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

Sponsored by:	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

Genetics Home Reference related topics: cholesteryl ester storage disease Farber lipogranulomatosis Gaucher disease long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency mitochondrial trifunctional protein deficiency primary carnitine deficiency

MedlinePlus related topics: Gaucher's Disease

Drug Information available for: Alglucerase Imiglucerase

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Dose Comparison, Single Group Assignment, Safety Study
Official Title:	A Phase I, Non-Randomized, Open Label, Single Dose-Escalation Safety Study of Recombinant Human Glucocerebrosidase (prGCD) in Healthy Volunteers

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

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

Study placed in the following topic categories:

Lipid Metabolism, Inborn Errors
Sphingolipidoses
Metabolic Diseases
Lysosomal Storage Diseases
Sphingolipidosis
Central Nervous System Diseases
Healthy
Brain Diseases
Lymphatic Diseases

Metabolism, Inborn Errors
Genetic Diseases, Inborn
Brain Diseases, Metabolic, Inborn
Lipidoses
Metabolic disorder
Gaucher Disease
Lipid Metabolism Disorders
Brain Diseases, Metabolic

Additional relevant MeSH terms:

Reticuloendotheliosis
Lysosomal Storage Diseases, Nervous System
Nervous System Diseases

ClinicalTrials.gov processed this record on January 14, 2009