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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00258778
Recruitment Status : Completed
First Posted : November 28, 2005
Last Update Posted : December 5, 2006
Information provided by:

November 23, 2005
November 28, 2005
December 5, 2006
November 2005
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  • Safety as measured by:
  • adverse events
  • change in vital signs
  • physical examination
  • laboratory test results
Same as current
Complete list of historical versions of study NCT00258778 on Archive Site
  • Pharmacokinetic parameters
  • Immunological profile including: IgE, anti human prGCD antibodies, eosinophils and proteinuria
Same as current
Not Provided
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Phase I Single Dose-Escalation Safety Study of Human Glucocerebrosidase (prGCD)
A Phase I, Non-Randomized, Open Label, Single Dose-Escalation Safety Study of Recombinant Human Glucocerebrosidase (prGCD) in Healthy Volunteers

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.

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Phase 1
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Gaucher Disease
Drug: Human Glucocerebrosidase (prGCD)
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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.

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Same as current
January 2006
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Inclusion Criteria:

  1. Healthy male or female between 18 and 45 years of age.
  2. 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.
  3. Females of child-bearing potential must be non-pregnant and not lactating and using adequate birth control such as oral contraceptives.
  4. Negative laboratory tests for HIV, HBsAg or HCV.
  5. Naive to any previous recombinant protein therapy.
  6. Provide written informed consent.
  7. Have the ability to understand the requirements of the study and to comply with the study protocol and dosing regimen.

Exclusion Criteria:

  1. 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.
  2. Are pregnant or nursing.
  3. Presence of any acute or chronic diseases.
  4. Have a history of any allergies.
  5. Have been exposed to long-term steroid treatment.
  6. Had a minor operation in the last 6 months.
  7. Have ever been exposed to any previous recombinant protein therapy.
  8. Have received immuno-suppressive treatment.
  9. Have a positive HIV, HBsAG and HCV laboratory result.
  10. Use any medication other than vitamins or oral contraceptives (for female).
  11. Have participated in another clinical trial during the previous 3 months
  12. Have history of alcohol or drug abuse.
  13. Are considered by the Investigator to be unsuitable candidate for this study.
Sexes Eligible for Study: All
18 Years to 45 Years   (Adult)
Contact information is only displayed when the study is recruiting subjects
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Principal Investigator: Eithan Galun, MD Protalix Ltd.
December 2006

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