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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT01685216
• Date of registration: 10/09/2012
• Prospective Registration: No
• Primary sponsor: Shire
• 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
• Date of first enrolment: September 2012
• Target sample size: 7
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01685216
• Study type: Interventional
• Phase: Phase 1/Phase 2

Countries of recruitment

Egypt, India, Tunisia

Contacts

• Name: Björn Mellgard, MD PhD
• Affiliation: Shire

Key inclusion & exclusion criteria

Inclusion Criteria: Each patient must meet the following criteria to be enrolled in this
study.

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 THREE 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).

Exclusion Criteria: Patients who meet any of the following criteria will be excluded from
this study.

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: 2 Years
Age maximum: 17 Years
Gender: All

Health Condition(s) or Problem(s) studied

Gaucher Disease, Type 3

Intervention(s)

Biological: velaglucerase alfa

Primary Outcome(s)

Change From Baseline to 12 Months (Week 53) in Hemoglobin Concentration [Time Frame: Baseline, Week 53 or end of study]

Secondary Outcome(s)

Number of Participants Who Experienced a Treatment-Emergent Adverse Event [Time Frame: 57 weeks]

Percent Change From Baseline to 12 Months (Week 51) in Normalized Liver Volume Measured Using Magnetic Resonance Imaging (MRI) [Time Frame: Baseline, Week 51 or end of study]

Number of Participants With Abnormal Neurological Status During The Study [Time Frame: Baseline, Weeks 13, 25, 37, and 53 or end of study]

Change From Baseline to 12 Months (Week 53) in Platelet Count [Time Frame: Baseline, Week 53]

Number of Participants Who Developed Anti-Velaglucerase Alfa Antibodies During The Study [Time Frame: Baseline, Weeks 13, 25, 37 and 53]

Percent Change From Baseline to 12 Months (Week 51) in Normalized Spleen Volume Measured Using Magnetic Resonance Imaging (MRI) [Time Frame: Baseline, Week 51]

Secondary ID(s)

HGT-GCB-068

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 30/10/2015
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01685216