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

Phase 3

Completed

• Conditions: Gaucher Disease
• Interventions: Drug: Taliglucerase alfa

• Registration Number: NCT01411228
• Lead Sponsor: Pfizer

Brief Summary

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).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2011-08-04
• Study First Submitted QC: 2011-08-04
• Study First Posted: 2011-08-08
• Study Start: 2011-09
• Primary Completion: 2014-07
• Study Completion: 2014-08
• Results First Submitted: 2015-11-06
• Results First Submitted QC: 2015-12-11
• Results First Posted: 2016-01-18
• Status Verified: 2018-09
• Last Update Submitted: 2018-09-05
• Last Update Posted: 2018-09-07

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 15

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
30 Units/kg	Taliglucerase alfa	-
60 Units/kg	Taliglucerase alfa	-

Primary Outcome Measures

Name	Time	Method
Hemoglobin	Baseline, months 9, 12 and 24	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.

Secondary Outcome Measures

Name	Time	Method
Chitotriosidase	Baseline, months 9, 12 and 24	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.
Spleen Volume	Baseline, months 12 and 24	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.
Platelet Count	Baseline, months 9, 12, 24 and 33-36	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.
Liver Volume	Baseline, months 12 and 24	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.

Trial Locations

Locations (3)

Shaare Zedek Medical Center; 🇮🇱 Jerusalem, Israel

Morningside Medi-Clinic; 🇿🇦 Morningside, South Africa

Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C); 🇵🇾 Barrio Asuncion, Paraguay