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Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

Primary Purpose

Gaucher Disease

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Taliglucerase alfa
Sponsored by
Pfizer
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Gaucher Disease focused on measuring Gaucher Disease, Enzyme replacement therapy

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Successful completion of Protocol PB-06-001
  • The patient signs informed consent

Exclusion Criteria:

  • Currently taking another experimental drug for any condition
  • Presence of severe neurological signs and symptoms, defined as complete ocular paralysis, overt myoclonus or history of seizures, characteristic of neuronopathic Gaucher disease
  • Pregnant or nursing
  • Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the patient's compliance with the requirements of the study

Sites / Locations

  • Department of Human Genetics, Emory University School of Medicine
  • Neurogenetics, NYU at Rivergate
  • Bone Marrow Transplant Service, The Royal Melbourne Hospital
  • Mount Sinai Hospital
  • Pontificia Universidad Catolica de Chile
  • Rambam Medical Center
  • Shaare Zedek Medical Center
  • Morningside Medi-Clinic
  • Hospital Universitario Miguel Servet
  • Lysosomal Disorders Service, Addenbrookes Hospital NHS Trust
  • Royal Free Hospital

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Experimental

Arm Label

Naive 30 Units/kg

Naive 60 Units/kg

Switchover

Arm Description

Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168)

Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168)

Continue taliglucerase alfa treatment from PB-06-002 (NCT00712348)

Outcomes

Primary Outcome Measures

Spleen Volume
Spleen volume measured by MRI

Secondary Outcome Measures

Liver Volume
Liver volume measured by MRI
Hemoglobin
Platelet Count

Full Information

First Posted
June 25, 2008
Last Updated
September 5, 2018
Sponsor
Pfizer
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1. Study Identification

Unique Protocol Identification Number
NCT00705939
Brief Title
Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial
Official Title
A Multicenter, Double-Blind, Extension Trial of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients With Gaucher Disease
Study Type
Interventional

2. Study Status

Record Verification Date
September 2018
Overall Recruitment Status
Completed
Study Start Date
June 2008 (undefined)
Primary Completion Date
May 2012 (Actual)
Study Completion Date
August 2013 (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
Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD) leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the accumulation of substrate glucocerebroside (GlcCer) in the cells of the monocyte-macrophage system. This is an extension trial to Study NCT00376168 and NCT00712348.
Detailed Description
This will be a multi-center, double-blind, parallel group, extension trial to assess the safety and efficacy of prGCD in patients completing NCT00376168. Patients will receive IV infusion of prGCD every two weeks at the selected medical center. The duration of the extension study will be fifteen months. There will be two treatment groups: 30 units/kg every 2 weeks or 60 units/kg every 2 weeks.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Gaucher Disease
Keywords
Gaucher Disease, Enzyme replacement therapy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
45 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Naive 30 Units/kg
Arm Type
Experimental
Arm Description
Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168)
Arm Title
Naive 60 Units/kg
Arm Type
Experimental
Arm Description
Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168)
Arm Title
Switchover
Arm Type
Experimental
Arm Description
Continue taliglucerase alfa treatment from PB-06-002 (NCT00712348)
Intervention Type
Drug
Intervention Name(s)
Taliglucerase alfa
Other Intervention Name(s)
Plant Cell Expressed Recombinant Human Glucocerebrosidase, prGCD
Intervention Description
Intravenous infusion every 2 weeks
Primary Outcome Measure Information:
Title
Spleen Volume
Description
Spleen volume measured by MRI
Time Frame
Spleen Volume at Baseline and Months 12, 24, and 36
Secondary Outcome Measure Information:
Title
Liver Volume
Description
Liver volume measured by MRI
Time Frame
Liver volume at Baseline and Months 12, 24 and 36
Title
Hemoglobin
Time Frame
Hemoglobin at Baseline and Months 12, 24 and 36
Title
Platelet Count
Time Frame
Platelet count at Baseline and Months 12, 24 and 36
Other Pre-specified Outcome Measures:
Title
Spleen Volume Multiples of Normal (MN)
Description
Spleen volume measured by MRI. Normal spleen volume is 2 mL/kg × body weight (kg)
Time Frame
Baseline and Months 12, 24, and 36
Title
Liver Volume Multiples of Normal (MN)
Description
Liver volume measured by MRI. Normal liver volume is 25 mL/kg × body weight (kg).
Time Frame
Baseline and Months 12, 24 and 36

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Successful completion of Protocol PB-06-001 The patient signs informed consent Exclusion Criteria: Currently taking another experimental drug for any condition Presence of severe neurological signs and symptoms, defined as complete ocular paralysis, overt myoclonus or history of seizures, characteristic of neuronopathic Gaucher disease Pregnant or nursing Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the patient's compliance with the requirements of the study
Facility Information:
Facility Name
Department of Human Genetics, Emory University School of Medicine
City
Decatur
State/Province
Georgia
ZIP/Postal Code
30033
Country
United States
Facility Name
Neurogenetics, NYU at Rivergate
City
New York
State/Province
New York
ZIP/Postal Code
10016
Country
United States
Facility Name
Bone Marrow Transplant Service, The Royal Melbourne Hospital
City
Parkville
State/Province
Victoria
Country
Australia
Facility Name
Mount Sinai Hospital
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 1X5
Country
Canada
Facility Name
Pontificia Universidad Catolica de Chile
City
Santiago
Country
Chile
Facility Name
Rambam Medical Center
City
Haifa
ZIP/Postal Code
31096
Country
Israel
Facility Name
Shaare Zedek Medical Center
City
Jerusalem
Country
Israel
Facility Name
Morningside Medi-Clinic
City
Morningside
ZIP/Postal Code
2196
Country
South Africa
Facility Name
Hospital Universitario Miguel Servet
City
Zaragoza
ZIP/Postal Code
50009
Country
Spain
Facility Name
Lysosomal Disorders Service, Addenbrookes Hospital NHS Trust
City
Cambridge
Country
United Kingdom
Facility Name
Royal Free Hospital
City
London
ZIP/Postal Code
NW3 2QG
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
27174694
Citation
Zimran A, Duran G, Mehta A, Giraldo P, Rosenbaum H, Giona F, Amato DJ, Petakov M, Munoz ET, Solorio-Meza SE, Cooper PA, Varughese S, Chertkoff R, Brill-Almon E. Long-term efficacy and safety results of taliglucerase alfa up to 36 months in adult treatment-naive patients with Gaucher disease. Am J Hematol. 2016 Jul;91(7):656-60. doi: 10.1002/ajh.24369. Epub 2016 Apr 24.
Results Reference
derived
PubMed Identifier
27102949
Citation
Pastores GM, Shankar SP, Petakov M, Giraldo P, Rosenbaum H, Amato DJ, Szer J, Chertkoff R, Brill-Almon E, Zimran A. Enzyme replacement therapy with taliglucerase alfa: 36-month safety and efficacy results in adult patients with Gaucher disease previously treated with imiglucerase. Am J Hematol. 2016 Jul;91(7):661-5. doi: 10.1002/ajh.24399. Epub 2016 May 18.
Results Reference
derived

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Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

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