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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 Baseline

Mean 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

Page last updated: August 20, 2015

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