A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
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ClinicalTrials.gov Identifier: NCT01411228 |
Recruitment Status :
Completed
First Posted : August 8, 2011
Results First Posted : January 18, 2016
Last Update Posted : September 7, 2018
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Gaucher Disease | Drug: Taliglucerase alfa | Phase 3 |
Expanded Access : An investigational treatment associated with this study is no longer available outside the clinical trial. More info ...
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 15 participants |
Allocation: | Non-Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Triple (Participant, Care Provider, Investigator) |
Primary Purpose: | Treatment |
Official Title: | A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease |
Study Start Date : | September 2011 |
Actual Primary Completion Date : | July 2014 |
Actual Study Completion Date : | August 2014 |

Arm | Intervention/treatment |
---|---|
Experimental: 60 Units/kg |
Drug: Taliglucerase alfa
Taliglucerase alfa for infusion every two weeks for 24 months
Other Name: prGCD, plant cell expressed glucocerebrosidase |
Experimental: 30 Units/kg |
Drug: Taliglucerase alfa
Taliglucerase alfa for infusion every two weeks for 24 months
Other Name: prGCD, plant cell expressed glucocerebrosidase |
- Hemoglobin [ Time Frame: Baseline, months 9, 12 and 24 ]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.
- Chitotriosidase [ Time Frame: Baseline, months 9, 12 and 24 ]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.
- Spleen Volume [ Time Frame: Baseline, months 12 and 24 ]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.
- Platelet Count [ Time Frame: Baseline, months 9, 12, 24 and 33-36 ]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.
- Liver Volume [ Time Frame: Baseline, months 12 and 24 ]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.

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Ages Eligible for Study: | 2 Years to 18 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01411228
Israel | |
Shaare Zedek Medical Center | |
Jerusalem, Israel | |
Paraguay | |
Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C) | |
Barrio Asuncion, Paraguay | |
South Africa | |
Morningside Medi-Clinic | |
Morningside, South Africa |
Responsible Party: | Pfizer |
ClinicalTrials.gov Identifier: | NCT01411228 |
Other Study ID Numbers: |
PB-06-006 |
First Posted: | August 8, 2011 Key Record Dates |
Results First Posted: | January 18, 2016 |
Last Update Posted: | September 7, 2018 |
Last Verified: | September 2018 |
Gaucher disease pediatrics |
Gaucher Disease Sphingolipidoses Lysosomal Storage Diseases, Nervous System Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases |
Metabolism, Inborn Errors Genetic Diseases, Inborn Lipidoses Lipid Metabolism, Inborn Errors Lysosomal Storage Diseases Metabolic Diseases Lipid Metabolism Disorders |