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A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease

NCT00430625 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 25

Last updated 2021-06-29

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 this 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 efficacy of every other week dosing of Gene-Activated® Human Glucocerebrosidase (GA-GCB, velaglucerase alfa) at doses of 45 and 60 U/kg in treatment-naïve patients with type 1 Gaucher disease.

Conditions

  • Gaucher Disease, Type 1

Interventions

BIOLOGICAL

VPRIV ®,

Intravenous (IV) infusion, every other week via intravenous infusion for 12 months

Sponsors & Collaborators

  • Shire

    lead INDUSTRY

Principal Investigators

  • Study Director · Takeda

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
PARALLEL

Eligibility

Min Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2007-02-15
Primary Completion
2009-04-01
Completion
2009-04-01

Countries

  • Argentina
  • Israel
  • Paraguay
  • Russia
  • Tunisia

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 NCT00430625 on ClinicalTrials.gov