Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

NCT00705939

Last updated date
Study Location
Department of Human Genetics, Emory University School of Medicine
Decatur, Georgia, 30033, United States
Contact
1-800-718-1021

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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

- Successful completion of Protocol PB-06-001

- The patient signs 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


- 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

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Advanced Information
Descriptive Information
Brief Title  ICMJE Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial
Official Title  ICMJE A Multicenter, Double-Blind, Extension Trial 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 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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Gaucher Disease
Intervention  ICMJE Drug: Taliglucerase alfa
Intravenous infusion every 2 weeks
Other Names:
  • Plant Cell Expressed Recombinant Human Glucocerebrosidase
  • prGCD
Study Arms  ICMJE
  • Experimental: Naive 30 Units/kg
    Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168)
    Intervention: Drug: Taliglucerase alfa
  • Experimental: Naive 60 Units/kg
    Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168)
    Intervention: Drug: Taliglucerase alfa
  • Experimental: Switchover
    Continue taliglucerase alfa treatment from PB-06-002 (NCT00712348)
    Intervention: Drug: Taliglucerase alfa
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: July 14, 2014)
45
Original Estimated Enrollment  ICMJE
 (submitted: June 25, 2008)
25
Actual Study Completion Date  ICMJE August 2013
Actual Primary Completion Date May 2012   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
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 Australia,   Canada,   Chile,   Israel,   South Africa,   Spain,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00705939
Other Study ID Numbers  ICMJE PB-06-003
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