Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease

NCT ID: NCT00364858

Last Updated: 2015-04-03

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE4
• Total Enrollment: 95 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2001-12-31
• Study Completion Date: 2007-02-28

Brief Summary

This is a multicenter, randomized trial to compare the safety and efficacy of two dosing frequencies of Cerezyme® in patients with Gaucher disease who are currently being treated with Cerezyme®.

Approximately 90 patients will be randomized in a 2:1 (q4 : q2) ratio to one of two treatment arms at up to 26 study centers worldwide. Patients will continue to receive the same total 4-week dose that they were receiving prior to study enrollment, however, they will be randomized to receive either their total 4-week dose in two infusions, one infusion every 2 weeks or their total 4-week dose in one infusion every 4 weeks. The randomization scheme will ensure a 2:1 balance between the every 4-week versus every 2-week infusion groups, respectively.

Conditions

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Q2 Cerezyme

Patients receiving Cerezyme one infusion every 2 weeks (Q2).

Group Type: OTHER

Cerezyme

Intervention Type: DRUG

Cerezyme doses of 20-60U/kg every 2 weeks (Q2 Arm) or 40-120 U/kg every 4 weeks (Q4 Arm).

Q4 Cerezyme

Patients receiving Cerezyme one infusion every 4 weeks (Q4).

Group Type: OTHER

Cerezyme

Intervention Type: DRUG

Cerezyme doses of 20-60U/kg every 2 weeks (Q2 Arm) or 40-120 U/kg every 4 weeks (Q4 Arm).

Interventions

Cerezyme

Cerezyme doses of 20-60U/kg every 2 weeks (Q2 Arm) or 40-120 U/kg every 4 weeks (Q4 Arm).

Intervention Type: DRUG

Eligibility Criteria

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Genzyme, a Sanofi Company

INDUSTRY

Sponsor Role: lead

Responsible Party

Genzyme Coporation

Principal Investigators

Edward Kaye, M.D.

Role: STUDY_DIRECTOR

Genzyme, a Sanofi Company

Locations

Children's National Medical Center

Washington D.C., District of Columbia, United States

University Research Foundation for Lysosomal Storage Disease, Inc.

Coral Springs, Florida, United States

Emory University

Atlanta, Georgia, United States

Children's Memorial Hospital

Chicago, Illinois, United States

Midwest Cancer Research Group, Inc.

Skokie, Illinois, United States

Massachusetts General Hospital

Boston, Massachusetts, United States

Institute for Genetics Medicine Saint Peter's University Hospital

New Brunswick, New Jersey, United States

Holy Name Hospital

Teaneck, New Jersey, United States

Hemophilia Center of Western New York

Buffalo, New York, United States

New York Oncology/Hematology PC

Latham, New York, United States

New York University

New York, New York, United States

Mt. Sinai Medical Center

New York, New York, United States

Duke University Medical Center

Durham, North Carolina, United States

Children's Hospital Research Foundation

Cincinnati, Ohio, United States

Oregon Health & Science University

Portland, Oregon, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

University of Pittsburgh

Pittsburgh, Pennsylvania, United States

University of Utah

Salt Lake City, Utah, United States

Estadual de Hematologia Arthur de Siqueira Cavalcanti (HEMORIO)

Rio de Janeiro, , Brazil

Mount Sinai Hospital

Toronto, Ontario, Canada

Istituto Giannina Gaslini

Genova, , Italy

Universita degli Studi di Napoli "Federico II"

Naples, , Italy

Istituto per l'Infanzia Burlo-Garofolo

Trieste, , Italy

Instytut Pomnik Centrum Zdrowia Dzeicka

Warsaw, , Poland

Hospital Vall d´Hebrón

Barcelona, , Spain

Royal Free Hospital

London, , United Kingdom

Countries

United States; Brazil; Canada; Italy; Poland; Spain; United Kingdom

Other Identifiers

CZ-011-01

Identifier Type: -

Identifier Source: org_study_id