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

NCT00258778 | Completed Phase 1

• 1 other identifier: P-01-2005
• Study Type: interventional
• Target: 6
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Gaucher Disease

Outcome Measures

Primary Outcomes (5)

• Safety as measured by:

• adverse events

• change in vital signs

• physical examination

• laboratory test results

Secondary Outcomes (2)

• Pharmacokinetic parameters

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

Interventions

Human Glucocerebrosidase (prGCD) DRUG

Eligibility Criteria

• Age: 18 Years - 45 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Protalix: lead

Related Publications (1)

• 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.

  PMID: 19277123 DERIVED

MeSH Terms

Conditions

Gaucher Disease

Interventions

taliglucerase alfa

Condition Hierarchy (Ancestors)

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; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Officials

• Eithan Galun, MD

  Protalix Ltd.

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY

Study Record Dates

• First Submitted: November 23, 2005
• First Posted: November 28, 2005
• Study Start: November 1, 2005
• Study Completion: January 1, 2006
• Last Updated: December 5, 2006

Record last verified: 2006-12