Overview

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

Status:
Completed
Trial end date:
2014-08-01
Target enrollment:
0
Participant gender:
All
Summary
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).
Phase:
Phase 3
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Pfizer
Protalix
Criteria
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.