Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease

• Register: EUCTR
• Last refreshed on: 11 April 2016
• Main ID: EUCTR2012-003427-38-Outside-EU/EEA
• Date of registration: 07/04/2016
• Prospective Registration: Yes
• Primary sponsor: Shire Human Genetic Therapies
• Public title: Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease
• Scientific title: A Multi-center, Open-label, Efficacy and Safety Study of Velaglucerase Alfa Enzyme Replacement Therapy in Children and Adolescents With Type 3 Gaucher Disease
• Target sample size: 6
• Recruitment status: NA
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2012-003427-38
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes 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

Countries of recruitment

Egypt, India, Tunisia

Contacts

• Name: Med Info EU/CEEMA
• Affiliation: Shire Human Genetic Therapies

• Name: Med Info EU/CEEMA
• Affiliation: Shire Human Genetic Therapies

Key inclusion & exclusion criteria

Inclusion criteria:
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

Exclusion criteria:
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.


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

Health Condition(s) or Problem(s) studied

Type 3 Gaucher disease
MedDRA version: 18.1 Level: PT Classification code 10075697 Term: Gaucher's disease type I System Organ Class: 10010331 - Congenital, familial and genetic disorders

Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Intervention(s)

Trade Name: VPRIV
Product Name: Gene-Activated Human Glucocerebrosidase 400U/vial
Product Code: GA-GCB
Pharmaceutical Form: Powder for solution for infusion
INN or Proposed INN: velaglucerase alfa
CAS Number: 884604-91-5
Current Sponsor code: GA-GCB
Other descriptive name: VELAGLUCERASE ALFA
Concentration unit: IU/ml international unit(s)/millilitre
Concentration type: equal
Concentration number: 100-

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: Baseline, Week 53/end of study

Secondary Objective: Not applicable

Main Objective: To explore the efficacy and safety of velaglucerase alfa enzyme replacement therapy (ERT) in children and adolescents with type 3 Gaucher disease.

Primary end point(s): Change From Baseline to 12 Months in Hemoglobin Concentration

Secondary Outcome(s)

Secondary end point(s): 1-Change From Baseline to 12 Months in Platelet Count
2-Change From Baseline to 12 Months in Normalized Liver And Spleen Volumes Measured Using Magnetic Resonance Imaging (MRI)
3-Change From Baseline to 12 Months in Neurological Symptoms
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
2- Baseline, Week 51
3-Baseline, Week 53
4-From ICF through to 30 day follow-up visit, including Baseline, Weeks 13, 25, 37, and 53

Secondary ID(s)

NCT01685216

HGT-GCB-068

Source(s) of Monetary Support

Shire Human Genetic Therapies