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

This study has been completed.
Information provided by (Responsible Party):
Shire Identifier:
First received: October 20, 2006
Last updated: May 13, 2016
Last verified: August 2015
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.

Condition Intervention Phase
Gaucher Disease
Drug: GA-GCB
Phase 1
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:

Further study details as provided by Shire:

Primary Outcome Measures:
  • Evaluation of Long Term Safety [ Time Frame: Up to 84 months ]
    Overall Summary of Treatment-emergent Adverse Events-Safety Population

Secondary Outcome Measures:
  • 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 ]

Enrollment: 10
Study Start Date: February 2005
Study Completion Date: December 2011
Primary Completion Date: December 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: GA-GCB
15-60 U/kg every other week via intravenous infusion
Drug: GA-GCB
15-60 U/kg every other week via intravenous infusion
Other Names:
  • VPRIV®
  • velaglucerase alfa
  • gene-activated glucocerebrosidase
  • DRX008

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 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

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • 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 Criteria:

  • 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.
  Contacts and Locations
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Please refer to this study by its identifier: NCT00391625

Shaare Zedek Medical Center
Jerusalem, Israel
Maria Sklodowska Curie Children's Hospital
Bucharest, Romania, 75544
Mother and Child Health Care Institute of Serbia
Belgrade, Serbia
Sponsors and Collaborators
Principal Investigator: Florea Iordachescu, MD Maria Sklodowska Curie Children's Hospital
Principal Investigator: Ari Zimran, M.D. Gaucher Clinic, Shaare Zedek Medical Center
Principal Investigator: Maja Djordjevic, M.D. Mother and Child Health Care Institute of Serbia
  More Information

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Shire Identifier: NCT00391625     History of Changes
Other Study ID Numbers: TKT025EXT
Study First Received: October 20, 2006
Results First Received: March 4, 2014
Last Updated: May 13, 2016

Keywords provided by Shire:
Gaucher disease, Enzyme Replacement Therapy

Additional relevant MeSH terms:
Gaucher Disease
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders processed this record on May 25, 2017