Gaucher Disease, Type 1Phase 4NCT00364858
Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease
A Phase 4 study testing Cerezyme in people with Gaucher Disease, Type 1 and Cerebroside Lipidosis Syndrome, run by Genzyme, a Sanofi Company at 26 sites in 7 countries.
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®. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Gaucher Disease, Type 1, Cerebroside Lipidosis Syndrome, Glucocerebrosidase Deficiency Disease
- What is being tested?
- Cerezyme
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 95 participants
- Where
- 26 sitesin 7 countries
- Study dates
- Dec 2001to Feb 2007
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Show all 11Show fewer
- 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.
Who cannot join
- 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.
Trial locations and contacts
26 sites in 7 countries.
26 sites in 7 countries
United States18 sites
Emory University
Atlanta, Georgia, 30322
Massachusetts General Hospital
Boston, Massachusetts, 02114
Hemophilia Center of Western New York
Buffalo, New York, 14215
Children's Memorial Hospital
Chicago, Illinois, 60614
Children's Hospital Research Foundation
Cincinnati, Ohio, 45229
University Research Foundation for Lysosomal Storage Disease, Inc.
Coral Springs, Florida, 33065
Duke University Medical Center
Durham, North Carolina, 27710
New York Oncology/Hematology PC
Latham, New York, 12110
Institute for Genetics Medicine Saint Peter's University Hospital
New Brunswick, New Jersey, 08903
New York University
New York, New York, 10016
Mt. Sinai Medical Center
New York, New York, 10029
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
University of Pittsburgh
Pittsburgh, Pennsylvania, 15261
Oregon Health & Science University
Portland, Oregon, 97239
University of Utah
Salt Lake City, Utah, 84132
Midwest Cancer Research Group, Inc.
Skokie, Illinois, 60076
Holy Name Hospital
Teaneck, New Jersey, 07666
Children's National Medical Center
Washington D.C., District of Columbia, 20010
Italy3 sites
Istituto Giannina Gaslini
Genova
Universita degli Studi di Napoli "Federico II"
Naples, 80131
Istituto per l'Infanzia Burlo-Garofolo
Trieste, 34137
Brazil1 site
Estadual de Hematologia Arthur de Siqueira Cavalcanti (HEMORIO)
Rio de Janeiro, 20211
About this study
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
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
- Being tested
- Cerezyme
- Keywords
- Type 1 Gaucher Disease, Glucocerebrosidase Deficiency Disease
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CerezymeDrug
Cerezyme doses of 20-60U/kg every 2 weeks (Q2 Arm) or 40-120 U/kg every 4 weeks (Q4 Arm).
| Study group | Role | What they receive |
|---|---|---|
| Q2 Cerezyme | Other | Patients receiving Cerezyme one infusion every 2 weeks (Q2). Receives: Drug: Cerezyme |
| Q4 Cerezyme | Other | Patients receiving Cerezyme one infusion every 4 weeks (Q4). Receives: Drug: Cerezyme |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Month 24 (or at time of discontinuation)
Secondary outcomes (3)
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.
Measured: Baseline
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.
Measured: Month 24 (or at time of discontinuation)
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.
Measured: Baseline and Month 24 (or at time of discontinuation)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Aug 2006 (estimated)
- Study startDec 2001
- Study completionFeb 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Gaucher Disease, Type 1Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 95 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Edward Kaye, M.D., Study director, Genzyme, a Sanofi Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00364858
- Sponsor's protocol ID
- CZ-011-01
- Status last verified
- Mar 2015
- First submitted
- 15 Aug 2006
- Last updated
- 3 Apr 2015
- Results posted
- Yes, on ClinicalTrials.gov
Study at a glance
From the registry record, last updated by the sponsor on 3 Apr 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00364858
- Acronym / sponsor ID
- CZ-011-01
- 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
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 95 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- Dec 2001
- Study completion
- Feb 2007 (actual)
- First posted
- 16 Aug 2006 (estimated)
- Results
- Posted 2 Sep 2009
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Q2 Cerezyme | Other | Drug: CerezymePatients receiving Cerezyme one infusion every 2 weeks (Q2). |
| Q4 Cerezyme | Other | Drug: CerezymePatients receiving Cerezyme one infusion every 4 weeks (Q4). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- 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.
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CerezymeCerezyme doses of 20-60U/kg every 2 weeks (Q2 Arm) or 40-120 U/kg every 4 weeks (Q4 Arm). | Drug | Q2 Cerezyme, Q4 Cerezyme |
Primary outcome
Number of Participants With Clinical Success at Month 24/Discontinuation
Time frame: Month 24 (or at time of 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.
3 secondary outcomes
Mean Composite Scores of the SF-36 Health Survey at Baseline
Time frame: Baseline
Mean Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation.
Time frame: Month 24 (or at time of discontinuation)
Mean Change From Baseline in Composite Scores of the SF-36 Health Survey at Month 24/Discontinuation
Time frame: Baseline and Month 24 (or at time of discontinuation)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Apr 2015.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Edward Kaye, M.D. study director
Genzyme, a Sanofi Company
26 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Cerezyme in people with Gaucher Disease, Type 1 and Cerebroside Lipidosis Syndrome, run by Genzyme, a Sanofi Company at 26 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Apr 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 26 sites in United States, Italy, Brazil, Canada, Poland and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 2 Sep 2009). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Clinical Success at Month 24/Discontinuation (time frame: Month 24 (or at time of discontinuation)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Apr 2015.