Status:
COMPLETED
Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)
Lead Sponsor:
Shire
Conditions:
Gaucher Disease
Eligibility:
All Genders
18+ years
Phase:
PHASE1
PHASE2
Brief Summary
Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside accumulates within macro...
Detailed Description
Type 1 Gaucher disease, the most common form, accounts for more than 90% of all cases and does not involve the central nervous system (CNS). Typical manifestations of type 1 Gaucher disease include he...
Eligibility Criteria
Inclusion
- Patients who have completed through Week 41 visit in the TKT025 study.
- Patients must have voluntarily signed an IRB/EC approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient.
- Patient must be sufficiently cooperative to participate in this clinical study as judged by the Investigator.
- Female and male patients of child bearing potential must agree to use a medically acceptable method of contraception at all times during the study. Female patients must have a negative serum pregnancy test on enrollment.
Exclusion
- Patient has received treatment with non-Gaucher disease related investigational drug or device within the past 30 days prior to study entry; such use during the study is not permitted.
- Patient has a clinically relevant medical condition (e.g., HIV, hepatitis B or C) that would make implementation of the protocol difficult and/or confound an assessment of the effects of the experimental therapy and its adverse events.
- Patient, patient's parent(s), or patient's legal guardian is unable to understand the nature, scope and possible consequences of the study.
- Patient is unable to comply with the protocol, e.g. uncooperative attitude, medical condition, inability to return for safety evaluations, or is otherwise unlikely to complete the study, as determined by the Investigator.
Key Trial Info
Start Date :
September 13 2004
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 31 2008
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT00391625
Start Date
September 13 2004
End Date
January 31 2008
Last Update
June 22 2021
Active Locations (3)
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1
Shaare Zedek Medical Center
Jerusalem, Israel
2
Maria Sklodowska Curie Children's Hospital
Bucharest, Romania, 75544
3
Mother and Child Health Care Institute of Serbia
Belgrade, Serbia