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

Pfizer

Status and phase: Completed; Phase 3

Conditions: Gaucher Disease

Treatments: Drug: Taliglucerase alfa

Study type: Interventional

Funder types: Industry

Identifiers

NCT01422187

PB-06-007

Details and patient eligibility

About

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.

Enrollment: 19 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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19 participants in 2 patient groups

Taliglucerase alfa 30 units/kg

Experimental group

Description:

Subjects randomized to receive 30 units/kg

Treatment:

Drug: Taliglucerase alfa

Taliglucerase alfa 60 units/kg

Experimental group

Description:

Subjects randomized to 60 units/kg

Treatment:

Drug: Taliglucerase alfa