Status:
COMPLETED
Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease
Lead Sponsor:
Shire
Collaborating Sponsors:
Quintiles, Inc.
Conditions:
Gaucher Disease
Eligibility:
All Genders
2+ years
Phase:
PHASE3
Brief 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 orga...
Detailed Description
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 orga...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
March 13 2013
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 8 2014
Estimated Enrollment :
5 Patients enrolled
Trial Details
Trial ID
NCT01842841
Start Date
March 13 2013
End Date
October 8 2014
Last Update
June 14 2021
Active Locations (5)
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1
Hamamatsu University School of Medicine
Hamamatsu, Shizuoka, Japan, 431-3192
2
Iwata City Hospital
Ōkubo, Shizuoka, Japan, 438-8550
3
The Jikei University School of Medicine
Minato-ku, Tokyo, Japan, 105-8471
4
Chiba Children's Hospital
Chiba, Japan, 266-0007