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A Phase IV, Multicenter, Randomized, Dose Frequency Study of the Safety and Efficacy of Cerezyme® Infusions Every Four Weeks Versus Every Two Weeks in the Maintenance Therapy of Patients With Type 1 Gaucher Disease


Phase 4
18 Years
N/A
Not Enrolling
Both
Gaucher Disease, Type 1, Cerebroside Lipidosis Syndrome, Glucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease, Gaucher Disease, Non-Neuronopathic Form

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Trial Information

A Phase IV, Multicenter, Randomized, Dose Frequency Study of the Safety and Efficacy of Cerezyme® Infusions Every Four Weeks Versus Every Two Weeks in the Maintenance Therapy of Patients With Type 1 Gaucher Disease


Inclusion Criteria:



- The patient must provide written informed consent prior to undergoing any
study-related procedures.

- The patient has a confirmed diagnosis of Gaucher disease with a documented deficiency
of glucocerebrosidase by enzyme assay

- The patient has been genotyped or will have genotyping performed within 3 months of
study enrollment.

- The patient has been treated with Cerezyme for at least 2 years prior to study
enrollment.

- The patient has been on a stable dose of between 20-60 U/kg every 2 weeks for at
least 6 months prior to study enrollment.

- The patient is at least 18 years old.

- The patient has a hemoglobin value of ≥ 11.0 g/dL for women and ≥ 12.0 g/dL for men
and a platelet count of ≥ 100,000 mm^3.

- The patient's liver volume is ≤ 1.8 x normal confirmed by MRI or CT within 6 months
of randomization.

- The patient's spleen volume is ≤ 10 x normal confirmed by MRI or CT within 6 months
of randomization.

- The patient has a serum creatinine < 2.0 mg/dL, an ASTand ALT < 2 x upper limit of
normal and a total bilirubin < 2.0 x upper limit of normal.

- Female patients of childbearing potential must have a negative pregnancy test within
2 weeks prior to randomization into the study.

Exclusion Criteria:

- The patient is pregnant.

- The patient has evidence of neurologic or pulmonary involvement with Gaucher disease
confirmed by medical history.

- The patient has evidence of current or prior bleeding varices or liver infarction
requiring hospitalization confirmed by medical history.

- The patient has evidence of pathologic bone fractures, medullary infarctions, lytic
lesions or avascular necrosis secondary to Gaucher disease confirmed by skeletal
evaluation within 6 months of randomization.

- The patient has had a bone crisis (defined as pain with acute onset which requires
immobilization of the affected area, narcotics for relief of pain and may be
accompanied by periosteal elevation, increased white cell count, fever or
debilitation of > 3 days) within 12 months of randomization.

- Patient has received an investigational drug within 30 days of the start of their
participation in this trial. Patients may not receive any other investigational
product throughout the course of the study.

- The patient has a clinically significant disease (with the exception of symptoms
relating to Gaucher disease), including clinically significant cardiovascular,
hepatic, immunologic, pulmonary, neurologic, or renal disease, or other medical
condition, serious intercurrent illness, or extenuating circumstances that, in the
opinion of the Investigator, would preclude participation in the trial or potentially
decrease survival

- Patient has a medical, emotional, behavioral or psychological condition that in the
judgment of the Investigator would interfere with the patient's compliance with the
requirements of the study.

Type of Study:

Interventional

Study Design:

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Number of Participants With Clinical Success at Month 24/Discontinuation

Outcome Time Frame:

Month 24 (or at time of discontinuation)

Safety Issue:

No

Principal Investigator

Edward Kaye, M.D.

Investigator Role:

Study Director

Investigator Affiliation:

Genzyme

Authority:

United States: Food and Drug Administration

Study ID:

CZ-011-01

NCT ID:

NCT00364858

Start Date:

December 2001

Completion Date:

February 2007

Related Keywords:

  • Gaucher Disease, Type 1
  • Cerebroside Lipidosis Syndrome
  • Glucocerebrosidase Deficiency Disease
  • Glucosylceramide Beta-Glucosidase Deficiency Disease
  • Gaucher Disease, Non-Neuronopathic Form
  • Type 1 Gaucher Disease
  • Glucocerebrosidase Deficiency Disease
  • Deficiency Diseases
  • Gaucher Disease
  • Lipidoses

Name

Location

Children's Hospital of Philadelphia Philadelphia, Pennsylvania  19104
Children's National Medical Center Washington, District of Columbia  20010-2970
Massachusetts General Hospital Boston, Massachusetts  02114-2617
Midwest Cancer Research Group, Inc. Skokie, Illinois  60077
University of Pittsburgh Pittsburgh, Pennsylvania  15261
University of Utah Salt Lake City, Utah  
Oregon Health & Science University Portland, Oregon  97201
Duke University Medical Center Durham, North Carolina  27710
Emory University Atlanta, Georgia  30322
Children's Memorial Hospital Chicago, Illinois  60614
New York University New York, New York  10016
Mt. Sinai Medical Center New York, New York  10029
Holy Name Hospital Teaneck, New Jersey  07666
University Research Foundation for Lysosomal Storage Disease, Inc. Coral Springs, Florida  33065
Institute for Genetics Medicine Saint Peter's University Hospital New Brunswick, New Jersey  08903
Hemophilia Center of Western New York Buffalo, New York  14215
New York Oncology/Hematology PC Latham, New York  12110
Children's Hospital Research Foundation Cincinnati, Ohio  45229