Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

Pfizer

Status and phase: Completed; Phase 3

Conditions: Gaucher Disease

Treatments: Drug: Taliglucerase alfa

Study type: Interventional

Funder types: Industry

Identifiers

NCT00705939

PB-06-003

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 45 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

---

45 participants in 3 patient groups

Naive 30 Units/kg

Experimental group

Description:

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

Treatment:

Drug: Taliglucerase alfa

Naive 60 Units/kg

Experimental group

Description:

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

Treatment:

Drug: Taliglucerase alfa

Switchover

Experimental group

Description:

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

Treatment:

Drug: Taliglucerase alfa