Phase I Single Dose-Escalation Safety Study of Human Gluc... | NCT00258778

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

NCT00258778

Sponsor

Protalix

Status

Completed

Last Update Posted

Dec 5, 2006Estimated

Enrollment

Phase

Phase 1

Conditions

Gaucher Disease

Interventions

Human Glucocerebrosidase (prGCD)

Countries

Not provided

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Dec 2006

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Not provided

Start Date

Nov 2005

Primary Completion Date

Not provided

Completion Date

Jan 2006

First Submitted Date

Nov 23, 2005

First Submission Date that Met QC Criteria

Nov 23, 2005

First Posted Date

Nov 28, 2005Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Dec 4, 2006

Last Update Posted Date

Dec 5, 2006Estimated

Description Module

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.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

Name	Type	Description	Arm Group Labels	Other Names
Human Glucocerebrosidase (prGCD)	Drug

Eligibility Module

Eligibility 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.

Accepts Healthy Volunteers

Yes

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

45 Years

Standard Ages

Adult

Study Population

Not provided

Sampling Method

Not provided

Name	Affiliation	Role
Eithan Galun, MD	Protalix Ltd.	Principal Investigator

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
19277123	Derived	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.

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

Measure	Description	Time Frame
Pharmacokinetic parameters
Immunological profile including: IgE, anti human prGCD antibodies, eosinophils and proteinuria

ID	Term
D013106	Sphingolipidoses
D020140	Lysosomal Storage Diseases, Nervous System
D020739	Brain Diseases, Metabolic, Inborn
D001928	Brain Diseases, Metabolic