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

Phase 4

Completed

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

Interventions: Drug: Cerezyme

Registration Number: NCT00364858

Lead Sponsor: Genzyme, a Sanofi Company

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.

Detailed Description: Not available

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 95

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.

Study & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Q4 Cerezyme	Cerezyme	Patients receiving Cerezyme one infusion every 4 weeks (Q4).
Q2 Cerezyme	Cerezyme	Patients receiving Cerezyme one infusion every 2 weeks (Q2).

Primary Outcome Measures

Name	Time	Method
Number of Participants With Clinical Success at Month 24/Discontinuation	Month 24 (or at time of discontinuation)	Patients are considered to be a clinical success if ALL of the following are met: The patient's hemoglobin does not fall more than 1.25g/dL for women or 1.5 g/dL for men below the patient's baseline value, platelet count does not fall more than 25% below the patient's baseline value or does not fall below 80,000 mm3, liver and spleen volumes are not greater than 20% above the patient's baseline value, no evidence of bone disease progression, including no incidence of pathologic fractures, medullary infarctions, lytic lesions or avascular necrosis and has had no bone crises during the study.

Secondary Outcome Measures

Name	Time	Method
Mean Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation.	Month 24 (or at time of discontinuation)	The mean composite scores (0 being worst and 100 being best) for both treatment groups at Month 24/Discontinuation. The mean composite scores for both treatment groups approximated those of the general population at baseline and at Month 24.
Mean Composite Scores of the SF-36 Health Survey at Baseline	Baseline	The mean composite scores (0 being worst and 100 being best) for both treatment groups at Baseline. Composite scores for both treatment groups approximated those of the general population at baseline and at Month 24.
Mean Change From Baseline in Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation	Baseline and Month 24 (or at time of discontinuation)	The mean composite scores (0 being worst and 100 being best) for both treatment groups approximated those of the general population at baseline. Composite score - The overall composite scores were comprised of a standardized physical and mental component score.

Trial Locations

Locations (26): Emory University
🇺🇸
Atlanta, Georgia, United States
University Research Foundation for Lysosomal Storage Disease, Inc.
🇺🇸
Coral Springs, Florida, United States
Midwest Cancer Research Group, Inc.
🇺🇸
Skokie, Illinois, United States
Children's Memorial Hospital
🇺🇸
Chicago, Illinois, United States
Massachusetts General Hospital
🇺🇸
Boston, Massachusetts, 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
Children's Hospital Research Foundation
🇺🇸
Cincinnati, Ohio, United States
Oregon Health & Science University
🇺🇸
Portland, Oregon, United States
Mt. Sinai Medical Center
🇺🇸
New York, New York, United States
Duke University Medical Center
🇺🇸
Durham, North Carolina, United States
Children's Hospital of Philadelphia
🇺🇸
Philadelphia, Pennsylvania, United States
University of Utah
🇺🇸
Salt Lake City, Utah, United States
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
Hospital Vall d´Hebrón
🇪🇸
Barcelona, Spain
Royal Free Hospital
🇬🇧
London, United Kingdom
Children's National Medical Center
🇺🇸
Washington, District of Columbia, 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
University of Pittsburgh
🇺🇸
Pittsburgh, Pennsylvania, United States
Instytut Pomnik Centrum Zdrowia Dzeicka
🇵🇱
Warsaw, Poland
Estadual de Hematologia Arthur de Siqueira Cavalcanti (HEMORIO)
🇧🇷
Rio de Janeiro, Brazil