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A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease

NCT00376168 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 32

Last updated 2018-10-04

Study results available
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Summary

Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD) leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the accumulation of substrate glucocerebroside (GlcCer) in the cells of the monocyte-macrophage system.

This is the second trial to utilize a recombinant active form of lysosomal enzyme, glucocerebrosidase, (human prGCD) which is expressed and purified in a bioreactor system from transformed carrot plant root cell line.

Conditions

  • Gaucher Disease

Interventions

DRUG

Plant cell expressed recombinant glucocerebrosidase (prGCD)

Intravenous infusion every two weeks for 9 months

DRUG

Plant cell expressed recombinant glucocerebrosidase (prGCD)

Intravenous infusion every 2 weeks for 9 months

Sponsors & Collaborators

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2007-08-31
Primary Completion
2009-09-30
Completion
2009-10-31

Countries

  • United States
  • Canada
  • Chile
  • Israel
  • Italy
  • South Africa
  • Spain
  • United Kingdom

Study Locations

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Entities

Companies

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 NCT00376168 on ClinicalTrials.gov