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

NCT00364858 | Completed Phase 4 Results

• 1 other identifier: CZ-011-01
• Study Type: interventional
• Target: 95
• Locations: 7 countries
• Sites: 26

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

Keywords

Type 1 Gaucher Disease; Glucocerebrosidase Deficiency Disease

Outcome Measures

Primary Outcomes (1)

• 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)

Secondary Outcomes (3)

• Mean Composite Scores of the SF-36 Health Survey at Baseline

  Baseline

• Mean Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation.

  Month 24 (or at time of discontinuation)

• 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)

Study Arms (2)

Q2 Cerezyme

OTHER

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

Drug: Cerezyme

Q4 Cerezyme

OTHER

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

Drug: Cerezyme

Interventions

Cerezyme DRUG

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

Q2 Cerezyme; Q4 Cerezyme

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Genzyme, a Sanofi Company: lead

Study Sites (26)

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

University Research Foundation for Lysosomal Storage Disease, Inc.

Coral Springs, Florida, 33065, United States

Location

Emory University

Atlanta, Georgia, 30322, United States

Location

Children's Memorial Hospital

Chicago, Illinois, 60614, United States

Location

Midwest Cancer Research Group, Inc.

Skokie, Illinois, 60076, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Institute for Genetics Medicine Saint Peter's University Hospital

New Brunswick, New Jersey, 08903, United States

Location

Holy Name Hospital

Teaneck, New Jersey, 07666, United States

Location

Hemophilia Center of Western New York

Buffalo, New York, 14215, United States

Location

New York Oncology/Hematology PC

Latham, New York, 12110, United States

Location

New York University

New York, New York, 10016, United States

Location

Mt. Sinai Medical Center

New York, New York, 10029, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Children's Hospital Research Foundation

Cincinnati, Ohio, 45229, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

University of Pittsburgh

Pittsburgh, Pennsylvania, 15261, United States

Location

University of Utah

Salt Lake City, Utah, 84132, United States

Location

Estadual de Hematologia Arthur de Siqueira Cavalcanti (HEMORIO)

Rio de Janeiro, 20211, Brazil

Location

Mount Sinai Hospital

Toronto, Ontario, ON M5G 1X5, Canada

Location

Istituto Giannina Gaslini

Genova, Italy

Location

Universita degli Studi di Napoli "Federico II"

Naples, 80131, Italy

Location

Istituto per l'Infanzia Burlo-Garofolo

Trieste, 34137, Italy

Location

Instytut Pomnik Centrum Zdrowia Dzeicka

Warsaw, 04-736, Poland

Location

Hospital Vall d´Hebrón

Barcelona, 08035, Spain

Location

Royal Free Hospital

London, NW3 2QG, United Kingdom

Location

MeSH Terms

Conditions

Gaucher Disease

Interventions

imiglucerase

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Results Point of Contact

• Title: Genzyme Medical Information
• Organization: Genzyme Corporation

800-745-4447

Study Officials

• Edward Kaye, M.D.

  Genzyme, a Sanofi Company

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY

Study Record Dates

• First Submitted: August 15, 2006
• First Posted: August 16, 2006
• Study Start: December 1, 2001
• Study Completion: February 1, 2007
• Last Updated: April 3, 2015
• Results First Posted: September 2, 2009

Record last verified: 2015-03

Locations

CA (1); GB (1); IT (3); ES (1); BR (1); US (18); PL (1)