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

Study Description

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Brief 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).

Condition or disease	Intervention/treatment	Phase
Gaucher Disease	Drug: Taliglucerase alfa	Phase 3

Access to an investigational treatment associated with this study is no longer available outside the clinical trial.   More info ...

Study Design

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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

Arms and Interventions

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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

Outcome Measures

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Primary Outcome Measures :

1. 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.

Secondary Outcome Measures :

1. 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.
2. 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.
3. 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.
4. 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.

Eligibility Criteria

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

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.

Contacts and Locations

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Locations

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

Sponsors and Collaborators

Protalix

Investigators

Principal Investigator:	Ari Zimran, MD	Shaare Zedek Medical Center

More Information

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Zimran A, Gonzalez-Rodriguez DE, Abrahamov A, Cooper PA, Varughese S, Giraldo P, Petakov M, Tan ES, Chertkoff R. Long-term safety and efficacy of taliglucerase alfa in pediatric Gaucher disease patients who were treatment-naïve or previously treated with imiglucerase. Blood Cells Mol Dis. 2018 Feb;68:163-172. doi: 10.1016/j.bcmd.2016.10.005. Epub 2016 Oct 20.

Abbas R, Park G, Damle B, Chertkoff R, Alon S. Pharmacokinetics of Novel Plant Cell-Expressed Taliglucerase Alfa in Adult and Pediatric Patients with Gaucher Disease. PLoS One. 2015 Jun 8;10(6):e0128986. doi: 10.1371/journal.pone.0128986. eCollection 2015.

Responsible Party:	Protalix
ClinicalTrials.gov Identifier:	NCT01411228 History of Changes
Other Study ID Numbers:	PB-06-006
First Posted:	August 8, 2011
Results First Posted:	January 18, 2016
Last Update Posted:	July 21, 2016
Last Verified:	June 2016

Keywords provided by Protalix:

Gaucher disease; pediatrics

Additional relevant MeSH terms:

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