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

This study is enrolling participants by invitation only.
Sponsor:
Information provided by (Responsible Party):
Protalix
ClinicalTrials.gov Identifier:
NCT01411228
First received: August 4, 2011
Last updated: September 21, 2012
Last verified: September 2012
History of Changes
  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 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:

U.S. FDA Resources

Further study details as provided by Protalix:

Primary Outcome Measures:
  • Hemoglobin [ Time Frame: Every 3 months for 2 years ] [ Designated as safety issue: No ]
    median percentage and the interquartile range for change from baseline in haemoglobin


Secondary Outcome Measures:
  • Chitotriosidase [ Time Frame: Every 3 months for 2 years ] [ Designated as safety issue: No ]
    Percent change from baseline in chitotriosidase

  • Spleen and liver volume [ Time Frame: at 1 and 2 years ] [ Designated as safety issue: No ]
    Percent change in spleen and liver volume measured by MRI (or ultrasound)

  • Platelet count [ Time Frame: Every 3 months for 2 years ] [ Designated as safety issue: No ]
    Percent change from baseline in platelet count

  • Anti taliglucerase alfa antibodies [ Time Frame: Every 3 months for 2 years ] [ Designated as safety issue: Yes ]
    Occurrence of positive antibody response


Estimated Enrollment: 15
Study Start Date: September 2011
Estimated Study Completion Date: March 2014
Estimated Primary Completion Date: December 2013 (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
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
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

No publications provided

Responsible Party: Protalix
ClinicalTrials.gov Identifier: NCT01411228     History of Changes
Other Study ID Numbers: PB-06-006
Study First Received: August 4, 2011
Last Updated: September 21, 2012
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 May 23, 2013