Safety and Efficacy of Cerezyme� Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease
Information source: Sanofi
ClinicalTrials.gov processed this data on August 20, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Gaucher Disease, Type 1; Cerebroside Lipidosis Syndrome; Glucocerebrosidase Deficiency Disease; Glucosylceramide Beta-Glucosidase Deficiency Disease; Gaucher Disease, Non-Neuronopathic Form
Intervention: Cerezyme (Drug)
Phase: Phase 4
Status: Completed
Sponsored by: Genzyme, a Sanofi Company Official(s) and/or principal investigator(s): Edward Kaye, M.D., Study Director, Affiliation: Genzyme, a Sanofi Company
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.
Clinical Details
Official title: 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
Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary outcome: Number of Participants With Clinical Success at Month 24/Discontinuation
Secondary outcome: Mean Composite Scores of the SF-36 Health Survey at BaselineMean Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation. Mean Change From Baseline in Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation
Eligibility
Minimum age: 18 Years.
Maximum age: N/A.
Gender(s): Both.
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.
Locations and Contacts
Estadual de Hematologia Arthur de Siqueira Cavalcanti (HEMORIO), Rio de Janeiro 20211, Brazil
Istituto Giannina Gaslini, Genova, Italy
Universita degli Studi di Napoli "Federico II", Naples 80131, Italy
Istituto per l'Infanzia Burlo-Garofolo, Trieste 34137, Italy
Instytut Pomnik Centrum Zdrowia Dzeicka, Warsaw 04-736, Poland
Hospital Vall d´Hebrón, Barcelona 08035, Spain
Royal Free Hospital, London NW3 2QG, United Kingdom
Children's National Medical Center, Washington, District of Columbia 20010, United States
University Research Foundation for Lysosomal Storage Disease, Inc., Coral Springs, Florida 33065, United States
Emory University, Atlanta, Georgia 30322, United States
Children's Memorial Hospital, Chicago, Illinois 60614, United States
Midwest Cancer Research Group, Inc., Skokie, Illinois 60076, United States
Massachusetts General Hospital, Boston, Massachusetts 02114, United States
Institute for Genetics Medicine Saint Peter's University Hospital, New Brunswick, New Jersey 08903, United States
Holy Name Hospital, Teaneck, New Jersey 07666, United States
Hemophilia Center of Western New York, Buffalo, New York 14215, United States
New York Oncology/Hematology PC, Latham, New York 12110, United States
Mt. Sinai Medical Center, New York, New York 10029, United States
New York University, New York, New York 10016, United States
Duke University Medical Center, Durham, North Carolina 27710, United States
Children's Hospital Research Foundation, Cincinnati, Ohio 45229, United States
Mount Sinai Hospital, Toronto, Ontario ON M5G 1X5, Canada
Oregon Health & Science University, Portland, Oregon 97239, United States
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, United States
University of Pittsburgh, Pittsburgh, Pennsylvania 15261, United States
University of Utah, Salt Lake City, Utah 84132, United States
Additional Information
US FDA Approved Full Prescribing Information for Cerezyme®
Starting date: December 2001
Last updated: March 17, 2015
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