Gaucher Disease Type IPhase 4NCT00365131
A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.
A Phase 4 study testing Cerezyme (imiglucerase for injection) in people with Gaucher Disease Type I and Cerebroside Lipidosis Syndrome, run by Genzyme, a Sanofi Company at 1 site in United States.
Trial summary
This is a multicenter, open-label, prospective study of the efficacy of Cerezyme in treating patients with skeletal manifestations secondary to Type I Gaucher disease. The study objective is to evaluate and quantify skeletal responses as compared to baseline in Type I gaucher disease patients receiving Cerezyme… Read more about this study
- Who is it for?
- Ages 10 to 65 years
- Conditions the study is for
- Gaucher Disease Type I, Cerebroside Lipidosis Syndrome, Clucocerebrosidase Deficiency Disease
- What is being tested?
- Cerezyme (imiglucerase for injection)
- Type of treatment
- Injection or infusion
- How many people
- 40 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 1997to Jul 2004
- Phase
- Phase 4
- Are there results?
- None posted in the registry
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 10 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Signed informed consent.
- Confirmed diagnosis of Type I Gaucher disease, with no prior enzyme replacement therapy, gene therapy or bone marrow transplantation, and who are ambulatory.
- Age 10-65 (patients 66-70 years of age are considered on a case-by-case basis following careful medical review).
- Dual energy X-ray absorptiometry (DEXA) of the femoral nech with a T-score ≤ -1.0.
- One of more of the following signs as documented by X-ray, computed tomography (CT), or magnetic resonance imaging (MRI), or symptoms of bone disease as documented in the patient's medical history or baseline examinations: a). history of at least one bone crises; b). Erlenmeyer flask deformity of the femora in children (10-17 years old); c). osteoarticular necrosis; d). medullary infarctions; e). lytic lesions; f). pathological fractures or fractures related to Gaucher disease; g). marrow infiltration to a degree such that Rosenthal's Magnetic Resonance Score was ≥ 3; h). bone density by quantitative computed tomography (QCT) or DEXA ≥ 1.5 standard deviation (SD) below age-adjusted normal value; and i). fat fraction ≤ 17%.
Who cannot join
- More than 1 joint replacement (revision surgery such as repair or replacement of a previously replaced joint is allowed).
- Pregnant, lactating or per-menopausal women.
- Active, uncontrolled infection, such as hepatitis B, hepatitis C or human immunodeficiency virus (HIV).
- Major concurrent disorders (i.e. cancer, renal disease) or disorders known to affect bone (e.g. uncontrolled thyroid disease, hyperparathyroidism, hypoparathyroidism, gastrectomy, malabsorption, inflammatory bowel disease, rheumatoid arthritis, ankylosing spondylitis).
- Medications known to affect bone homeostasis (e.g. chronic oral corticosteroids, anticonvulsants, phenytoin and phenobarbital, hyper-physiological doses of estrogen, defined as > 0.625mg, or androgens, bisphosphates, calcitonin) within the first 2 months of the first Cerezyme infusion.
- Emotional, behavioral or psychological problems, which in the judgment of the principal investigator, would interfere with the patient adequately complying with the requirement of the study.
Trial locations and contacts
1 site in 1 country.
About this study
This is a multicenter, open-label, prospective study of the efficacy of Cerezyme in treating patients with skeletal manifestations secondary to Type I Gaucher disease.
The study objective is to evaluate and quantify skeletal responses as compared to baseline in Type I gaucher disease patients receiving Cerezyme therapy for 48 months. Additional objectives were to assess the usefulness of various skeletal parameters, such as bone pain, bone crises, bone mineral density, and serum and urine bone markers, as indicative of treatment response and may be useful in dose management.
- Conditions
- Gaucher Disease Type I, Cerebroside Lipidosis Syndrome, Clucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease, Gaucher Disease, Non-Neuronopathic Form
- Being tested
- Cerezyme (imiglucerase for injection)
- Keywords
- Type I 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.
Cerezyme (imiglucerase for injection)Drug
What is being measured
The primary outcome is the main question the study is designed to answer.
Skeletal response over 4 years of Cerezyme therapy
Assess use of skeletal parameter as indicative of treatment response and use in dose management
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Aug 2006 (estimated)
- Study startDec 1997
- Study completionJul 2004
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gaucher Disease Type IStudies 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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Medical Monitor, Study director, Genzyme, a Sanofi Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00365131
- Sponsor's protocol ID
- RC96-1101
- Status last verified
- Mar 2015
- First submitted
- 15 Aug 2006
- Last updated
- 5 Mar 2015
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.
- ClinicalTrials.gov ID
- NCT00365131
- Acronym / sponsor ID
- RC96-1101
- Conditions
- Gaucher Disease Type I, Cerebroside Lipidosis Syndrome, Clucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease, Gaucher Disease, Non-Neuronopathic Form
- Keywords
- Type I Gaucher disease, Glucocerebrosidase Deficiency Disease
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- Dec 1997
- Study completion
- Jul 2004 (actual)
- First posted
- 17 Aug 2006 (estimated)
- Results
- Not posted in the source record
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, open-label, prospective study of the efficacy of Cerezyme in treating patients with skeletal manifestations secondary to Type I Gaucher disease.
The study objective is to evaluate and quantify skeletal responses as compared to baseline in Type I gaucher disease patients receiving Cerezyme therapy for 48 months. Additional objectives were to assess the usefulness of various skeletal parameters, such as bone pain, bone crises, bone mineral density, and serum and urine bone markers, as indicative of treatment response and may be useful in dose management.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 10 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Signed informed consent.
- Confirmed diagnosis of Type I Gaucher disease, with no prior enzyme replacement therapy, gene therapy or bone marrow transplantation, and who are ambulatory.
- Age 10-65 (patients 66-70 years of age are considered on a case-by-case basis following careful medical review).
- Dual energy X-ray absorptiometry (DEXA) of the femoral nech with a T-score ≤ -1.0.
- One of more of the following signs as documented by X-ray, computed tomography (CT), or magnetic resonance imaging (MRI), or symptoms of bone disease as documented in the patient's medical history or baseline examinations: a). history of at least one bone crises; b). Erlenmeyer flask deformity of the femora in children (10-17 years old); c). osteoarticular necrosis; d). medullary infarctions; e). lytic lesions; f). pathological fractures or fractures related to Gaucher disease; g). marrow infiltration to a degree such that Rosenthal's Magnetic Resonance Score was ≥ 3; h). bone density by quantitative computed tomography (QCT) or DEXA ≥ 1.5 standard deviation (SD) below age-adjusted normal value; and i). fat fraction ≤ 17%.
Key exclusion criteria (6 listed)
- More than 1 joint replacement (revision surgery such as repair or replacement of a previously replaced joint is allowed).
- Pregnant, lactating or per-menopausal women.
- Active, uncontrolled infection, such as hepatitis B, hepatitis C or human immunodeficiency virus (HIV).
- Major concurrent disorders (i.e. cancer, renal disease) or disorders known to affect bone (e.g. uncontrolled thyroid disease, hyperparathyroidism, hypoparathyroidism, gastrectomy, malabsorption, inflammatory bowel disease, rheumatoid arthritis, ankylosing spondylitis).
- Medications known to affect bone homeostasis (e.g. chronic oral corticosteroids, anticonvulsants, phenytoin and phenobarbital, hyper-physiological doses of estrogen, defined as > 0.625mg, or androgens, bisphosphates, calcitonin) within the first 2 months of the first Cerezyme infusion.
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 |
|---|---|---|
| Cerezyme (imiglucerase for injection) | Drug | – |
Primary outcome
Skeletal response over 4 years of Cerezyme therapy
Assess use of skeletal parameter as indicative of treatment response and use in dose management
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Mar 2015.
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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.
Medical Monitor study director
Genzyme, a Sanofi Company
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Cerezyme (imiglucerase for injection) in people with Gaucher Disease Type I and Cerebroside Lipidosis Syndrome, run by Genzyme, a Sanofi Company at 1 site in United States.
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 5 inclusion and 6 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 5 Mar 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Skeletal response over 4 years of Cerezyme therapy. The study lists 2 primary outcomes in all. 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 5 Mar 2015.