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

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NCT00376168

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Study Location
University Research Foundation for Lysosomal Storage Diseases
Coral Springs, Florida, 33065, United States
See other locations
Contact
1-800-718-1021
[email protected]
Related Links

FOR MORE INFORMATION

Contact a representative by phone, email, or visiting the study website. Please see the references below:

By phone

Pfizer Clinical Trials Contact Center

1-800-718-1021

By email

Contact

[email protected]

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Eligibility Criteria
condition
The disease, disorder, syndrome, illness, or injury that is being studied.
Gaucher Disease
Sex
Females and Males
Age
Pediatric Trials: 0-17 Years
Adult Trials: 18+ Years
18 + years
Inclusion Criteria
The factors, or reasons, that allow a person to participate in a clinical study.
Show details

- 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
The factors, or reasons, that prevent a person from participating in a clinical study.
Show details


- 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.

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Advanced Information
Descriptive Information
Brief Title  ICMJE A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease
Official Title  ICMJE 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
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Gaucher Disease
Intervention  ICMJE
  • Drug: Plant cell expressed recombinant glucocerebrosidase (prGCD)
    Intravenous infusion every two weeks for 9 months
    Other Name: Taliglucerase alfa
  • Drug: Plant cell expressed recombinant glucocerebrosidase (prGCD)
    Intravenous infusion every 2 weeks for 9 months
    Other Name: Taliglucerase alfa
Study Arms  ICMJE
  • Experimental: prGCD 30 Units/kg
    Intervention: Drug: Plant cell expressed recombinant glucocerebrosidase (prGCD)
  • Experimental: prGCD 60 Units/kg
    Intervention: Drug: Plant cell expressed recombinant glucocerebrosidase (prGCD)
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: October 6, 2009)		32
Original Enrollment  ICMJE
 (submitted: September 12, 2006)		30
Actual Study Completion Date  ICMJE October 2009
Actual Primary Completion Date September 2009   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study:All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada,   Chile,   Israel,   Italy,   South Africa,   Spain,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00376168
Other Study ID Numbers  ICMJE PB-06-001
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Pfizer
Study Sponsor  ICMJE Pfizer
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Pfizer
Verification Date September 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP