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A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease

Primary Purpose

Gaucher Disease

Status

Completed

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

Plant cell expressed recombinant glucocerebrosidase (prGCD)

About this trial

This is an interventional treatment trial for Gaucher Disease

Eligibility Criteria

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

Inclusion Criteria:

Males and females, 18 years or older
Confirmed enzymatic diagnosis of Gaucher disease
Splenomegaly defined as greater than eight times the expected volume (measured volume divided by estimated volume (0.2% of body weight)] as determined by MRI volumetric analysis
Female patients of child-bearing potential who agree to use a medically acceptable method of contraception
Thrombocytopenia (defined as platelet counts below the lower limit of normal) and/or anemia (defined by hemoglobin level at least 1 g/dL below normal range according to sex and age).
Patients who have not received ERT in the past or patients whoc have not received ERT in the past 12 months and have a negative anti-glucocerebrosidase antibody test.
Patients who have not received substrate reduction therapy (SRT) in the past 12 months.
Ability to provide a written informed consent.

Exclusion Criteria:

Currently taking another experimental drug for any condition
Pregnant or nursing
Presence of HIV and/or, HBsAg and/or hepatitis C infections
Presence of severe neurological signs and symptoms, defined as complete ocular paralysis, overt myoclonus or history of seizures, characteristic of neuronopathic Gaucher disease.
Previous anaphylactoid reaction to Cerezyme® or Ceredase®.
History of allergy to carrots.

Sites / Locations

University Research Foundation for Lysosomal Storage Diseases
Division of Medical Genetics, Emory University School of Medicine
New York University Medical Center
Mount Sinai Hospital
Pontificia Universidad Catolica de Chile
Rambam Medical Center
Shaare Zedek Medical Center
Universita "La Sapienza"
Morningside Medi-Clinic
Hospital Universitario Miguel Servet
Royal Free Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

prGCD 30 Units/kg

prGCD 60 Units/kg

Arm Description

Outcomes

Primary Outcome Measures

Change From Baseline in Spleen Volume Measured by MRI.

Calculated as percent change in spleen volume from Baseline to 9 months

Secondary Outcome Measures

Change From Baseline in Liver Volume

Calculated as percent change in liver volume from Baseline to 9 months

Change in Hemoglobin

Absolute change in Hemoglobin concentration from Baseline to Month 9

Change in Platelet Count

Change in Platelet count from Baseline to Month 9

Full Information

NCT ID

NCT00376168

First Posted

September 12, 2006

Last Updated

September 5, 2018

Sponsor

Pfizer

1. Study Identification

Unique Protocol Identification Number

NCT00376168

Brief Title

A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease

Official Title

A Phase III, Multicenter, Randomized, Double-Blind Trial to Assess the Safety and Efficacy 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

August 2007 (undefined)

Primary Completion Date

September 2009 (Actual)

Study Completion Date

October 2009 (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 the second trial to utilize a recombinant active form of lysosomal enzyme, glucocerebrosidase, (human prGCD) which is expressed and purified in a bioreactor system from transformed carrot plant root cell line.

Detailed Description

This will be a multi-center, randomized, double-blind, parallel group, dose-ranging trial to assess the safety and efficacy of prGCD in 30 untreated patients with Gaucher disease. Patients will receive IV infusion of prGCD every two weeks at the selected medical center. The duration of the study will be nine months. At the end of the 9-month treatment period (20 visits, 38 weeks) eligible patients will be offered enrollment in an open-label extension study. There will be two treatment groups, 15 patients in each treatment group. Treatment Group I: 30 units/kg every 2 weeks. Treatment Group II: 60 units/kg every 2 weeks. All patients will have pharmacokinetic data collected over approximately 3 hours with frequent blood samples following the first and final doses of prGCD.

6. Conditions and Keywords

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

Gaucher Disease

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

32 (Actual)

8. Arms, Groups, and Interventions

Arm Title

prGCD 30 Units/kg

Arm Type

Experimental

Arm Title

prGCD 60 Units/kg

Arm Type

Experimental

Intervention Type

Drug

Intervention Name(s)

Plant cell expressed recombinant glucocerebrosidase (prGCD)

Other Intervention Name(s)

Taliglucerase alfa

Intervention Description

Intravenous infusion every two weeks for 9 months

Intervention Type

Drug

Intervention Name(s)

Plant cell expressed recombinant glucocerebrosidase (prGCD)

Other Intervention Name(s)

Taliglucerase alfa

Intervention Description

Intravenous infusion every 2 weeks for 9 months

Primary Outcome Measure Information:

Title

Change From Baseline in Spleen Volume Measured by MRI.

Description

Calculated as percent change in spleen volume from Baseline to 9 months

Time Frame

Baseline and 9 months

Secondary Outcome Measure Information:

Title

Change From Baseline in Liver Volume

Description

Calculated as percent change in liver volume from Baseline to 9 months

Time Frame

Baseline and 9 months

Title

Change in Hemoglobin

Description

Absolute change in Hemoglobin concentration from Baseline to Month 9

Time Frame

Baseline and Month 9

Title

Change in Platelet Count

Description

Change in Platelet count from Baseline to Month 9

Time Frame

Baseline and Month 9

Other Pre-specified Outcome Measures:

Title

Change in Chitotriosidase

Description

Change in Chitotriosidase from Baseline to Month 9

Time Frame

Baseline and Month 9

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Males and females, 18 years or older Confirmed enzymatic diagnosis of Gaucher disease Splenomegaly defined as greater than eight times the expected volume (measured volume divided by estimated volume (0.2% of body weight)] as determined by MRI volumetric analysis Female patients of child-bearing potential who agree to use a medically acceptable method of contraception Thrombocytopenia (defined as platelet counts below the lower limit of normal) and/or anemia (defined by hemoglobin level at least 1 g/dL below normal range according to sex and age). Patients who have not received ERT in the past or patients whoc have not received ERT in the past 12 months and have a negative anti-glucocerebrosidase antibody test. Patients who have not received substrate reduction therapy (SRT) in the past 12 months. Ability to provide a written informed consent. Exclusion Criteria: Currently taking another experimental drug for any condition Pregnant or nursing Presence of HIV and/or, HBsAg and/or hepatitis C infections Presence of severe neurological signs and symptoms, defined as complete ocular paralysis, overt myoclonus or history of seizures, characteristic of neuronopathic Gaucher disease. Previous anaphylactoid reaction to Cerezyme® or Ceredase®. History of allergy to carrots.

Facility Information:

Facility Name

University Research Foundation for Lysosomal Storage Diseases

City

Coral Springs

State/Province

Florida

ZIP/Postal Code

33065

Country

United States

Facility Name

Division of Medical Genetics, Emory University School of Medicine

City

Decatur

State/Province

Georgia

ZIP/Postal Code

30033

Country

United States

Facility Name

New York University Medical Center

City

New York

State/Province

New York

ZIP/Postal Code

10016

Country

United States

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

ZIP/Postal Code

91031

Country

Israel

Facility Name

Universita "La Sapienza"

City

Rome

ZIP/Postal Code

00161

Country

Italy

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

Royal Free Hospital

City

London

ZIP/Postal Code

NW3 2QG

Country

United Kingdom

12. IPD Sharing Statement

Citations:

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

PubMed Identifier

21900191

Citation

Zimran A, Brill-Almon E, Chertkoff R, Petakov M, Blanco-Favela F, Munoz ET, Solorio-Meza SE, Amato D, Duran G, Giona F, Heitner R, Rosenbaum H, Giraldo P, Mehta A, Park G, Phillips M, Elstein D, Altarescu G, Szleifer M, Hashmueli S, Aviezer D. Pivotal trial with plant cell-expressed recombinant glucocerebrosidase, taliglucerase alfa, a novel enzyme replacement therapy for Gaucher disease. Blood. 2011 Nov 24;118(22):5767-73. doi: 10.1182/blood-2011-07-366955. Epub 2011 Sep 6. Erratum In: Blood. 2012 May 10;119(19):4577.

Results Reference

derived

PubMed Identifier

18729283

Citation

Winckler T. [Enzyme replacement therapy for Gaucher's Disease]. Pharm Unserer Zeit. 2008;37(5):352-3. doi: 10.1002/pauz.200890067. No abstract available. German.

Results Reference

derived

Learn more about this trial

A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease

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