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

Sponsor:	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

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

Resource links provided by NLM:

Genetics Home Reference related topics: argininosuccinic aciduria ataxia neuropathy spectrum carbamoyl phosphate synthetase I deficiency Chanarin-Dorfman syndrome childhood myocerebrohepatopathy spectrum cholesteryl ester storage disease citrullinemia deoxyguanosine kinase deficiency Farber lipogranulomatosis Gaucher disease mitochondrial neurogastrointestinal encephalopathy disease myoclonic epilepsy myopathy sensory ataxia N-acetylglutamate synthase deficiency ornithine translocase deficiency Schindler disease succinic semialdehyde dehydrogenase deficiency

MedlinePlus related topics: Gaucher's Disease

Drug Information available for: Alglucerase Imiglucerase

U.S. FDA Resources

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:

Medical Monitor

Genzyme

More Information

Additional Information:

Results synopsis for RC96-1101 This link exits the ClinicalTrials.gov site

US FDA Approved Full Prescribing Information for Cerezyme® This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party:	Medical Monitor, Genzyme Corporation
ClinicalTrials.gov Identifier:	NCT00365131 History of Changes
Other Study ID Numbers:	RC96-1101
Study First Received:	August 15, 2006
Last Updated:	July 31, 2009
Health Authority:	United States: Food and Drug Administration

Keywords provided by Genzyme:

Type I Gaucher disease
Glucocerebrosidase Deficiency Disease

Additional relevant MeSH terms:

Deficiency Diseases
Carbamoyl-Phosphate Synthase I Deficiency Disease
Gaucher Disease
Lipidoses
Malnutrition
Nutrition Disorders
Urea Cycle Disorders, Inborn
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases

Nervous System Diseases
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
Mitochondrial Diseases
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Lipid Metabolism Disorders

ClinicalTrials.gov processed this record on February 09, 2012