Skip to main content
OpenTrials
Completed

NCT Number: NCT00258778

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

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Gaucher Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Sphingolipidoses

Age range

18 year–45 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Human Glucocerebrosidase (prGCD)

Drug

Primary outcomes

  1. Safety as measured by:

  2. adverse events

  3. change in vital signs

  4. physical examination

  5. laboratory test results

Secondary outcomes

  1. Pharmacokinetic parameters

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

Sponsors and collaborators

Lead sponsor

Protalix

Industry

Registry information

Official study title

A Phase I, Non-Randomized, Open Label, Single Dose-Escalation Safety Study of Recombinant Human Glucocerebrosidase (prGCD) in Healthy Volunteers

Important dates

Study start
2005
Study completion
2006
First posted
Nov 28, 2005
Registry last updated
Dec 5, 2006

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.