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

Sponsor:	Protalix
Information provided by:	Protalix
ClinicalTrials.gov Identifier:	NCT00258778

Purpose

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	Intervention	Phase
Gaucher Disease	Drug: Human Glucocerebrosidase (prGCD)	Phase I

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized Endpoint Classification: Safety Study Intervention Model: Single Group Assignment Masking: 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

Resource links provided by NLM:

Genetics Home Reference related topics: Chanarin-Dorfman syndrome cholesteryl ester storage disease Farber lipogranulomatosis Gaucher disease Schindler disease succinic semialdehyde dehydrogenase deficiency

MedlinePlus related topics: Gaucher's Disease

Drug Information available for: Alglucerase Imiglucerase

U.S. FDA Resources

Further study details as provided by Protalix:

Primary Outcome Measures:

Safety as measured by:
adverse events
change in vital signs
physical examination
laboratory test results

Secondary Outcome Measures:

Pharmacokinetic parameters
Immunological profile including: IgE, anti human prGCD antibodies, eosinophils and proteinuria

Estimated Enrollment:	6
Study Start Date:	November 2005
Estimated Study Completion Date:	January 2006

Eligibility

Ages Eligible for Study:	18 Years to 45 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	Yes

Criteria

Inclusion Criteria:

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

Exclusion Criteria:

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.
Are pregnant or nursing.
Presence of any acute or chronic diseases.
Have a history of any allergies.
Have been exposed to long-term steroid treatment.
Had a minor operation in the last 6 months.
Have ever been exposed to any previous recombinant protein therapy.
Have received immuno-suppressive treatment.
Have a positive HIV, HBsAG and HCV laboratory result.
Use any medication other than vitamins or oral contraceptives (for female).
Have participated in another clinical trial during the previous 3 months
Have history of alcohol or drug abuse.
Are considered by the Investigator to be unsuitable candidate for this study.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00258778

Sponsors and Collaborators

Protalix

Investigators

Principal Investigator:

Eithan Galun, MD

Protalix Ltd.

More Information

No publications provided by Protalix

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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. Epub 2009 Mar 11.

ClinicalTrials.gov Identifier:	NCT00258778 History of Changes
Other Study ID Numbers:	P-01-2005
Study First Received:	November 23, 2005
Last Updated:	December 4, 2006
Health Authority:	United States: Food and Drug Administration

Additional relevant MeSH terms:

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

ClinicalTrials.gov processed this record on February 09, 2012