Clinical Trial: Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: An Open-Label Extension of Study TKT025 Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A Enzyme Replacement Therapy

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 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.

Detailed Summary: 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 hepatomegaly, splenomegaly, thrombocytopenia, bleeding tendencies, anemia, hypermetabolism, skeletal pathology, growth retardation, pulmonary disease, and decreased quality of life. Gene-Activated® human glucocerebrosidase (the long term safety of enzyme replacement therapy with DRX008A (GA-GCB; velaglucerase alfa) is produced in a continuous human cell line using proprietary gene-activation technology and has an identical amino acid sequence to the naturally occurring human enzyme. GA-GCB (velaglucerase alfa) contains terminal mannose residues that target the enzyme to the macrophages-the primary target cells in Gaucher disease. This study was designed to evaluate the long term safety of GA-GCB (velaglucerase alfa) in patients with Type 1 Gaucher disease
Sponsor: Shire

Current Primary Outcome: Evaluation of Long Term Safety [ Time Frame: Up to 84 months ]

Overall Summary of Treatment-emergent Adverse Events-Safety Population


Original Primary Outcome:

Current Secondary Outcome:

  • Percent Change From Baseline in Hemoglobin Concentration [ Time Frame: Baseline, then every 12 months ]
  • Percent Change From Baseline in Platelet Counts [ Time Frame: Baseline, then every 12 months ]
  • Percent Change From Baseline in Liver Volume [ Time Frame: Baseline, Month 24, then every 9 or 12 months ]
  • Percent Change From Baseline in Spleen Size [ Time Frame: Baseline, Month 24, then every 9 or 12 months ]


Original Secondary Outcome:

Information By: Shire

Dates:
Date Received: October 20, 2006
Date Started: February 2005
Date Completion:
Last Updated: May 13, 2016
Last Verified: August 2015