NA

• Register: EUCTR
• Last refreshed on: 19 April 2022
• Main ID: EUCTR2011-003676-37-ES
• Date of registration: 14/09/2011
• Prospective Registration: Yes
• Primary sponsor: Protalix Biotherapeutics
• Public title: NA
• Scientific title: A Multicenter Extension Study of Taliglucerase alfa in Pediatric Subjects with Gaucher Disease - NA
• Date of first enrolment: 15/11/2011
• Target sample size: 15
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2011-003676-37
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: no; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Israel, South Africa, Spain

Contacts

• Name: Raul Chertkoff
• Affiliation: Protalix Biotherapeutics

• Name: Raul Chertkoff
• Affiliation: Protalix Biotherapeutics

Key inclusion & exclusion criteria

Inclusion criteria:
1. Successful completion of Protocol PB-06-002 or PB-06-005.
2. The subject, parent(s) or legal guardian(s) signs an informed consent and/or assent
Are the trial subjects under 18? yes
Number of subjects for this age range: 2
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. Subjects currently taking another investigational drug for any condition.
2. The presence of neurological signs and symptoms characteristic of Gaucher disease
with complex neuronopathic features other than longstanding oculomotor gaze palsy.
3. The presence of a medical, emotional, behavioral or psychological condition that, in
the judgment of the Investigator, would interfere with the subject?s compliance with
the requirements of the study.


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

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Gaucher disease
MedDRA version: 14.0 Level: PT Classification code 10018048 Term: Gaucher's disease System Organ Class: 10010331 - Congenital, familial and genetic disorders

Intervention(s)

Product Name: recombinant human glucerebrosidase
Product Code: Taliglucerase Alfa
Pharmaceutical Form: Powder for solution for infusion
INN or Proposed INN: Taliglucerase alfa
CAS Number: 37228-64-1
Current Sponsor code: Taliglucerase alfa
Other descriptive name: prGCD
Concentration unit: IU/ml international unit(s)/millilitre
Concentration type: up to
Concentration number: 40-

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: NA

Secondary Objective: NA

Main Objective: To extend the assessment of the safety and efficacy of taliglucerase alfa in pediatric subjects (2 to <18 years old) with symptoms and clinical manifestations of Gaucher disease who completed treatment in Protocols PB-06-002 (switchover study from imiglucerase) or PB-06-005 (naïve treatment with taliglucerase alfa).

Primary end point(s): 1- The following efficacy variables will be assessed:
- Spleen volume
- Liver volume
- Platelet count
- Hemoglobin
- Biomarkers (chitotriosidase and PARC/CCL18)

- Exploratory endpoints are:
o Change in height and weight
o Change in Tanner Stage
o Change in bone age by X-ray of left hand and wrist
o Change in bone density by DEXA
o Occurrence of bone crises
o Qualify of Life using the Child Health Questionnaire? (CHQ) PF-28

2- The safety of taliglucerase alfa will be assessed by the following:
- Clinical laboratory
- Echocardiography
- Antitaliglucerase alfa antibodies
- Adverse events.

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: NA

Secondary end point(s): NA

Secondary ID(s)

PB-06-006

NCT01411228

Source(s) of Monetary Support

Protalix Biotherapeutics

Ethics review

Status: Approved
Approval date: 28/09/2011
Contact:

Results

• Results available: Yes
• Date Posted: 09/07/2021
• Date Completed: 07/07/2013
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2011-003676-37/results