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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.

Register:	EUCTR
Last refreshed on:	25 November 2019
Main ID:	EUCTR2007-000498-42-GB
Date of registration:	07/01/2008
Prospective Registration:	Yes
Primary sponsor:	Protalix Biotherapeutics
Public title:	A Phase III Multicenter, Randomized, Double-Blind Trial to Assess the Safety and Efficacy of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease - NA
Scientific title:	A Phase III Multicenter, Randomized, Double-Blind Trial to Assess the Safety and Efficacy of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease - NA
Date of first enrolment:	08/05/2008
Target sample size:	30
Recruitment status:	Not Recruiting
URL:	https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2007-000498-42
Study type:	Interventional clinical trial of medicinal product
Study design:	Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: yes Other specify the comparator: two different dose were of the same IMP are tested in this study
Phase:	Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment
Italy	Spain	United Kingdom

Contacts

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Key inclusion & exclusion criteria

Inclusion criteria:
- Males and females, 18 years or older.
- Female patients of child-bearing potential or male patients with female partners of child-bearing potential must agree to use two methods of contraception, one of which must be a barrier method. Acceptable methods of contraception include hormonal products, intrauterine device, or male or female condoms.
- Diagnosis of Gaucher disease with leukocyte GCD activity level =3 nmol/mg*hr (=30 % of the mean activity of the reference range)
- Splenomegaly defined as greater than eight times the expected volume [measured volume divided by estimated volume (0.2% of body weight)] as determined by MRI volumetric analysis
- Thrombocytopenia (defined as platelet counts <120,000 per mm3) with or without anemia (defined by hemoglobin at least 1 g/dL below normal range according to sex and age).
? Patients who have not received ERT in the past or patients who have not received ERT in the past 12 months and have a negative anti-glucocerebrosidase antibody
? Patients who have not received substrate reduction therapy (SRT) in the past 12 months.
? Ability to provide a written informed consent.

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
- Currently taking another experimental drug for any condition
- Presence of severe neurological signs and symptoms, defined as complete ocular paralysis, overt myoclonus or history of seizures, characteristic of neuronopathic Gaucher disease
- Previous anaphylactoid reaction to Cerezyme® or Ceredase®
- History of allergy to carrots
- Pregnant or nursing
- Presence of HIV and/or, HBsAg and/or hepatitis C infections
- 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.

Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Gaucher disease
MedDRA version: 9.1 Level: PT Classification code 10018048 Term: Gaucher's disease

Intervention(s)

Product Name: recombinant human glucocerebrosidase
Product Code: prGCD
Pharmaceutical Form: Powder for solution for infusion
Current Sponsor code: prGCD
Other descriptive name: recombinant human glucocerebrosidase
Concentration unit: U unit(s)
Concentration type: equal
Concentration number: 200-

Product Name: recombinant human glucocerebrosidase
Product Code: prGCD
Pharmaceutical Form: Powder for solution for infusion
Current Sponsor code: prGCD
Other descriptive name: recombinant human glucocerebrosidase
Concentration unit: U unit(s)
Concentration type: equal
Concentration number: 200-

Primary Outcome(s)

Primary end point(s): Primary efficacy endpoint:
Percent change from baseline in spleen volume measured by MRI at 9 months

Secondary Objective: Change from baseline in:
- Liver volume
- Platelet count
- Hemoglobin

other secondary analysis parameter:
-Biomarkers (chitotriosidase and pulmonary and activation-regulated chemokine (PARC/CCL18)
- Proportion of patients with greater than 10% reduction in spleen volume at 9 months

Main Objective: The change in spleen volume (in procent) from baseline at 9 months measured by MRI.

Secondary Outcome(s)

Secondary ID(s)

PB-06-001

Source(s) of Monetary Support

Secondary Sponsor(s)

Ethics review

Status: Approved
Approval date:
Contact:

Results

Results available:
Date Posted:
Date Completed:
URL:

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