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

Study Overview
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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).

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

Conditions
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Gaucher Disease

Intervention / Treatment
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* Drug: Taliglucerase alfa


Participation Criteria
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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.

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 60 Units/kg<br> | Drug: Taliglucerase alfa<br>* Taliglucerase alfa for infusion every two weeks for 24 months<br>* Other names: prGCD, plant cell expressed glucocerebrosidase;|
| Experimental: 30 Units/kg<br> | Drug: Taliglucerase alfa<br>* Taliglucerase alfa for infusion every two weeks for 24 months<br>* Other names: prGCD, plant cell expressed glucocerebrosidase;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Hemoglobin | Median and interquartile range. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm. | Baseline, months 9, 12 and 24 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Chitotriosidase | Chitotriosidase. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm. | Baseline, months 9, 12 and 24 | 
| Spleen Volume | Spleen volume measured by MRI (or ultrasound). Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm. | Baseline, months 12 and 24 | 
| Platelet Count | Platelet count. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm. | Baseline, months 9, 12, 24 and 33-36 | 
| Liver Volume | Liver volume by MRI or Ultrasound. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm. | Baseline, months 12 and 24 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Gaucher disease, pediatrics