A Study of Genz-112638 in Patients With Gaucher Disease Who Have Been Stabilized on Cerezyme
Information source: Genzyme
Information obtained from ClinicalTrials.gov on October 19, 2009
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Gaucher Disease, Type 1
Intervention: Genz-112638 (Drug); Cerezyme (Biological)
Phase: Phase 3
Status: Recruiting
Sponsored by: Genzyme Official(s) and/or principal investigator(s):
Medical Monitor, Study Director, Affiliation: Genzyme
Overall contact:
Medical Information, Phone: 800-745-4447, Email: medinfo@genzyme.com
Summary
This Phase 3 study was designed to confirm the efficacy and safety of Genz-112638 in
patients with Gaucher disease type 1 who have been stabilized on Cerezyme
Clinical Details
Official title: A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients With Gaucher Disease Type 1 Who Have Been Stabilized With Cerezyme
Study design: Treatment, Randomized, Open Label, Parallel Assignment, Safety/Efficacy Study
Primary outcome: The primary objective of this study in patients with Gaucher disease type 1 who have been stabilized with Cerezyme is to demonstrate that, the majority of patients who receive Genz-112638 remain stable.
Secondary outcome: The secondary objective is to assess the stability rates and safety of patients treated with Genz-112638 to Cerezyme.
Detailed description:
Gaucher disease is characterized by lysosomal accumulation of glucosylceramide due to
impaired glucosylceramide hydrolysis. Gaucher disease type 1, which is the most common form,
accounts for >90% of cases and does not involve the CNS. Typical manifestations of Gaucher
disease type 1 include splenomegaly, hepatomegaly, thrombocytopenia, anemia, bone disease,
and decreased quality of life. The disease manifestations are caused by the accumulation of
glucosylceramide (storage material) in macrophages (called Gaucher cells) which have
infiltrated the spleen and liver as well as other tissues.
Genz-112638 is a small molecule drug developed as an oral therapy which acts to specifically
inhibit production of this storage material in Gaucher cells.
This study is designed to determine the efficacy, safety, and pharmacokinetics (PK) of
Genz-112638 in adult patients with Gaucher disease type 1 who have been stabilized on
Cerezyme.
Eligibility
Minimum age: 18 Years.
Maximum age: 65 Years.
Gender(s): Both.
Criteria:
Inclusion Criteria:
- The patient (and/or their parent/legal guardian) is willing and able to provide
signed informed consent prior to any study-related procedures to be performed.
- The patient is 18 to 65 years old at the time of randomization.
- The patient has a confirmed diagnosis of Gaucher disease type 1.
- The patient has received treatment with Cerezyme for at least 3 years and has
received the equivalent of ≥ 20 U/kg to ≤ 60 U/kg (± 5 U/kg) q2w at a stable dose.
- The patient has clinically stable Gaucher disease prior to randomization.
- Male patients agree to use a medically accepted method of contraception throughout
the study.
- Female patients of childbearing potential must have a documented negative pregnancy
test prior to dosing. In addition, all female patients of childbearing potential must
use a medically accepted form of contraception throughout the study.
Exclusion Criteria:
- The patient has had a partial or total splenectomy within 3 years prior to
randomization.
- The patient has received pharmacological chaperones or miglustat within 6 months
prior to randomization.
- The patient has Gaucher disease type 2 or 3 or is suspected of having Gaucher disease
type 3.
- The patient has any clinically significant disease, other than Gaucher
disease,including cardiovascular, renal, hepatic, gastrointestinal (GI),
pulmonary,neurologic, endocrine, metabolic (e. g. hypokalemia, hypomagnesemia), or
psychiatric disease, other medical conditions, or serious intercurrent illnesses that
may confound the study results or, in the opinion of the Investigator, may preclude
participation in the study.
- The patient has tested positive for the human immunodeficiency virus (HIV) antibody,
Hepatitis C antibody, or Hepatitis B surface antigen.
- The patient has received an investigational product within 30 days prior to
randomization.
- The patient is pregnant or lactating.
Locations and Contacts
Medical Information, Phone: 800-745-4447, Email: medinfo@genzyme.com
Hospital de Pediatria Ricardo Gutierrez, Buenos Aires, Argentina; Not yet recruiting
IGEIM, Sao Paulo 04020-041, Brazil; Not yet recruiting
Santa Casa de Misericordia de SP, Sao Paulo 01221-001, Brazil; Not yet recruiting
Hemorio, Rio de Janeiro 20211-030, Brazil; Not yet recruiting
National Centre for Treatment of Gaucher Disease, Praha 2 12109, Czech Republic; Not yet recruiting
Hopital Beaujon, Clichy 92110, France; Not yet recruiting
Universitätsklinikum Düsseldorf, Klinik für Gastroenterologie Moorenstrasse 5, Dusseldorf 40225, Germany; Not yet recruiting
Katholische Kliniken Oberhausen, St Josef-Hospital, Oberhausen 46045, Germany; Not yet recruiting
Universitaria Careggi, Firenze 50139, Italy; Not yet recruiting
Academic Medical Center, Dept of Internal Medicine, Amsterdam 1105 AZ, Netherlands; Not yet recruiting
Hematology Research Center of Russian Academy of Sciences, Moscow 125167, Russian Federation; Not yet recruiting
Hospital University Meguel Servet, Zaragoza 50009, Spain; Not yet recruiting
Royal Free Hospital, London NW3 2QG, United Kingdom; Not yet recruiting
Addenbrookes Hospital, Cambridge CB2 2QQ, United Kingdom; Not yet recruiting
Tower Hematology Oncology Medical Group, Beverly Hills, California, United States; Recruiting
UCSF MS Center, San Francisco, California, United States; Not yet recruiting
Northwest Oncology Hematology Associates PA, Coral Springs, Florida, United States; Recruiting
Emory University Medical Genetics, Decatur, Georgia, United States; Not yet recruiting
Mount Sinai School of Medicine, New York, New York, United States; Recruiting
Duke University Medical Center, Durham, North Carolina, United States; Recruiting
Monash Medical Center Dept of Hematology, Clayton, Victoria 3168, Australia; Not yet recruiting
Additional Information
Starting date: August 2009
Ending date: December 2011
Last updated: September 17, 2009
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