Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)
Information source: Shire Human Genetic Therapies, Inc.
Information obtained from ClinicalTrials.gov on June 20, 2008
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Gaucher Disease, Type 1
Intervention: Gene-Activated® human glucocerebrosidase (GA-GCB) (Biological)
Phase: Phase 1/Phase 2
Status: Active, not recruiting
Sponsored by: Shire Human Genetic Therapies, Inc. Official(s) and/or principal investigator(s):
Ari Zimran, M.D., Principal Investigator, Affiliation: Gaucher Clinic, Shaare Zedek Medical Center
Florea Iordachescu, MD, Principal Investigator, Affiliation: Maria Sklodowska Curie Children's Hospital
Maja Djordjevic, M.D., Principal Investigator, Affiliation: Mother and Child Health Care Institute of Serbia
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 (GA-GCB; velaglucerase alfa) in patients with type 1 Gaucher
disease.
Clinical Details
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 design: Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Primary outcome: Evaluation of safety assessments
Secondary outcome: Evaluation of hematological parameters and organomegaly
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 (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
Minimum age: 18 Years.
Maximum age: N/A.
Gender(s): Both.
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 an investigational therapy within the past 30 days
other than GA-GCB.
- 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.
Locations and Contacts
Shaare Zedek Medical Center, Jerusalem, Israel
Maria Sklodwska Curie Children's Hospital, Bucharest 75544, Romania
Mother and Child Health Care Institute of Serbia, Belgrade, Serbia and Montenegro
Additional Information
Starting date: April 2005
Ending date: April 2009
Last updated: March 6, 2008
|
