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A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.
This study has been completed.
First Received: August 15, 2006   Last Updated: July 16, 2007   History of Changes
Sponsored by: Genzyme
Information provided by: Genzyme
ClinicalTrials.gov Identifier: NCT00365131
  Purpose

This is a multicenter, open-label, prospective study of the efficacy of Cerezyme in treating patients with skeletal manifestations secondary to Type I Gaucher disease. The study objective is to evaluate and quantify skeletal responses as compared to baseline in Type I gaucher disease patients receiving Cerezyme therapy for 48 months. Additional objectives were to assess the usefulness of various skeletal parameters, such as bone pain, bone crises, bone mineral density, and serum and urine bone markers, as indicative of treatment response and may be useful in dose management.


Condition Intervention Phase
Gaucher Disease Type I
Cerebroside Lipidosis Syndrome
Clucocerebrosidase Deficiency Disease
Glucosylceramide Beta-Glucosidase Deficiency Disease
Gaucher Disease, Non-Neuronopathic Form
Drug: Cerezyme (imiglucerase for injection)
Phase IV

Genetics Home Reference related topics: Chanarin-Dorfman syndrome cholesteryl ester storage disease Farber lipogranulomatosis Gaucher disease long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency mitochondrial trifunctional protein deficiency primary carnitine deficiency succinic semialdehyde dehydrogenase deficiency
MedlinePlus related topics: Gaucher's Disease
Drug Information available for: Alglucerase Imiglucerase
U.S. FDA Resources
Study Type: Interventional
Study Design: Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Efficacy Study

Further study details as provided by Genzyme:

Primary Outcome Measures:
  • Skeletal response over 4 years of Cerezyme therapy
  • Assess use of skeletal parameter as indicative of treatment response and use in dose management

Estimated Enrollment: 40
Study Start Date: December 1997
  Eligibility

Ages Eligible for Study:   10 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Signed informed consent.
  • Confirmed diagnosis of Type I Gaucher disease, with no prior enzyme replacement therapy, gene therapy or bone marrow transplantation, and who are ambulatory.
  • Age 10-65 (patients 66-70 years of age are considered on a case-by-case basis following careful medical review).
  • Dual energy X-ray absorptiometry (DEXA) of the femoral nech with a T-score ≤ -1.0.
  • One of more of the following signs as documented by X-ray, computed tomography (CT), or magnetic resonance imaging (MRI), or symptoms of bone disease as documented in the patient’s medical history or baseline examinations: a). history of at least one bone crises; b). Erlenmeyer flask deformity of the femora in children (10-17 years old); c). osteoarticular necrosis; d). medullary infarctions; e). lytic lesions; f). pathological fractures or fractures related to Gaucher disease; g). marrow infiltration to a degree such that Rosenthal’s Magnetic Resonance Score was ≥ 3; h). bone density by quantitative computed tomography (QCT) or DEXA ≥ 1.5 standard deviation (SD) below age-adjusted normal value; and i). fat fraction ≤ 17%.

Exclusion Criteria:

  • More than 1 joint replacement (revision surgery such as repair or replacement of a previously replaced joint is allowed).
  • Pregnant, lactating or per-menopausal women.
  • Active, uncontrolled infection, such as hepatitis B, hepatitis C or human immunodeficiency virus (HIV).
  • Major concurrent disorders (i.e. cancer, renal disease) or disorders known to affect bone (e.g. uncontrolled thyroid disease, hyperparathyroidism, hypoparathyroidism, gastrectomy, malabsorption, inflammatory bowel disease, rheumatoid arthritis, ankylosing spondylitis).
  • Medications known to affect bone homeostasis (e.g. chronic oral corticosteroids, anticonvulsants, phenytoin and phenobarbital, hyper-physiological doses of estrogen, defined as > 0.625mg, or androgens, bisphosphates, calcitonin) within the first 2 months of the first Cerezyme infusion.
  • Emotional, behavioral or psychological problems, which in the judgment of the principal investigator, would interfere with the patient adequately complying with the requirement of the study.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00365131

Locations
United States, Florida
Coral Springs, Florida, United States, 33065
Sponsors and Collaborators
Genzyme
Investigators
Study Director: Edward M. Kaye, M.D. Genzyme
  More Information

Additional Information:
No publications provided

Study ID Numbers: RC96-1101
Study First Received: August 15, 2006
Last Updated: July 16, 2007
ClinicalTrials.gov Identifier: NCT00365131     History of Changes
Health Authority: United States: Food and Drug Administration

Keywords provided by Genzyme:
Type I Gaucher disease
Glucocerebrosidase Deficiency Disease

Study placed in the following topic categories:
Lipid Metabolism, Inborn Errors
Sphingolipidoses
Metabolic Diseases
Gaucher Disease Type 1
Lysosomal Storage Diseases
Sphingolipidosis
Central Nervous System Diseases
Brain Diseases
Lymphatic Diseases
Metabolism, Inborn Errors
Malnutrition
Genetic Diseases, Inborn
Nutrition Disorders
Lipidoses
Brain Diseases, Metabolic, Inborn
Gaucher Disease
Metabolic Disorder
Lipid Metabolism Disorders
Deficiency Diseases
Brain Diseases, Metabolic

Additional relevant MeSH terms:
Lipid Metabolism, Inborn Errors
Sphingolipidoses
Disease
Metabolic Diseases
Reticuloendotheliosis
Lysosomal Storage Diseases, Nervous System
Lysosomal Storage Diseases
Nervous System Diseases
Central Nervous System Diseases
Brain Diseases
Metabolism, Inborn Errors
Lymphatic Diseases
Pathologic Processes
Malnutrition
Genetic Diseases, Inborn
Syndrome
Nutrition Disorders
Lipidoses
Brain Diseases, Metabolic, Inborn
Gaucher Disease
Lipid Metabolism Disorders
Deficiency Diseases
Brain Diseases, Metabolic

ClinicalTrials.gov processed this record on May 06, 2009