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

Last updated on October 6, 2018

FOR MORE INFORMATION
Study Location
University Research Foundation for Lysosomal Storage Diseases
Coral Springs, Florida, 33065 United States
Contact
1-800-718-1021
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.

NCT00376168
Pfizer
Completed
A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease

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

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.

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.

Interventional
Phase 3
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Gaucher Disease
  • 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
  • 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)


*   Includes publications given by the data provider as well as publications
identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
32
30
October 2009
September 2009   (Final data collection date for primary outcome measure)

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.
Sexes Eligible for Study: All
18 Years and older   (Adult, Older Adult)
No
Contact information is only displayed when the study is recruiting subjects
Canada,   Chile,   Israel,   Italy,   South Africa,   Spain,   United Kingdom,   United States
 
 
NCT00376168
PB-06-001
Yes
Not Provided
Not Provided
Pfizer
Pfizer
Not Provided
Not Provided
Pfizer
September 2018

ICMJE     Data element required by the

International Committee of Medical Journal Editors
and the
World Health Organization ICTRP

FOR MORE INFORMATION

Contact a representative by phone, email, or visiting thte study website. To get updates and notications about this trail, sign up using the form below.

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1-800-718-1021

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[email protected]



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