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

A Multicenter, Double-blind, Randomized 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.

Study Overview

• Status: Completed
• Conditions: Gaucher Disease
• Intervention / Treatment: Drug: Taliglucerase alfa

• Study Type: Interventional
• Enrollment (Actual): 11
• Phase: Phase 4

Expanded Access

No longer available outside the clinical trial. See expanded access record.

Contacts and Locations

Study Locations

• Israel
  • Jerusalem, Israel
    • Shaare Zedek Medical Center
• Paraguay
  • Barrio Sajonia Asunción, Paraguay
    • Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C)
• South Africa
  • Morningside, South Africa, 2196
    • Morningside Medi-Clinic

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 17 years (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: QUADRUPLE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: 30 units/kg	Drug: Taliglucerase alfa; Taliglucerase alfa for infusion every two weeks for 12 months; Other Names: prGCD, plant cell expressed glucocerebrosidase
EXPERIMENTAL: 60 units/kg	Drug: Taliglucerase alfa; Taliglucerase alfa for infusion every two weeks for 12 months; Other Names: prGCD, plant cell expressed glucocerebrosidase

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Hemoglobin	median and interquartile range for change from baseline in haemoglobin	Every 3 months for 12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Chitotriosidase	Percent change from baseline in chitotriosidase	Every 3 months for 12 months
Spleen Volume	Spleen volume measured by MRI	Baseline and Month 12
Platelet Count	Mean and standard deviation of platelet count per cubic mm	Baseline and 12 months
Chemokine (C-C Motif) Ligand 18 (CCL18)	Percent change from baseline in CCL18	Every 3 months for 12 months
Liver Volume	Liver volume measured by MRI	Baseline and Month 12

Collaborators and Investigators

• Sponsor: Pfizer

Publications and helpful links

General Publications

• Zimran A, Gonzalez-Rodriguez DE, Abrahamov A, Elstein D, Paz A, Brill-Almon E, Chertkoff R. Safety and efficacy of two dose levels of taliglucerase alfa in pediatric patients with Gaucher disease. Blood Cells Mol Dis. 2015 Jan;54(1):9-16. doi: 10.1016/j.bcmd.2014.10.002. Epub 2014 Nov 7.

Study record dates

Study Major Dates

• Study Start: August 1, 2010
• Primary Completion (ACTUAL): May 1, 2012
• Study Completion (ACTUAL): July 1, 2012

Study Registration Dates

• First Submitted: May 26, 2010
• First Submitted That Met QC Criteria: May 26, 2010
• First Posted (ESTIMATE): May 28, 2010

Study Record Updates

• Last Update Posted (ACTUAL): October 5, 2018
• Last Update Submitted That Met QC Criteria: September 5, 2018
• Last Verified: September 1, 2018

More Information

Terms related to this study

• Keywords: pediatric; gaucher disease
• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Lipid Metabolism Disorders; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Lipidoses; Lipid Metabolism, Inborn Errors; Gaucher Disease
• Other Study ID Numbers: PB-06-005