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

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

19 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-08-31

Study Completion Date

2014-09-30

Brief Summary

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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.

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Detailed Description

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Conditions

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Gaucher Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Taliglucerase alfa 30 units/kg

Subjects randomized to receive 30 units/kg

Group Type EXPERIMENTAL

Taliglucerase alfa

Intervention Type DRUG

Taliglucerase infusion every two weeks for 21 months

Taliglucerase alfa 60 units/kg

Subjects randomized to 60 units/kg

Group Type EXPERIMENTAL

Taliglucerase alfa

Intervention Type DRUG

Taliglucerase infusion every two weeks for 21 months

Interventions

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Taliglucerase alfa

Taliglucerase infusion every two weeks for 21 months

Intervention Type DRUG

Eligibility Criteria

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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.

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No