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NCT Number: NCT01132690

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

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.

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Key information

Conditions

Gaucher Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Sphingolipidoses

Age range

2 year–17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Shaare Zedek Medical Center, Jerusalem, Israel

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Taliglucerase alfa

Drug

Taliglucerase alfa for infusion every two weeks for 12 months

Other names: prGCD, plant cell expressed glucocerebrosidase

Primary outcomes

  1. Hemoglobin

    Time frame: Every 3 months for 12 months

    median and interquartile range for change from baseline in haemoglobin

Secondary outcomes

  1. Chitotriosidase

    Time frame: Every 3 months for 12 months

    Percent change from baseline in chitotriosidase

  2. Spleen Volume

    Time frame: Baseline and Month 12

    Spleen volume measured by MRI

  3. Platelet Count

    Time frame: Baseline and 12 months

    Mean and standard deviation of platelet count per cubic mm

  4. Chemokine (C-C Motif) Ligand 18 (CCL18)

    Time frame: Every 3 months for 12 months

    Percent change from baseline in CCL18

  5. Liver Volume

    Time frame: Baseline and Month 12

    Liver volume measured by MRI

Sponsors and collaborators

Lead sponsor

Pfizer

Industry

Registry information

Official study title

A Multicenter, Double-blind, Randomized Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

Important dates

Study start
2010
Primary completion
2012
Study completion
2012
First posted
May 28, 2010
Registry last updated
Oct 5, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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