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A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Protalix
ClinicalTrials.gov Identifier:
NCT01132690
First received: May 26, 2010
Last updated: May 10, 2012
Last verified: May 2012
History of Changes
  Purpose

This is a multi-center, double-blind trial to assess the safety and efficacy of taliglucerase alfa in untreated subjects (2 to <18 years old) with Gaucher disease randomly assigned to treatment with one of two doses, 30 or 60 units/kg. Subjects will receive an intravenous (IV) infusion of taliglucerase alfa every two weeks. The total duration of treatment will be 12 months. At the end of the 12-month treatment period eligible subjects will be offered enrollment in an open-label extension study if taliglucerase alfa is not commercially available.


Condition Intervention Phase
Gaucher Disease
 Drug: Taliglucerase alfa
 Phase 4

Access to an investigational treatment associated with this study is available outside the clinical trial.   More info ...

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Multicenter, Double-blind, Randomized Safety and Efficacy Study of Two Dose Levels 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 12 months ] [ Designated as safety issue: No ]
    median percentage and the interquartile range for change from baseline in haemoglobin


Secondary Outcome Measures:
  • Chitotriosidase or CCL18 [ Time Frame: Every 3 months for 12 months ] [ Designated as safety issue: No ]
    Percent change from baseline in chitotriosidase or CCL18

  • Spleen and liver volume [ Time Frame: Baseline and Month 12 ] [ 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 12 months ] [ Designated as safety issue: No ]
    Percent change from baseline in platelet count

  • Anti-taliglucerase alfa antibodies [ Time Frame: Every 3 months for 12 months ] [ Designated as safety issue: Yes ]
    Occurrence of positive antibody response


Estimated Enrollment: 10
Study Start Date: August 2010
Estimated Study Completion Date: July 2012
Primary Completion Date: May 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 30 units/kg Drug: Taliglucerase alfa
Taliglucerase alfa for infusion every two weeks for 12 months
Other Name: prGCD, plant cell expressed glucocerebrosidase
Experimental: 60 units/kg Drug: Taliglucerase alfa
Taliglucerase alfa for infusion every two weeks for 12 months
Other Name: prGCD, plant cell expressed glucocerebrosidase

  Eligibility

Ages Eligible for Study:   2 Years to 17 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Males and females 2 to <18 years old.
  • Diagnosis of Gaucher disease with leukocyte acid β-glucosidase activity ≤30% of the mean of the reference range for healthy subjects.
  • Subjects who have not received enzyme replacement therapy (ERT) in the past or who have not received ERT in the past 12 months and have a negative anti-glucocerebrosidase antibody assay.
  • Subjects who have not received substrate reduction therapy (SRT) in the past 12 months.
  • Subjects whose clinical condition, in the opinion of the investigator, requires treatment with enzyme replacement therapy (ERT).

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 unresolved anemia due to iron, folic acid, or vitamin B12 deficiency
  • Previous hypersensitivity reaction to Cerezyme® (imiglucerase) or Ceredase® (alglucerase).
  • History of allergy to carrots.
  • Presence of HIV, HBsAg or hepatitis C infections.
  • Subject's parent(s) or legal guardian(s) are unable to understand the nature, scope and possible consequences of the study.
  • 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: NCT01132690

Locations
Israel
Shaare Zedek Medical Center
Jerusalem, Israel
Paraguay
Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C)
Barrio Sajonia Asunción, Paraguay
South Africa
Morningside Medi-Clinic
Morningside, South Africa, 2196
Sponsors and Collaborators
Protalix
Investigators
Study Director: Ari Zimran, MD Shaare Zedek Medical Center
  More Information

No publications provided

Responsible Party: Protalix
ClinicalTrials.gov Identifier: NCT01132690     History of Changes
Other Study ID Numbers: PB-06-005
Study First Received: May 26, 2010
Last Updated: May 10, 2012
Health Authority: United States: Food and Drug Administration
Israel: Ministry of Health
Canada: Health Canada

Keywords provided by Protalix:
gaucher disease
pediatric

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