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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Shire Human Genetic Therapies, Inc.
ClinicalTrials.gov Identifier:
NCT00391625
First received: October 20, 2006
Last updated: February 6, 2012
Last verified: February 2012
  Purpose

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, Type 1
Drug: GA-GCB
Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
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 Human Genetic Therapies, Inc.:

Primary Outcome Measures:
  • Evaluation of long term safety [ Time Frame: duration of study ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Evaluation of hematological parameters and organomegaly [ Time Frame: Duration of study ] [ Designated as safety issue: Yes ]

Enrollment: 9
Study Start Date: April 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 Name: 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 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

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
Please refer to this study by its ClinicalTrials.gov identifier: NCT00391625

Locations
Israel
Shaare Zedek Medical Center
Jerusalem, Israel
Serbia
Mother and Child Health Care Institute of Serbia
Belgrade, Serbia
Sponsors and Collaborators
Shire Human Genetic Therapies, Inc.
Investigators
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

No publications provided by Shire Human Genetic Therapies, Inc.

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Shire Human Genetic Therapies, Inc.
ClinicalTrials.gov Identifier: NCT00391625     History of Changes
Other Study ID Numbers: TKT025EXT
Study First Received: October 20, 2006
Last Updated: February 6, 2012
Health Authority: United States: Food and Drug Administration
Israel: Israeli Health Ministry Pharmaceutical Administration
Romania: State Institute for Drug Control
Serbia and Montenegro: Agency for Drugs and Medicinal Devices

Keywords provided by Shire Human Genetic Therapies, Inc.:
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
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders
Sphingolipidoses
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors

ClinicalTrials.gov processed this record on April 22, 2014