Gaucher Disease, Type 1 Clinical Trial
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
Verified date | March 2015 |
Source | Sanofi |
Contact | n/a |
Is FDA regulated | No |
Health authority | United States: Food and Drug Administration |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 95 |
Est. completion date | February 2007 |
Est. primary completion date | |
Accepts healthy volunteers | No |
Gender | Both |
Age group | 18 Years and older |
Eligibility |
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. |
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
Brazil | Estadual de Hematologia Arthur de Siqueira Cavalcanti (HEMORIO) | Rio de Janeiro | |
Canada | Mount Sinai Hospital | Toronto | Ontario |
Italy | Istituto Giannina Gaslini | Genova | |
Italy | Universita degli Studi di Napoli "Federico II" | Naples | |
Italy | Istituto per l'Infanzia Burlo-Garofolo | Trieste | |
Poland | Instytut Pomnik Centrum Zdrowia Dzeicka | Warsaw | |
Spain | Hospital Vall d´Hebrón | Barcelona | |
United Kingdom | Royal Free Hospital | London | |
United States | Emory University | Atlanta | Georgia |
United States | Massachusetts General Hospital | Boston | Massachusetts |
United States | Hemophilia Center of Western New York | Buffalo | New York |
United States | Children's Memorial Hospital | Chicago | Illinois |
United States | Children's Hospital Research Foundation | Cincinnati | Ohio |
United States | University Research Foundation for Lysosomal Storage Disease, Inc. | Coral Springs | Florida |
United States | Duke University Medical Center | Durham | North Carolina |
United States | New York Oncology/Hematology PC | Latham | New York |
United States | Institute for Genetics Medicine Saint Peter's University Hospital | New Brunswick | New Jersey |
United States | Mt. Sinai Medical Center | New York | New York |
United States | New York University | New York | New York |
United States | Children's Hospital of Philadelphia | Philadelphia | Pennsylvania |
United States | University of Pittsburgh | Pittsburgh | Pennsylvania |
United States | Oregon Health & Science University | Portland | Oregon |
United States | University of Utah | Salt Lake City | Utah |
United States | Midwest Cancer Research Group, Inc. | Skokie | Illinois |
United States | Holy Name Hospital | Teaneck | New Jersey |
United States | Children's National Medical Center | Washington | District of Columbia |
Lead Sponsor | Collaborator |
---|---|
Genzyme, a Sanofi Company |
United States, Brazil, Canada, Italy, Poland, Spain, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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) | No |
Secondary | 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. | Baseline | No |
Secondary | 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. | Month 24 (or at time of discontinuation) | No |
Secondary | 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. | Baseline and Month 24 (or at time of discontinuation) | No |
Status | Clinical Trial | Phase | |
---|---|---|---|
Active, not recruiting |
NCT00943111 -
A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)
|
Phase 3 | |
Recruiting |
NCT05687474 -
Baby Detect : Genomic Newborn Screening
|
||
Active, not recruiting |
NCT00891202 -
A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)
|
Phase 3 | |
Recruiting |
NCT05253560 -
Prodromal Parkinsonian Features in GBA1 Mutation Carriers
|
||
Completed |
NCT00813865 -
A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients
|
Phase 2 | |
Completed |
NCT00433147 -
A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy
|
Phase 2 | |
Completed |
NCT00635427 -
An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease
|
Phase 3 | |
Completed |
NCT03950050 -
Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy
|
Phase 2 | |
Terminated |
NCT03746587 -
Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3
|
Phase 2 | |
Active, not recruiting |
NCT00358150 -
A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients
|
Phase 2 | |
Completed |
NCT00446550 -
A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease
|
Phase 2 | |
Recruiting |
NCT05447494 -
Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease
|
Phase 1/Phase 2 | |
Completed |
NCT00553631 -
Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease
|
Phase 3 | |
Completed |
NCT03333447 -
Retrospective and Prospective Observational Study of MRI Changes in Bone and Visceral Lesions of Patients With Type 1 Gaucher Disease Treated With VPRIV® (Velaglucerase Alfa)
|
||
Completed |
NCT04120506 -
Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)
|
Phase 4 | |
Completed |
NCT04353466 -
Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs
|
N/A | |
Completed |
NCT00430625 -
A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease
|
Phase 3 | |
Withdrawn |
NCT00795197 -
A Screening Study Evaluating Disease Status of Gaucher Type I Patients
|
N/A | |
Recruiting |
NCT05487599 -
A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)
|
Phase 1/Phase 2 | |
Recruiting |
NCT05324943 -
A Gene Therapy Study in Patients With Gaucher Disease Type 1
|
Phase 1 |