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.

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Key information

Conditions

Gaucher Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Sphingolipidoses

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Taliglucerase alfa

Drug

Taliglucerase infusion every two weeks for 21 months

Primary outcomes

Spleen Volume

Time frame: 60 months

Spleen volume measured by MRI

Secondary outcomes

Liver Volume

Time frame: 60 months

Liver volume by MRI
Platelet Count

Time frame: 60 months

Platelet count measure annually
Hemoglobin

Time frame: 60 months

Hemoglobin measure yearly

Sponsors and collaborators

Lead sponsor

Pfizer

Industry

Registry information

Important dates

Study start: 2011
Primary completion: 2014
Study completion: 2014
First posted: Aug 23, 2011
Registry last updated: Apr 19, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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