A double-blind, randomised efficacy and safety study of taliglucerase alfa enzyme replacement therapy in children and adolescents with Gaucher disease (non-neuronopathic and chronic neuronopathic)
- Conditions
- Gaucher diseaseMedDRA version: 14.1Level: PTClassification code 10018048Term: Gaucher's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Registration Number
- EUCTR2007-000498-42-Outside-EU/EEA
- Lead Sponsor
- Protalix Biotherapeutics
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- A
- Sex
- All
- Target Recruitment
- 10
•Males and females 2 to <18 years old.
•Diagnosis of Gaucher disease with leukocyte acid ß-glucosidase activity =30% of the mean of the reference range for healthy subjects.
•Subjects who have not received enzyme replacement therapy (ERT) in the past or who have not received ERT in the past 12 months and have a negative anti-glucocerebrosidase antibody assay.
•Subjects who have not received substrate reduction therapy (SRT) in the past 12 months.
•Subjects whose clinical condition, in the opinion of the investigator, requires treatment with enzyme replacement therapy (ERT).
Are the trial subjects under 18? yes
Number of subjects for this age range: 10
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
•Currently taking another investigational drug for any condition.
•Presence of neurological signs and symptoms characteristic of Gaucher disease with complex neuronopathic features other than longstanding oculomotor gaze palsy.
•Presence of unresolved anemia due to iron, folic acid, or vitamin B12 deficiency
•Previous hypersensitivity reaction to Cerezyme® (imiglucerase) or Ceredase® (alglucerase).
•History of allergy to carrots.
•Presence of HIV, HBsAg or hepatitis C infections.
•Subject's parent(s) or legal guardian(s) are unable to understand the nature, scope and possible consequences of the study.
•Presence of any 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.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate the safety and efficacy of taliglucerase alfa in untreated children;Secondary Objective: N/A;Primary end point(s): median percentage and the interquartile range for change from baseline in haemoglobin<br><br><br><br>;Timepoint(s) of evaluation of this end point: Every 3 months for 12 months
- Secondary Outcome Measures
Name Time Method Secondary end point(s): Percent change from baseline in chitotriosidase or CCL18<br><br>Percent change in spleen and liver volume measured by MRI (or ultrasound)<br><br>Percent change from baseline in platelet count<br><br>Occurrence of positive antibody response<br><br><br><br><br>;Timepoint(s) of evaluation of this end point: •Chitotriosidase or CCL18 [ Time Frame: Every 3 months for 12 months ] •Spleen and liver volume [ Time Frame: Baseline and Month 12 ] <br>•Platelet count [ Time Frame: Every 3 months for 12 months ] <br>•Anti-taliglucerase alfa antibodies [ Time Frame: Every 3 months for 12 months ]