Full Text View
Tabular View
No Study Results Posted
Related Studies
A Study of Genz-112638 in Patients With Gaucher Disease
This study is not yet open for participant recruitment.
Verified by Genzyme, April 2009
First Received: April 30, 2009   No Changes Posted
Sponsored by: Genzyme
Information provided by: Genzyme
ClinicalTrials.gov Identifier: NCT00891202
  Purpose

This Phase 3, Study was designed to confirm the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1


Condition Intervention Phase
Gaucher Disease Type 1
Drug: Genz-112638
Drug: Placebo
Phase III

Genetics Home Reference related topics: Chanarin-Dorfman syndrome cholesteryl ester storage disease Farber lipogranulomatosis Gaucher disease long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency mitochondrial trifunctional protein deficiency primary carnitine deficiency succinic semialdehyde dehydrogenase deficiency
MedlinePlus related topics: Gaucher's Disease
U.S. FDA Resources
Study Type: Interventional
Study Design: Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Parallel Assignment, Safety/Efficacy Study
Official Title: A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Conforming the Efficacy and Safety of Genz-112638 in Patients With Gaucher Disease Type 1

Further study details as provided by Genzyme:

Primary Outcome Measures:
  • The Primary objective is to compare the effects of Genz-112638 as compared to placebo in patients with Type 1 Gaucher Disease [ Time Frame: 39 weeks ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • The secondary objectives for this study include an evaluation of safety and the effects on disease manifestations and disease specific bio-markers [ Time Frame: 39 weeks ] [ Designated as safety issue: No ]

Estimated Enrollment: 36
Study Start Date: June 2009
Estimated Study Completion Date: December 2011
Estimated Primary Completion Date: September 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active: Experimental
Genz-112638
Drug: Genz-112638
Capsule: 50 or 100 mg BID
Placebo: Placebo Comparator
Placebo
Drug: Placebo
Capsule

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

Ages Eligible for Study:   16 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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 conformed diagnosis of Gaucher Disease type 1 confirmed
  • 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 chaperons 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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00891202

Contacts
Contact: Genzyme Medical Information 800-745-4447 medinfo@genzyme.com

Sponsors and Collaborators
Genzyme
Investigators
Study Director: Medical Monitor Genzyme
  More Information

No publications provided

Responsible Party: Genzyme Corporation ( Medical Monitor )
Study ID Numbers: GZGD02507
Study First Received: April 30, 2009
Last Updated: April 30, 2009
ClinicalTrials.gov Identifier: NCT00891202     History of Changes
Health Authority: United States: Food and Drug Administration

Keywords provided by Genzyme:
Gaucher, beta-glucosidase, acid ß-glucosidase, glucocerebrosidase, glucosylceramide, D-glucosyl-N-acylsphingosine glucohydrolase, substrate reduction therapy

Study placed in the following topic categories:
Lipid Metabolism, Inborn Errors
Sphingolipidoses
Metabolic Diseases
Gaucher Disease Type 1
Lysosomal Storage Diseases
Sphingolipidosis
Central Nervous System Diseases
Brain Diseases
Lymphatic Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Brain Diseases, Metabolic, Inborn
Lipidoses
Gaucher Disease
Metabolic Disorder
Lipid Metabolism Disorders
Brain Diseases, Metabolic

Additional relevant MeSH terms:
Lipid Metabolism, Inborn Errors
Sphingolipidoses
Metabolic Diseases
Reticuloendotheliosis
Lysosomal Storage Diseases, Nervous System
Lysosomal Storage Diseases
Nervous System Diseases
Central Nervous System Diseases
Brain Diseases
Lymphatic Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Brain Diseases, Metabolic, Inborn
Lipidoses
Gaucher Disease
Lipid Metabolism Disorders
Brain Diseases, Metabolic

ClinicalTrials.gov processed this record on May 07, 2009