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

• Register: ClinicalTrials.gov
• Last refreshed on: 16 March 2015
• Main ID: NCT00365131
• Date of registration: 15/08/2006
• Prospective Registration: No
• Primary sponsor: Genzyme, a Sanofi Company
• Public title: A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.
• Date of first enrolment: December 1997
• Target sample size: 40
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00365131
• Study type: Interventional
• Study design: Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 4

Countries of recruitment

United States

Contacts

• Name: Medical Monitor
• Affiliation: Genzyme, a Sanofi Company

Key inclusion & exclusion 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.



Age minimum: 10 Years
Age maximum: 65 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Gaucher Disease Type I

Glucosylceramide Beta-Glucosidase Deficiency Disease

Cerebroside Lipidosis Syndrome

Gaucher Disease, Non-Neuronopathic Form

Clucocerebrosidase Deficiency Disease

Intervention(s)

Drug: Cerezyme (imiglucerase for injection)

Primary Outcome(s)

Assess use of skeletal parameter as indicative of treatment response and use in dose management

Skeletal response over 4 years of Cerezyme therapy

Secondary ID(s)

RC96-1101

Source(s) of Monetary Support

Please refer to primary and secondary sponsors