A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease - Full Text View

Purpose

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	Intervention	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 Type:	Interventional
Study Design:	Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, Investigator) Primary Purpose: Treatment
Official Title:	A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

Resource links provided by NLM:

Genetics Home Reference related topics: Chanarin-Dorfman syndrome Gaucher disease Schindler disease cholesteryl ester storage disease succinic semialdehyde dehydrogenase deficiency

MedlinePlus related topics: Gaucher Disease

Drug Information available for: Taliglucerase alfa

Genetic and Rare Diseases Information Center resources: Gaucher Disease Sphingolipidosis

U.S. FDA Resources

Further study details as provided by Protalix:

Primary Outcome Measures:

Hemoglobin [ Time Frame: Baseline, months 9, 12 and 24 ] [ Designated as safety issue: No ]
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:

Chitotriosidase [ Time Frame: Baseline, months 9, 12 and 24 ] [ Designated as safety issue: No ]
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 ] [ Designated as safety issue: No ]
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 ] [ Designated as safety issue: No ]
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 ] [ Designated as safety issue: No ]
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.


Enrollment:	15
Study Start Date:	September 2011
Study Completion Date:	August 2014
Primary Completion Date:	July 2014 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
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

Eligibility


Ages Eligible for Study:	2 Years to 18 Years (Child, Adult)
Genders Eligible for Study:	Both
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

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01411228

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

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

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
Study First Received:	August 4, 2011
Results First Received:	November 6, 2015
Last Updated:	June 20, 2016
Health Authority:	United States: Food and Drug Administration Israel: Ministry of Health

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

ClinicalTrials.gov processed this record on September 29, 2016