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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT00258778
Other study ID # P-01-2005
Secondary ID
Status Completed
Phase Phase 1
First received November 23, 2005
Last updated December 4, 2006
Start date November 2005
Est. completion date January 2006

Study information

Verified date December 2006
Source Protalix
Contact n/a
Is FDA regulated No
Health authority United States: Food and Drug Administration
Study type Interventional

Clinical Trial 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.


Recruitment information / eligibility

Status Completed
Enrollment 6
Est. completion date January 2006
Est. primary completion date
Accepts healthy volunteers Accepts Healthy Volunteers
Gender Both
Age group 18 Years to 45 Years
Eligibility 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.

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment


Related Conditions & MeSH terms


Intervention

Drug:
Human Glucocerebrosidase (prGCD)


Locations

Country Name City State
n/a

Sponsors (1)

Lead Sponsor Collaborator
Protalix

Outcome

Type Measure Description Time frame Safety issue
Primary Safety as measured by:
Primary adverse events
Primary change in vital signs
Primary physical examination
Primary laboratory test results
Secondary Pharmacokinetic parameters
Secondary Immunological profile including: IgE, anti human prGCD antibodies, eosinophils and proteinuria
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