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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.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00365131
First Posted: August 17, 2006
Last Update Posted: March 5, 2015
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by:
Sanofi
  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 4

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment

Resource links provided by NLM:


Further study details as provided by Sanofi:

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
Study Completion Date: July 2004
  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   10 Years to 65 Years   (Child, Adult)
Sexes Eligible for Study:   All
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
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): NCT00365131


Locations
United States, Florida
Coral Springs, Florida, United States, 33065
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

Additional Information:
Responsible Party: Medical Monitor, Genzyme Corporation
ClinicalTrials.gov Identifier: NCT00365131     History of Changes
Other Study ID Numbers: RC96-1101
First Submitted: August 15, 2006
First Posted: August 17, 2006
Last Update Posted: March 5, 2015
Last Verified: March 2015

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

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