647 · ClinicalTrials.gov registry record · Phase 2

Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase

A Phase 2 study of Gaucher Disease, sponsored by Shire.

Completed
Registry status
Phase 2
Development phase
40
Enrollment target
15
Study locations

NCT00478647 is a Phase 2 study of Gaucher Disease that has completed, run by Shire. The registered enrollment target is 40 participants, below the 1,056-participant average among 13 other Gaucher Disease trials with a reported enrollment target (96% lower). The trial reports 15 study locations across 11 states.

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The verdict

NCT00478647, a Phase 2 study of Gaucher Disease, has completed, sponsored by Shire.

COMPLETED
Registry status
Phase 2
Development phase
40 participants
Enrollment target
15
Study locations

Study Summary

Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular engorgement, organomegaly, and organ system dysfunction. The purpose of this study is to evaluate the safety and efficacy of every other week dosing of GA-GCB (velaglucerase alfa) in participants with type 1 Gaucher disease who were previously treated with imiglucerase.

Conditions Studied

Gaucher Disease

Interventions

  • BIOLOGICAL GA-GCB (velaglucerase alfa)

Study Locations (15)

Other

  • Shaare Zedek Medical Center - Jerusalem
  • Children's Memorial Health Institute - Warsaw
  • Hospital Universitario Miguel Servet - Zaragoza
  • The Royal Free Hospital - London

California

  • Regional Metabolic Center - Los Angeles
  • Children's Hospital Oakland - Oakland

Georgia

  • Emory University - Decatur

Illinois

  • Feinberg School of Medicine - Chicago

Minnesota

  • Children's of Minnesota - Minneapolis

Missouri

  • Children's Mercy Hospital and Clinic - Kansas City

New York

  • NYU School of Medicine - New York

Ohio

  • Cincinatti Children's Hospital - Cincinnati

Trial Details

FieldValue
Enrollment Target 40 participants
Start Date 2007-07-25
Est. Completion 2009-06-26
Phase Phase 2

Sponsor

Shire

245 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT00478647

The ClinicalTrials.gov registry entry for NCT00478647 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 40 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 1,056-participant average among 13 other Gaucher Disease trials with a reported enrollment target (96% lower). The listed sponsor is Shire, which has 245 total studies on file at ClinicalTrials.gov.

The record links to 1 condition, with Gaucher Disease appearing as the primary indexed condition, and to 1 intervention - of which GA-GCB (velaglucerase alfa) is the first listed.

NCT00478647 reports 15 study locations spanning 11 distinct geographic areas - top geographies include Other, California, Georgia.

Frequently Asked Questions

What is clinical trial NCT00478647 about?

NCT00478647 is a clinical study titled "Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase". Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular engorgement, organomegaly, and organ system dysfunction. The purpose of t...

What is the current status of trial NCT00478647?

This trial is currently completed. It is a Phase 2 study. The enrollment target is 40 participants. The study started on 2007-07-25. Estimated completion is 2009-06-26.

What conditions does trial NCT00478647 study?

This clinical trial studies the following conditions: Gaucher Disease.

What interventions are being tested in trial NCT00478647?

The interventions under investigation include: GA-GCB (velaglucerase alfa) (BIOLOGICAL).

Who is sponsoring clinical trial NCT00478647?

This trial is sponsored by Shire, which has 245 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT00478647 being conducted?

This trial has 15 study locations across California, Georgia, Illinois, Minnesota, Missouri. 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.