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Trial record 18 of 31 for:    "Gaucher disease type 1"

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

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ClinicalTrials.gov Identifier: NCT00391625
Recruitment Status : Completed
First Posted : October 24, 2006
Results First Posted : June 23, 2014
Last Update Posted : June 17, 2016
Sponsor:
Information provided by (Responsible Party):
Shire

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.

Condition or disease Intervention/treatment Phase
Gaucher Disease Drug: GA-GCB Phase 1 Phase 2

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

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Intervention Model: Single Group Assignment
Masking: None (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
Study Start Date : February 2005
Actual Primary Completion Date : December 2011
Actual Study Completion Date : December 2011


Arm Intervention/treatment
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




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


Secondary Outcome Measures :
  1. Percent Change From Baseline in Hemoglobin Concentration [ Time Frame: Baseline, then every 12 months ]
  2. Percent Change From Baseline in Platelet Counts [ Time Frame: Baseline, then every 12 months ]
  3. Percent Change From Baseline in Liver Volume [ Time Frame: Baseline, Month 24, then every 9 or 12 months ]
  4. Percent Change From Baseline in Spleen Size [ Time Frame: Baseline, Month 24, then every 9 or 12 months ]


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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
Investigators
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

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT00391625     History of Changes
Other Study ID Numbers: TKT025EXT
First Posted: October 24, 2006    Key Record Dates
Results First Posted: June 23, 2014
Last Update Posted: June 17, 2016
Last Verified: August 2015

Keywords provided by Shire:
Gaucher disease, Enzyme Replacement Therapy

Additional relevant MeSH terms:
Gaucher Disease
Sphingolipidoses
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
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders