A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.
Information source: Genzyme
Information obtained from ClinicalTrials.gov on June 20, 2008
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Gaucher Disease Type I; Cerebroside Lipidosis Syndrome; Clucocerebrosidase Deficiency Disease; Glucosylceramide Beta-Glucosidase Deficiency Disease; Gaucher Disease, Non-Neuronopathic Form
Intervention: Cerezyme (imiglucerase for injection) (Drug)
Phase: Phase 4
Sponsored by: Genzyme
Official(s) and/or principal investigator(s):
Edward M. Kaye, M.D., Study Director, Affiliation: Genzyme
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.
Study design: Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Efficacy Study
Skeletal response over 4 years of Cerezyme therapy
Assess use of skeletal parameter as indicative of treatment response and use in dose management
Minimum age: 10 Years.
Maximum age: 65 Years.
- 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%.
- 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,
- 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.
Locations and Contacts
Coral Springs, Florida 33065, United States
Results synopsis for RC96-1101
US FDA Approved Full Prescribing Information for Cerezyme®
Starting date: December 1997
Last updated: July 16, 2007