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Phase I Single Dose-Escalation Safety Study of Human Glucocerebrosidase (prGCD)

Primary Purpose

Gaucher Disease

Status
Completed
Phase
Phase 1
Locations
Study Type
Interventional
Intervention
Human Glucocerebrosidase (prGCD)
Sponsored by
Protalix
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Gaucher Disease

Eligibility Criteria

18 Years - 45 Years (Adult)All SexesAccepts Healthy Volunteers

Inclusion Criteria: Healthy male or female between 18 and 45 years of age. Female subjects must agree to use a medically acceptable method of contraception at all times during the study and must have a negative serum pregnancy test at baseline and during the study period. Females of child-bearing potential must be non-pregnant and not lactating and using adequate birth control such as oral contraceptives. Negative laboratory tests for HIV, HBsAg or HCV. Naive to any previous recombinant protein therapy. Provide written informed consent. Have the ability to understand the requirements of the study and to comply with the study protocol and dosing regimen. Exclusion Criteria: Have clinical evidence of any active significant disease that could potentially compromise the ability of the investigator to evaluate or interpret the effects of the study treatment on safety assessment and thus increase the risk to the subject to unacceptable levels. Are pregnant or nursing. Presence of any acute or chronic diseases. Have a history of any allergies. Have been exposed to long-term steroid treatment. Had a minor operation in the last 6 months. Have ever been exposed to any previous recombinant protein therapy. Have received immuno-suppressive treatment. Have a positive HIV, HBsAG and HCV laboratory result. Use any medication other than vitamins or oral contraceptives (for female). Have participated in another clinical trial during the previous 3 months Have history of alcohol or drug abuse. Are considered by the Investigator to be unsuitable candidate for this study.

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Safety as measured by:
    adverse events
    change in vital signs
    physical examination
    laboratory test results

    Secondary Outcome Measures

    Pharmacokinetic parameters
    Immunological profile including: IgE, anti human prGCD antibodies, eosinophils and proteinuria

    Full Information

    First Posted
    November 23, 2005
    Last Updated
    December 4, 2006
    Sponsor
    Protalix
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    1. Study Identification

    Unique Protocol Identification Number
    NCT00258778
    Brief Title
    Phase I Single Dose-Escalation Safety Study of Human Glucocerebrosidase (prGCD)
    Official Title
    A Phase I, Non-Randomized, Open Label, Single Dose-Escalation Safety Study of Recombinant Human Glucocerebrosidase (prGCD) in Healthy Volunteers
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    December 2006
    Overall Recruitment Status
    Completed
    Study Start Date
    November 2005 (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    January 2006 (undefined)

    3. Sponsor/Collaborators

    Name of the Sponsor
    Protalix

    4. Oversight

    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 first 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.

    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 1
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    Non-Randomized
    Enrollment
    6 (false)

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    Human Glucocerebrosidase (prGCD)
    Primary Outcome Measure Information:
    Title
    Safety as measured by:
    Title
    adverse events
    Title
    change in vital signs
    Title
    physical examination
    Title
    laboratory test results
    Secondary Outcome Measure Information:
    Title
    Pharmacokinetic parameters
    Title
    Immunological profile including: IgE, anti human prGCD antibodies, eosinophils and proteinuria

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Maximum Age & Unit of Time
    45 Years
    Accepts Healthy Volunteers
    Accepts Healthy Volunteers
    Eligibility Criteria
    Inclusion Criteria: Healthy male or female between 18 and 45 years of age. Female subjects must agree to use a medically acceptable method of contraception at all times during the study and must have a negative serum pregnancy test at baseline and during the study period. Females of child-bearing potential must be non-pregnant and not lactating and using adequate birth control such as oral contraceptives. Negative laboratory tests for HIV, HBsAg or HCV. Naive to any previous recombinant protein therapy. Provide written informed consent. Have the ability to understand the requirements of the study and to comply with the study protocol and dosing regimen. Exclusion Criteria: Have clinical evidence of any active significant disease that could potentially compromise the ability of the investigator to evaluate or interpret the effects of the study treatment on safety assessment and thus increase the risk to the subject to unacceptable levels. Are pregnant or nursing. Presence of any acute or chronic diseases. Have a history of any allergies. Have been exposed to long-term steroid treatment. Had a minor operation in the last 6 months. Have ever been exposed to any previous recombinant protein therapy. Have received immuno-suppressive treatment. Have a positive HIV, HBsAG and HCV laboratory result. Use any medication other than vitamins or oral contraceptives (for female). Have participated in another clinical trial during the previous 3 months Have history of alcohol or drug abuse. Are considered by the Investigator to be unsuitable candidate for this study.
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Eithan Galun, MD
    Organizational Affiliation
    Protalix Ltd.
    Official's Role
    Principal Investigator

    12. IPD Sharing Statement

    Citations:
    PubMed Identifier
    19277123
    Citation
    Aviezer D, Brill-Almon E, Shaaltiel Y, Hashmueli S, Bartfeld D, Mizrachi S, Liberman Y, Freeman A, Zimran A, Galun E. A plant-derived recombinant human glucocerebrosidase enzyme--a preclinical and phase I investigation. PLoS One. 2009;4(3):e4792. doi: 10.1371/journal.pone.0004792. Epub 2009 Mar 11.
    Results Reference
    derived

    Learn more about this trial

    Phase I Single Dose-Escalation Safety Study of Human Glucocerebrosidase (prGCD)

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