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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

6 participants

Study Classification

INTERVENTIONAL

Study Start Date

2005-11-30

Study Completion Date

2006-01-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Gaucher Disease

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Human Glucocerebrosidase (prGCD)

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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.

Minimum Eligible Age

18 Years

Maximum Eligible Age

45 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Eithan Galun, MD

Role: PRINCIPAL_INVESTIGATOR

Protalix Ltd.

Countries

Review the countries where the study has at least one active or historical site.

Israel

References

Explore related publications, articles, or registry entries linked to this study.

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.

Reference Type DERIVED

PMID: 19277123 (View on PubMed)

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

P-01-2005

Identifier Type: -

Identifier Source: org_study_id