At a glance
ClinicalIndex Comparison RecordPhase 2Completed· 10 enrolled
Drug / intervention
GA-GCBdrug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
An Open-Label Extension of Study TKT025 Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A Enzyme Replacement Therapy
In Brief
A Phase 2 clinical trial evaluating GA-GCB for Gaucher Disease. Completed, enrolled 10 participants across 3 sites in 3 countries.
Detailed 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 macrophages leading to cellular engorgement, organomegaly, and organ system dysfunction. The purpose of this study is to evaluate the long term safety of enzyme replacement therapy with DRX008A (VPRIV®, GA-GCB; velaglucerase alfa) in patients with type 1 Gaucher disease.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsGaucher Disease
CountriesIsrael, Romania, Serbia
Collaborators--
Timeline
Phase 2CompletedFinished
20052006200720082009201020112012201320142015201620172018201920202021202220232024202520262027
Enrollment StartSep 2004
First PostedOct 2006
Primary CompletionJan 2008
TodayJul 2026
First PostedOct 24, 2006
Enrollment StartSep 13, 2004
Primary CompletionJan 31, 2008
TodayJul 2, 2026
Enrollment to primary: 3.4 yearsPosted 19.7 years ago
Interventions
GA-GCBdrug
15-60 U/kg every other week via intravenous infusion