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

Primary Purpose

Gaucher Disease

Status

Completed

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

Taliglucerase alfa

About this trial

This is an interventional treatment trial for Gaucher Disease focused on measuring Gaucher disease, pediatrics

Eligibility Criteria

2 Years - 18 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Shaare Zedek Medical Center
Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C)
Morningside Medi-Clinic

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

60 Units/kg

30 Units/kg

Arm Description

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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.

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.

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.

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.

Full Information

NCT ID

NCT01411228

First Posted

August 4, 2011

Last Updated

September 5, 2018

Sponsor

Pfizer

1. Study Identification

Unique Protocol Identification Number

NCT01411228

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

September 2018

Overall Recruitment Status

Completed

Study Start Date

September 2011 (undefined)

Primary Completion Date

July 2014 (Actual)

Study Completion Date

August 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Pfizer

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Gaucher Disease

Keywords

Gaucher disease, pediatrics

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigator

Allocation

Non-Randomized

Enrollment

15 (Actual)

8. Arms, Groups, and Interventions

Arm Title

60 Units/kg

Arm Type

Experimental

Arm Title

30 Units/kg

Arm Type

Experimental

Intervention Type

Drug

Intervention Name(s)

Taliglucerase alfa

Other Intervention Name(s)

prGCD, plant cell expressed glucocerebrosidase

Intervention Description

Taliglucerase alfa for infusion every two weeks for 24 months

Primary Outcome Measure Information:

Title

Hemoglobin

Description

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 Outcome Measure Information:

Title

Chitotriosidase

Description

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

Title

Spleen Volume

Description

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

Title

Platelet Count

Description

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

Title

Liver Volume

Description

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

10. Eligibility

Sex

All

Minimum Age & Unit of Time

2 Years

Maximum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

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.

Facility Information:

Facility Name

Shaare Zedek Medical Center

City

Jerusalem

Country

Israel

Facility Name

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

City

Barrio Asuncion

Country

Paraguay

Facility Name

Morningside Medi-Clinic

City

Morningside

Country

South Africa

12. IPD Sharing Statement

Citations:

PubMed Identifier

27839981

Citation

Zimran A, Gonzalez-Rodriguez DE, Abrahamov A, Cooper PA, Varughese S, Giraldo P, Petakov M, Tan ES, Chertkoff R. Long-term safety and efficacy of taliglucerase alfa in pediatric Gaucher disease patients who were treatment-naive or previously treated with imiglucerase. Blood Cells Mol Dis. 2018 Feb;68:163-172. doi: 10.1016/j.bcmd.2016.10.005. Epub 2016 Oct 20.

Results Reference

derived

PubMed Identifier

26053270

Citation

Abbas R, Park G, Damle B, Chertkoff R, Alon S. Pharmacokinetics of Novel Plant Cell-Expressed Taliglucerase Alfa in Adult and Pediatric Patients with Gaucher Disease. PLoS One. 2015 Jun 8;10(6):e0128986. doi: 10.1371/journal.pone.0128986. eCollection 2015.

Results Reference

derived

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

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