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

Plain-language summary

This study evaluates Velaglucerase Alfa enzyme replacement therapy in children and adolescents with Type 3 Gaucher disease. It included patients aged 2-18 with confirmed Type 3 Gaucher and Gaucher-related anemia. It was a Phase 1/Phase 2 study and has been completed.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

PHASE1/PHASE2

Status

Completed

Sponsor

Shire

Intervention

velaglucerase alfa

Start date

9/14/2012

Estimated completion

3/15/2015

Countries

Egypt, India, Tunisia

Related genes

Locations

• Alexandria, Egypt
• Cairo, Egypt
• Cairo, Egypt
• Pune, India
• Tunis, Tunisia

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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.