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Current Gaucher Disease Research and Clinical TrialsTo better help our readers understand what is involved with clinical trials, click here for more information. What is a clinical trial? Although there are many definitions of clinical trials, they are generally considered to be biomedical or health-related research studies in human beings that follow a pre-defined protocol. Amicus Therapeutics, Inc. Cedars-Sinai Medical Center Genzyme Therapeutics Study: "A Study of the Efficacy and Safety of Genz-112638 in Type I Gaucher patients". This study is ongoing, but, not recruiting participants. For information: http://www.clinicaltrials.gov/ NCT00358150 Study: "International Collaborative Gaucher Group (ICGG) Gaucher Registry". This study is currently recruiting participants. For information: http://www.clinicaltrials.gov/ NCT00358943 Massachusetts General Hospital National Institutes of Health National Institutes of Neurological Disorders and Stroke (NIND) New York University School of Medicine The New York University School of Medicine, Division of Neurogenetics, has been selected to participate in a worldwide clinical study to test a new oral medication for the treatment of patients with type 1 Gaucher disease. The investigational drug is called Genz-112638 and it has been previously tested in three Phase 1 studies in normal volunteers. This Phase 2 study is designed to assess the serum concentrations of Genz-112638 in patients with type 1 Gaucher disease and to determine whether the drug shows to be safe and effective treatment for patients with the disease. As physicians with expertise in treating patients with Gaucher disease, we would like to inform you that we are currently enrolling patients in this study. As you know, the current standard of care for patients with type 1 Gaucher disease is enzyme replacement therapy (ERT) with recombinant human glucocerebrosidase (CerezymeÒ [imiglucerase for injection)). Cerezyme is usually administered intravenously, every two weeks. Genz-112638 is a new oral medication that functions by decreasing the synthesis of glucosylceramide, the substrate that accumulates in the lysosomes of patients with Gaucher disease. Male and female patients with Gaucher type 1 disease who are between the ages of 18 and 65 years old and who weigh between 50 and 120 kg may be eligible to participate in the study, if they also meet the following criteria: 1. Hemoglobin levels of 8.0 to 10.0 g/dL if female or 8.0 to 11.0 g/dL if male, OR a platelet count of 60,000 to 100,000/mm3, if either gender. 2. Splenomegaly, by MRI or spiral computed tomography (CT), defined as a spleen volume ³ 10 x normal. The patients who have had a partial or total splenectomy, who received miglustat at any time prior to enrollment, or who have received enzyme replacement therapy (ERT) in the past 12 months will not be eligible for the screening. The study drug is an oral capsule of Genz-112638 taken twice daily. The sponsor of the trial, Genzyme, will cover the cost of all assessments and the cost of the study drug. In addition, Genzyme will provide reimbursement for all study-related travel, including any necessary hotel stays, airfare, taxi fare, or train rides as well as compensation for participation in the study. For more information please contact the primary investigator at NYU, Dr. Maryam Banikazemi at 212 263 8344 or maryam.banikazemi@med.nyu.edu Name of Study: A questionnaire based computerized review of clinical, sociologic and psychological status of 123 patients with Gaucher disease. Shaare Zedek Medical Center, Israel Shire Human Genetic Therapies, Inc. Recruiting A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease Condition: Gaucher Disease, Type 1 Interventions: Biological: GA-GCB; Biological: Cerezyme Recruiting Multicenter Open-Label Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase Condition: Gaucher Disease, Type 1 Intervention: Drug: Gene-Activated® human glucocerebrosidase (GA-GCB) Recruiting A Study of GA-GCB Enzyme Replacement Therapy in Gaucher Disease Condition: Gaucher Disease,Type 1 Intervention: Drug: Gene-Activated® human glucocerebrosidase (GA-GCB) Active, not recruiting Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT) Condition: Gaucher Disease, Type 1 Intervention: Drug: Gene-Activated® human glucocerebrosidase (GA-GCB) An Emerging New Treatment for Type 1 Gaucher Disease: Results of a 9-Month Phase I/II study Shire Human Genetic Therapies, Inc. (Shire HGT) has recently completed a 9-month Phase I/II trial with Gene-Activated® human glucocerebrosidase (GA-GCB), an investigational new product, at the Gaucher Clinic at Shaare Zedek Medical Center. GA-GCB is human glucocerebrosidase which is produced in a well-characterized, continuous human cell line using proprietary Gene-Activated® technology. Unlike Cerezyme®, GA-GCB has an identical amino acid sequence to the naturally occurring human enzyme, which may be an advantage. This innovative technology has been used to develop an alternative enzyme replacement therapeutic option for patients with Gaucher disease. The purpose of the Phase I/II study was to assess the safety and efficacy of GA-GCB in adult patients with type 1 Gaucher disease. Twelve patients received GA-GCB every other week for 9 months. Of 12 patients treated, 11 patients completed the study. There were no drug-related serious adverse events and no patient discontinued participation because of adverse events. Infusion-related reactions were limited, mild and transient. None of the treated patients developed anti-GA-GCB antibodies. The results with GA-GCB were very impressive. Notably, the GA-GCB study did not allow children to enroll, who typically respond better to treatment, yet similar results were obtained to the seminal trial of Ceredase®, where 8 out of 12 patients were younger than 18 years of age. In the GA-GCB trial, there were significant increases in hemoglobin (on average: 2.24g/dL; mean increase of 19.2% from baseline) and in platelet counts (on average: 40,600/mm3; mean increase of 67.6% from baseline) and also significant decreases in spleen and liver volume (by 49.5% and 18.2% from baseline, respectively). Similarly, there were significant decreases in the biomarkers chitotriosidase (by 74.2%) and CCL18 (by 57.1%). Improvements in some parameters were apparent as early as three months after the start of the therapy. These results suggest that Gene-Activated® human glucocerebrosidase (GA-GCB), which has an identical amino acid sequence to the naturally occurring human enzyme, holds promise as a new enzyme replacement therapy for Gaucher disease and warrants further evaluation in clinical studies in a broader population of patients. Phase III studies with GA-GCB will begin late 2006. Yale University, New Haven, CT |
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