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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT00364858
Other study ID # CZ-011-01
Secondary ID
Status Completed
Phase Phase 4
First received August 15, 2006
Last updated March 17, 2015
Start date December 2001
Est. completion date February 2007

Study information

Verified date March 2015
Source Sanofi
Contact n/a
Is FDA regulated No
Health authority United States: Food and Drug Administration
Study type Interventional

Clinical Trial 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.


Recruitment information / eligibility

Status Completed
Enrollment 95
Est. completion date February 2007
Est. primary completion date
Accepts healthy volunteers No
Gender Both
Age group 18 Years and older
Eligibility 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

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


Related Conditions & MeSH terms


Intervention

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

Locations

Country Name City State
Brazil Estadual de Hematologia Arthur de Siqueira Cavalcanti (HEMORIO) Rio de Janeiro
Canada Mount Sinai Hospital Toronto Ontario
Italy Istituto Giannina Gaslini Genova
Italy Universita degli Studi di Napoli "Federico II" Naples
Italy Istituto per l'Infanzia Burlo-Garofolo Trieste
Poland Instytut Pomnik Centrum Zdrowia Dzeicka Warsaw
Spain Hospital Vall d´Hebrón Barcelona
United Kingdom Royal Free Hospital London
United States Emory University Atlanta Georgia
United States Massachusetts General Hospital Boston Massachusetts
United States Hemophilia Center of Western New York Buffalo New York
United States Children's Memorial Hospital Chicago Illinois
United States Children's Hospital Research Foundation Cincinnati Ohio
United States University Research Foundation for Lysosomal Storage Disease, Inc. Coral Springs Florida
United States Duke University Medical Center Durham North Carolina
United States New York Oncology/Hematology PC Latham New York
United States Institute for Genetics Medicine Saint Peter's University Hospital New Brunswick New Jersey
United States Mt. Sinai Medical Center New York New York
United States New York University New York New York
United States Children's Hospital of Philadelphia Philadelphia Pennsylvania
United States University of Pittsburgh Pittsburgh Pennsylvania
United States Oregon Health & Science University Portland Oregon
United States University of Utah Salt Lake City Utah
United States Midwest Cancer Research Group, Inc. Skokie Illinois
United States Holy Name Hospital Teaneck New Jersey
United States Children's National Medical Center Washington District of Columbia

Sponsors (1)

Lead Sponsor Collaborator
Genzyme, a Sanofi Company

Countries where clinical trial is conducted

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

Outcome

Type Measure Description Time frame Safety issue
Primary Number of Participants With Clinical Success at Month 24/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. Month 24 (or at time of discontinuation) No
Secondary Mean Composite Scores of the SF-36 Health Survey at 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. Baseline No
Secondary Mean Composite Scores of the SF-36 Health Survey at Month 24/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. Month 24 (or at time of discontinuation) No
Secondary Mean Change From Baseline in Composite Scores of the SF-36 Health Survey at Month 24/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. Baseline and Month 24 (or at time of discontinuation) No
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