A Multicenter Extension Study of Taliglucerase Alfa in Pediatric 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

NCT01411228

PB-06-006

Details and patient eligibility

About

A protocol to extend the assessment of the safety and efficacy of taliglucerase alfa in pediatric subjects (2 to <18 years old) with symptoms and clinical manifestations of Gaucher disease who completed treatment in Protocols PB-06-002 (switchover study from imiglucerase) or PB-06-005 (naïve treatment with taliglucerase alfa).

Enrollment

15 patients

Sex

All

Ages

2 to 18 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Successful completion of Protocol PB-06-002 or PB-06-005
The subject, parent(s) or legal guardian(s) signs an informed consent and/or assent

Exclusion criteria

Currently taking another investigational drug for any condition.
Presence of neurological signs and symptoms characteristic of Gaucher disease with complex neuronopathic features other than longstanding oculomotor gaze palsy.
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.