Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

45 participants

Study Classification

INTERVENTIONAL

Study Start Date

2008-06-30

Study Completion Date

2013-08-31

Brief Summary

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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 an extension trial to Study NCT00376168 and NCT00712348.

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Detailed Description

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This will be a multi-center, double-blind, parallel group, extension trial to assess the safety and efficacy of prGCD in patients completing NCT00376168. Patients will receive IV infusion of prGCD every two weeks at the selected medical center. The duration of the extension study will be fifteen months. There will be two treatment groups: 30 units/kg every 2 weeks or 60 units/kg every 2 weeks.

Conditions

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Gaucher Disease

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Naive 30 Units/kg

Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168)

Group Type EXPERIMENTAL

Taliglucerase alfa

Intervention Type DRUG

Intravenous infusion every 2 weeks

Naive 60 Units/kg

Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168)

Group Type EXPERIMENTAL

Taliglucerase alfa

Intervention Type DRUG

Intravenous infusion every 2 weeks

Switchover

Continue taliglucerase alfa treatment from PB-06-002 (NCT00712348)

Group Type EXPERIMENTAL

Taliglucerase alfa

Intervention Type DRUG

Intravenous infusion every 2 weeks

Interventions

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Taliglucerase alfa

Intravenous infusion every 2 weeks

Intervention Type DRUG

Other Intervention Names

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Plant Cell Expressed Recombinant Human Glucocerebrosidase prGCD

Eligibility Criteria

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Inclusion Criteria

* Successful completion of Protocol PB-06-001
* The patient signs informed consent

Exclusion Criteria

* Currently taking another experimental drug for any condition
* Presence of severe neurological signs and symptoms, defined as complete ocular paralysis, overt myoclonus or history of seizures, characteristic of neuronopathic Gaucher disease
* Pregnant or nursing
* Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the patient's compliance with the requirements of the study

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No