Clinical trial NCT00258778: 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
Overall status: COMPLETED
Phase: PHASE1
Conditions: Gaucher Disease
Lead sponsor: Protalix
Sponsor class: INDUSTRY
First posted: 2005-11-28
Enrollment: 6
Sex: ALL
Minimum age (years): 18
Maximum age (years): 45
Standard ages: ADULT
Accepts healthy volunteers: yes
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility 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.

Primary outcomes:
measure: Safety as measured by:
measure: adverse events
measure: change in vital signs
measure: physical examination
measure: laboratory test results
Secondary outcomes:
measure: Pharmacokinetic parameters
measure: Immunological profile including: IgE, anti human prGCD antibodies, eosinophils and proteinuria
Other outcomes:
Locations:
Study officials:
affiliation: Protalix Ltd.
name: Eithan Galun, MD
role: PRINCIPAL_INVESTIGATOR
References:
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.