A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease

Pfizer19 enrolled

Overview

This is a multi-center trial to further extend the assessment of the safety and efficacy of taliglucerase alfa in adult subjects (≥18 years old) with Gaucher disease who have enrolled in Protocol PB-06-003. Subjects will continue to receive an intravenous (IV) infusion of taliglucerase alfa every two weeks. The duration of treatment will be a maximum of 21 months or until taliglucerase alfa is commercially available to the subject at the discretion of the Sponsor.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Gaucher Disease

Interventions

Taliglucerase alfaDRUG

Taliglucerase infusion every two weeks for 21 months

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Successfully completed Protocol PB-06-001 and enrolled in Protocol PB-06-003 * The subject signs an informed consent Exclusion Criteria: * Currently taking another investigational drug for any condition. * Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the subject's compliance with the requirements of the study.

Outcomes

Primary Outcomes

Spleen Volume

Spleen volume measured by MRI

Time frame: 60 months

Secondary Outcomes

Liver Volume

Liver volume by MRI

Time frame: 60 months

Platelet Count

Platelet count measure annually

Time frame: 60 months

Hemoglobin

Hemoglobin measure yearly

Time frame: 60 months

Data from ClinicalTrials.gov

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