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

Pfizer15 enrolled

Overview

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).

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

Conditions

Gaucher Disease

Interventions

Taliglucerase alfaDRUG

Taliglucerase alfa for infusion every two weeks for 24 months

Eligibility

Sex: ALLMin age: 2 YearsMax age: 18 Years

Medical Language ↔ Plain English

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.

Locations (3)

Shaare Zedek Medical Center

Jerusalem, Israel

Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C)

Barrio Asunción, Paraguay

Morningside Medi-Clinic

Morningside, South Africa

Outcomes

Primary Outcomes

Hemoglobin

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.

Time frame: Baseline, months 9, 12 and 24

Secondary Outcomes

Chitotriosidase

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.

Time frame: Baseline, months 9, 12 and 24

Spleen Volume

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.

Time frame: Baseline, months 12 and 24

Platelet Count

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.

Time frame: Baseline, months 9, 12, 24 and 33-36

Liver Volume

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.

Time frame: Baseline, months 12 and 24

Data from ClinicalTrials.gov

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