A Study of Genz-112638 in Patients With Gaucher Disease(ENGAGE)
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); Placebo (Drug)
Phase: Phase 3
Status: Recruiting
Sponsored by: Genzyme Official(s) and/or principal investigator(s):
Medical Monitor, Study Director, Affiliation: Genzyme
Overall contact:
Genzyme 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
Clinical Details
Official title: A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Confirming the Efficacy and Safety of Genz-112638 in Patients With Gaucher Disease Type 1
Study design: Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Parallel Assignment, Safety/Efficacy Study
Primary outcome: The Primary objective is to compare the effects of Genz-112638 as compared to placebo in patients with Type 1 Gaucher Disease
Secondary outcome: The secondary objectives for this study include an evaluation of safety and the effects on disease manifestations and disease specific bio-markers
Detailed description:
Gaucher disease is characterised by lysosomal accumulation of glucosylceramide due to
impaired glucosylceramide hydrolysis. Type 1 Gaucher Disease, the most common form accounts
for > 90% of cases and does not involve the CNS. Typical manifestations of type 1 Gaucher
Disease include splenomegaly, hepatomegaly, thrombocytopenia, anemia, skeletal pathology and
decreased quality of life. The disease manifestations are caused by the accumulations of
glucosylceramide (storage material) in Gaucher cells which have infiltrated the spleen and
liver as well as other tissue. Genz-112638 is a small molecule 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 (> 16 years) with Gaucher Disease type 1
Eligibility
Minimum age: 16 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 16 to 65 years old at the time of randomization.
- The patient has a confirmed diagnosis of Gaucher disease type 1.
- 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.
- The patient has received pharmacological chaperones or miglustat within 6 months
prior to randomization
- The patient has received enzyme replacement therapy within 12 months prior to
randomization.
- The patient has type 2 or 3 Gaucher Disease or is suspected of having type 3 Gaucher
Disease
- 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 illness that may confound
the study results, or, on the opinion of the investigator, may preclude participation
in the study.
- Tha 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
Genzyme Medical Information, Phone: 800-745-4447, Email: medinfo@genzyme.com
Emory Genetics, Decatur, Georgia; Recruiting
Jaslok Hospital and Research Centre, Mumbai, India; Recruiting
Christian Medical College Hospital, Vellore, India; Recruiting
C/O Northwest Oncology Hematology Associates PA, Coral Springs, Florida, United States; Recruiting
University of Kansas Medical Center, Kansas City, Kansas, United States; Recruiting
Additional Information
Starting date: October 2009
Ending date: December 2011
Last updated: October 7, 2009
|
