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

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 Overview

• Status: Completed
• Conditions: Gaucher Disease
• Intervention / Treatment: Drug: Taliglucerase alfa

• Study Type: Interventional
• Enrollment (Actual): 19
• Phase: Phase 3

Expanded Access

No longer available outside the clinical trial. See expanded access record.

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Taliglucerase alfa 30 units/kg; Subjects randomized to receive 30 units/kg	Drug: Taliglucerase alfa; Taliglucerase infusion every two weeks for 21 months
Experimental: Taliglucerase alfa 60 units/kg; Subjects randomized to 60 units/kg	Drug: Taliglucerase alfa; Taliglucerase infusion every two weeks for 21 months

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Spleen Volume	Spleen volume measured by MRI	60 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Liver Volume	Liver volume by MRI	60 months
Platelet Count	Platelet count measure annually	60 months
Hemoglobin	Hemoglobin measure yearly	60 months

Collaborators and Investigators

• Sponsor: Pfizer

Publications and helpful links

General Publications

• Zimran A, Duran G, Giraldo P, Rosenbaum H, Giona F, Petakov M, Terreros Munoz E, Solorio-Meza SE, Cooper PA, Varughese S, Alon S, Chertkoff R. Long-term efficacy and safety results of taliglucerase alfa through 5years in adult treatment-naive patients with Gaucher disease. Blood Cells Mol Dis. 2019 Sep;78:14-21. doi: 10.1016/j.bcmd.2016.07.002. Epub 2016 Jul 18.

Study record dates

Study Major Dates

• Study Start: August 1, 2011
• Primary Completion (Actual): May 1, 2014
• Study Completion (Actual): September 1, 2014

Study Registration Dates

• First Submitted: August 22, 2011
• First Submitted That Met QC Criteria: August 22, 2011
• First Posted (Estimate): August 23, 2011

Study Record Updates

• Last Update Posted (Actual): April 19, 2023
• Last Update Submitted That Met QC Criteria: April 18, 2023
• Last Verified: April 1, 2023

More Information

Terms related to this study

• Keywords: enzyme replacement therapy
• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Lipid Metabolism Disorders; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Lipidoses; Lipid Metabolism, Inborn Errors; Gaucher Disease
• Other Study ID Numbers: PB-06-007