Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase - Tabular View

Tracking Information

First Received Date ^ICMJE

July 7, 2008

Last Updated Date

August 19, 2009

Start Date ^ICMJE

December 2008

Estimated Primary Completion Date

February 2010 (final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

Adverse events [ Time Frame: Continuous ] [ Designated as safety issue: Yes ]

Original Primary Outcome Measures ^ICMJE

Same as current

Change History

Complete list of historical versions of study NCT00712348 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

Platelet count [ Time Frame: Monthly ] [ Designated as safety issue: Yes ]

Original Secondary Outcome Measures ^ICMJE

Same as current

Descriptive Information

Brief Title ^ICMJE

Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase

Official Title ^ICMJE

A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase in Patients With Gaucher Disease Treated With Imiglucerase

Brief Summary

This is a multi-center, open-label, switchover trial to assess the safety of prGCD in 15 patients with Gaucher disease who are currently being treated with imiglucerase (Cerezyme®) ERT.

Detailed Description

This is a multi-center, open-label, switchover trial to assess the safety of prGCD in 15 patients with Gaucher disease who are currently being treated with imiglucerase (Cerezyme®) ERT. Eligible patients will enter a 12-week Stability Evaluation Period to establish the stability of their disease. Patients with stable disease will then be switched from their imiglucerase treatment to receive intravenous (IV) infusions of prGCD every two weeks for a total of 20 IV infusions. The dose of prGCD will be equal to each patient's previous imiglucerase dose. The infusions will be administered at the selected investigational site (clinic/hospital) At the end of the 9-month treatment period (20 visits, 38 weeks) eligible patients will be offered enrollment in an open-label extension study.

Study Phase

Phase III

Study Type ^ICMJE

Interventional

Study Design ^ICMJE

Treatment, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study

Condition ^ICMJE

Gaucher Disease

Intervention ^ICMJE

Drug: plant expressed recombinant human glucocerebrosidase (prGCD)

Study Arms / Comparison Groups

Experimental: Open label prGCD treatment

Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Recruiting

Estimated Enrollment ^ICMJE

Estimated Completion Date

February 2010

Estimated Primary Completion Date

February 2010 (final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

Inclusion Criteria:

Males and females, 18 years or older
Female patients of child-bearing potential and male patients with female partners of child-bearing potential must agree to use a medically acceptable method of contraception, not including the rhythm method
Confirmed diagnosis of Gaucher disease by the enzymatic activity assay
Stable Gaucher disease
Treatment with imiglucerase (Cerezyme®) for at least 2 years and on a stable maintenance regimen (dose unchanged) for at least the last six months
Able to provide written informed consent

Exclusion Criteria:

Currently taking another experimental drug for any condition
Pregnant or nursing or planning to become pregnant
History of allergy to carrots
Presence of anti-glucocerebrosidase (GCD) antibodies
Previous infusion reaction suspected to be allergic in nature to Cerezyme® or Ceredase® or receiving premedication to prevent infusion reactions
Presence of HIV and/or HBsAg and/or hepatitis C infection
Presence of unresolved anemia due to iron, folic acid or vitamin B12 deficiency
Presence of any significant comorbidity that could confound the interpretation of the clinical response to prGCD
Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the patient's compliance with the requirements of the study

Gender

Both

Ages

18 Years and older

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact: Raul Chertkoff, MD

+972-4-9889488

raul@protalix.com

Location Countries ^ICMJE

United States, Israel

Administrative Information

NCT ID ^ICMJE

NCT00712348

Responsible Party

Einat Almon, Protalix Biotherapeutics

Study ID Numbers ^ICMJE

PB-06-002

Study Sponsor ^ICMJE

Protalix

Collaborators ^ICMJE

Investigators ^ICMJE

Principal Investigator:

Ari Zimran, MD

Shaare Zedek Medical Center, Jerusalem

Information Provided By

Protalix

Verification Date

August 2009

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP