Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease

• Status: Completed
• Trial end date: 2014-10-08
• Target enrollment: 0
• Participant gender: All

Summary

Gaucher disease is an inherited deficiency of the lysosomal enzyme glucocerebrosidase (GCB) that leads to progressive accumulation of glucocerebroside within macrophages and subsequent tissue and organ damage; typically of the liver, spleen, bone marrow, and brain. Type 1 Gaucher disease affects an estimated 30,000 persons worldwide and is the most common. Type 1 Gaucher disease does not involve the central nervous system. Patients with Type 2 Gaucher disease present with acute neurological deterioration, which leads to early death. Those with Type 3 disease typically display a more sub-acute neurological course, with later onset and slower progression.

The primary objective of this study is to evaluate the long-term safety of every other week (EOW) dosing of velaglucerase alfa in Japanese patients with Gaucher disease who completed study HGT-GCB-087 and elected to continue treatment with velaglucerase alfa.

Velaglucerase alfa has been developed and approved as an enzyme replacement therapy for Type 1 Gaucher disease.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Shire
• Collaborator: Quintiles, Inc.

Criteria

Inclusion Criteria:

• The patient has completed treatment with EOW velaglucerase alfa through Week 51 of study HGT-GCB-087.

• Female patients of child bearing potential must agree to use a medically acceptable method of contraception at all times during the study.

• The patient, the patient's parent(s)or legal guardian(s) has provided written informed consent that has been approved by the Institutional Review Board/Independent Ethics Committee(IRB/IEC)

• The patient must be sufficiently cooperative to participate in this clinical study as judged by the Investigator.

Exclusion Criteria:

• The patient has received treatment with any investigational drug, other than velaglucerase alfa, or investigational device within 30 days prior to study entry; such use during the study is not permitted.

• The patient, patient's parent(s), or patient's legal guardian(s) is/are unable to understand the nature, scope, and possible consequences of the study.

• The patient has a significant comorbidity, as determined by the Investigator that might affect study data or confound the study results.

• The patient is unable to comply with the protocol as determined by the Investigator.