Clinical trial NCT00364858: Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease
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 type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
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
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2006-08-16
Enrollment: 95
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility 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.

Primary outcomes:
measure: Number of Participants With Clinical Success at Month 24/Discontinuation
timeFrame: Month 24 (or at time of discontinuation)
Secondary outcomes:
measure: Mean Composite Scores of the SF-36 Health Survey at Baseline
timeFrame: Baseline
measure: Mean Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation.
timeFrame: Month 24 (or at time of discontinuation)
measure: Mean Change From Baseline in Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation
timeFrame: Baseline and Month 24 (or at time of discontinuation)
Other outcomes:
Locations:
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
city: Coral Springs
country: United States
facility: University Research Foundation for Lysosomal Storage Disease, Inc.
state: Florida
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Chicago
country: United States
facility: Children's Memorial Hospital
state: Illinois
city: Skokie
country: United States
facility: Midwest Cancer Research Group, Inc.
state: Illinois
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: New Brunswick
country: United States
facility: Institute for Genetics Medicine Saint Peter's University Hospital
state: New Jersey
city: Teaneck
country: United States
facility: Holy Name Hospital
state: New Jersey
city: Buffalo
country: United States
facility: Hemophilia Center of Western New York
state: New York
city: Latham
country: United States
facility: New York Oncology/Hematology PC
state: New York
city: New York
country: United States
facility: New York University
state: New York
city: New York
country: United States
facility: Mt. Sinai Medical Center
state: New York
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Cincinnati
country: United States
facility: Children's Hospital Research Foundation
state: Ohio
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh
state: Pennsylvania
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Rio de Janeiro
country: Brazil
facility: Estadual de Hematologia Arthur de Siqueira Cavalcanti (HEMORIO)
city: Toronto
country: Canada
facility: Mount Sinai Hospital
state: Ontario
city: Genova
country: Italy
facility: Istituto Giannina Gaslini
city: Naples
country: Italy
facility: Universita degli Studi di Napoli "Federico II"
city: Trieste
country: Italy
facility: Istituto per l'Infanzia Burlo-Garofolo
city: Warsaw
country: Poland
facility: Instytut Pomnik Centrum Zdrowia Dzeicka
city: Barcelona
country: Spain
facility: Hospital Vall d´Hebrón
city: London
country: United Kingdom
facility: Royal Free Hospital
Study officials:
affiliation: Genzyme, a Sanofi Company
name: Edward Kaye, M.D.
role: STUDY_DIRECTOR
References: