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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.
 
ClinicalTrials.gov Identifier: NCT00258778
Recruitment Status : Completed
First Posted : November 28, 2005
Last Update Posted : December 5, 2006
Sponsor:
Information provided by:
Protalix

Study Description
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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.


Condition or disease Intervention/treatment Phase
Gaucher Disease Drug: Human Glucocerebrosidase (prGCD) Phase 1

Study Design
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Study Type : Interventional  (Clinical Trial)
Enrollment : 6 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I, Non-Randomized, Open Label, Single Dose-Escalation Safety Study of Recombinant Human Glucocerebrosidase (prGCD) in Healthy Volunteers
Study Start Date : November 2005
Study Completion Date : January 2006

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Gaucher Disease

Arms and Interventions
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Outcome Measures
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Primary Outcome Measures :
  1. Safety as measured by:
  2. adverse events
  3. change in vital signs
  4. physical examination
  5. laboratory test results

Secondary Outcome Measures :
  1. Pharmacokinetic parameters
  2. Immunological profile including: IgE, anti human prGCD antibodies, eosinophils and proteinuria

Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 45 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00258778


Sponsors and Collaborators
Protalix
Investigators
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Principal Investigator: Eithan Galun, MD Protalix Ltd.
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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ClinicalTrials.gov Identifier: NCT00258778    
Other Study ID Numbers: P-01-2005
First Posted: November 28, 2005    Key Record Dates
Last Update Posted: December 5, 2006
Last Verified: December 2006
Additional relevant MeSH terms:
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Gaucher Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
 Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders