Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease
- Conditions
- Type 3 Gaucher diseaseMedDRA version: 18.1Level: PTClassification code 10075697Term: Gaucher's disease type ISystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2012-003427-38-Outside-EU/EEA
- Lead Sponsor
- Shire Human Genetic Therapies
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- A
- Sex
- All
- Target Recruitment
- 6
PRINCIPAL INCLUSION CRITERIA
1-The patient has a confirmed diagnosis of type 3 Gaucher disease.
2-The patient is = 2 and < 18 years of age at the time of enrollment.
3-The patient is either näive to treatment or has not received treatment (investigational or approved) for Gaucher disease within 12 months prior to study entry.
4-The patient has Gaucher disease-related anemia, defined as hemoglobin concentration below the lower limit of normal for age and sex.
AND ONE OR MORE OF THE FOLLOWING CRITERIA
The patient has at least moderate splenomegaly (2 to 3 cm below the left costal margin) by palpation.
The patient has Gaucher disease-related thrombocytopenia, defined as platelet count < 120 x 10,000 platelets/cubic mm.
The patient has a Gaucher disease-related readily palpable enlarged liver.
5-Patients who have undergone splenectomy may still be eligible to participate in the study.
6-Female patients of child-bearing potential must agree to use a medically acceptable method of contraception at all times during the study. Pregnancy testing will be performed at the time of enrollment and as required throughout participation in the study. Male patients must agree to use a medically acceptable method of contraception at all times during the study and report a partner's pregnancy to the Investigator.
7-The patient's parent(s) or the patient's legally authorized representative(s) has provided written informed consent that has been approved by the Institutional Review Board/Independent Ethics Committee (IRB/IEC).
Are the trial subjects under 18? yes
Number of subjects for this age range: 7
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range 0
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0
PRINCIPAL EXCLUSION CRITERIA
1-The patient is suspected of having type 2 or type 1 Gaucher disease.
2-The patient is < 2 years of age.
3-The patient has experienced a severe (Grade 3 or higher) infusion-related hypersensitivity reaction (anaphylactic or anaphylactoid reaction) to any enzyme replacement therapy for Gaucher disease (approved or investigational).
4-The patient has received any non-Gaucher disease-related treatment with an investigational drug within 30 days prior to study entry.
5-The patient is a pregnant and/or lactating female.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To explore the efficacy and safety of velaglucerase alfa enzyme replacement therapy (ERT) in children and adolescents with type 3 Gaucher disease.;Secondary Objective: Not applicable;Primary end point(s): Change From Baseline to 12 Months in Hemoglobin Concentration;Timepoint(s) of evaluation of this end point: Baseline, Week 53/end of study
- Secondary Outcome Measures
Name Time Method Secondary end point(s): 1-Change From Baseline to 12 Months in Platelet Count<br>2-Change From Baseline to 12 Months in Normalized Liver And Spleen Volumes Measured Using Magnetic Resonance Imaging (MRI)<br>3-Change From Baseline to 12 Months in Neurological Symptoms<br>4-Safety endpoints to include adverse events (AEs) and infusion-related AEs, serious adverse events (SAEs), clinical laboratory values, urinalysis, vital signs, 12-lead electrocardiogram (ECG) recordings, physical examination, and anti-velaglucerase alfa antibody formation;Timepoint(s) of evaluation of this end point: 1-Baseline, Week 53/end of study<br>2- Baseline, Week 51<br>3-Baseline, Week 53<br>4-From ICF through to 30 day follow-up visit, including Baseline, Weeks 13, 25, 37, and 53