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

September 5, 2018 updated by: Pfizer
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 Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

15

Phase

  • Phase 3

Expanded Access

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

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 18 years (ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NON_RANDOMIZED
  • Interventional Model: PARALLEL
  • Masking: TRIPLE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: 60 Units/kg
Drug: Taliglucerase alfa
Taliglucerase alfa for infusion every two weeks for 24 months
Other Names:
  • prGCD, plant cell expressed glucocerebrosidase
EXPERIMENTAL: 30 Units/kg
Drug: Taliglucerase alfa
Taliglucerase alfa for infusion every two weeks for 24 months
Other Names:
  • prGCD, plant cell expressed glucocerebrosidase

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Hemoglobin
Time Frame: 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.
Baseline, months 9, 12 and 24

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Chitotriosidase
Time Frame: 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.
Baseline, months 9, 12 and 24
Spleen Volume
Time Frame: 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.
Baseline, months 12 and 24
Platelet Count
Time Frame: 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.
Baseline, months 9, 12, 24 and 33-36
Liver Volume
Time Frame: 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.
Baseline, months 12 and 24

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Pfizer

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

September 1, 2011

Primary Completion (ACTUAL)

July 1, 2014

Study Completion (ACTUAL)

August 1, 2014

Study Registration Dates

First Submitted

August 4, 2011

First Submitted That Met QC Criteria

August 4, 2011

First Posted (ESTIMATE)

August 8, 2011

Study Record Updates

Last Update Posted (ACTUAL)

September 7, 2018

Last Update Submitted That Met QC Criteria

September 5, 2018

Last Verified

September 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • PB-06-006

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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