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Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease

NCT00553631 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 34

Last updated 2021-06-08

Study results available
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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 non-inferiority study is to evaluate the efficacy and safety of GA-GCB (velaglucerase alfa) administered every other week in comparison to imiglucerase in treatment naive patients with type 1 Gaucher disease.

Conditions

  • Gaucher Disease, Type 1

Interventions

BIOLOGICAL

velaglucerase alfa

IV infusion, 60 U/kg every other week for 9 months

BIOLOGICAL

imiglucerase

IV infusion, 60 U/kg every other week for 9 months

Sponsors & Collaborators

  • Shire

    lead INDUSTRY

Principal Investigators

  • Study Director · Takeda

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2008-01-29
Primary Completion
2009-05-05
Completion
2009-05-05

Countries

  • United States
  • Argentina
  • India
  • Israel
  • Paraguay
  • Russia
  • Spain
  • Tunisia
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00553631 on ClinicalTrials.gov