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

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

15 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-09-30

Study Completion Date

2014-08-31

Brief Summary

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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).

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Detailed Description

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Conditions

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Gaucher Disease

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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60 Units/kg

Group Type EXPERIMENTAL

Taliglucerase alfa

Intervention Type DRUG

Taliglucerase alfa for infusion every two weeks for 24 months

30 Units/kg

Group Type EXPERIMENTAL

Taliglucerase alfa

Intervention Type DRUG

Taliglucerase alfa for infusion every two weeks for 24 months

Interventions

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Taliglucerase alfa

Taliglucerase alfa for infusion every two weeks for 24 months

Intervention Type DRUG

Other Intervention Names

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prGCD, plant cell expressed glucocerebrosidase

Eligibility Criteria

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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.

Minimum Eligible Age

2 Years

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No