A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.

Cerebroside Lipidosis Syndrome Clinical Trial

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00365131
• Other study ID #: RC96-1101
• Status: Completed
• Phase: Phase 4
• First received: August 15, 2006
• Last updated: March 4, 2015
• Start date: December 1997
• Est. completion date: July 2004

Study information

• Verified date: March 2015
• Source: Sanofi
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 40
• Est. completion date: July 2004
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 10 Years to 65 Years

Eligibility

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.

Study Design

Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Cerebroside Lipidosis Syndrome
• Clucocerebrosidase Deficiency Disease
• Deficiency Diseases
• Gaucher Disease
• Gaucher Disease Type I
• Gaucher Disease, Non-Neuronopathic Form
• Glucosylceramide Beta-Glucosidase Deficiency Disease

Intervention

Drug:
• Cerezyme (imiglucerase for injection)

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Genzyme, a Sanofi Company

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Skeletal response over 4 years of Cerezyme therapy
Primary	Assess use of skeletal parameter as indicative of treatment response and use in dose management

External Links

•   US FDA Approved Full Prescribing Information for Cerezyme®