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: May 21, 2014
Last verified: May 2014
  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: 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: Up to 84 months ] [ Designated as safety issue: Yes ]
    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 ] [ Designated as safety issue: No ]
  • Percent Change From Baseline in Platelet Counts [ Time Frame: Baseline, then every 12 months ] [ Designated as safety issue: No ]
  • Percent Change From Baseline in Liver Volume [ Time Frame: Baseline, Month 24, then every 9 or 12 months ] [ Designated as safety issue: No ]
  • Percent Change From Baseline in Spleen Size [ Time Frame: Baseline, Month 24, then every 9 or 12 months ] [ Designated as safety issue: No ]

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 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 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

  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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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

Locations
Israel
Shaare Zedek Medical Center
Jerusalem, Israel
Romania
Maria Sklodowska Curie Children's Hospital
Bucharest, Romania, 75544
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
Principal Investigator: Florea Iordachescu, MD Maria Sklodowska Curie Children's Hospital
  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
Results First Received: March 4, 2014
Last Updated: May 21, 2014
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
Brain Diseases
Brain Diseases, Metabolic
Brain Diseases, Metabolic, Inborn
Central Nervous System Diseases
Genetic Diseases, Inborn
Lipid Metabolism Disorders
Lipid Metabolism, Inborn Errors
Lipidoses
Lysosomal Storage Diseases
Lysosomal Storage Diseases, Nervous System
Metabolic Diseases
Metabolism, Inborn Errors
Nervous System Diseases
Sphingolipidoses

ClinicalTrials.gov processed this record on October 29, 2014