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A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing

2014-08-27 03:15:41 | BioPortfolio

Summary

The primary objective of this study is to evaluate the efficacy and safety of once daily (QD) versus twice daily (BID) dosing of eliglustat tartrate (Genz-112638) in patients with Gaucher disease type 1 who have demonstrated clinical stability on BID dosing of eliglustat tartrate (Genz-112638). The secondary objective is to evaluate the pharmacokinetics (PK) of Genz-99067 when eliglustat tartrate (Genz-112638) is administered QD and BID in patients with Gaucher disease type 1 who have demonstrated clinical stability on BID dosing of eliglustat tartrate (Genz-112638).

Description

NOTE: Other Phase 3 studies being conducted with eliglustat tartrate (Genz-112638) are GZGD02507(ENGAGE): NCT00891202 and GZGD02607(ENCORE): NCT00943111

Study Design

Allocation: Randomized, Control: Dose Comparison, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Conditions

Gaucher Disease

Intervention

eliglustat tartrate

Location

University of California, San Diego Medical Center
San Diego
California
United States

Status

Recruiting

Source

Genzyme

Results (where available)

View Results

Links

Published on BioPortfolio: 2014-08-27T03:15:41-0400

Clinical Trials [132 Associated Clinical Trials listed on BioPortfolio]

A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients

Gaucher disease is a genetic disease that results in a deficiency of an enzyme acid β-glucosidase, also known as glucocerebrosidase. This enzyme is needed to digest a substrate (lipid) c...

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease(ENGAGE)

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

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)

This Phase 3 study was designed to confirm the efficacy and safety of eliglustat tartrate (Genz-112638) in patients with Gaucher disease type 1 who have reached therapeutic goals with enzy...

A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate

Primary Objective: To study the effect of mild and moderate hepatic impairment on the pharmacokinetics (PK) of eliglustat. Secondary Objective: To assess the tolerability of...

A Study of the Effects of Renal Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate

Primary Objective: To study the effect of mild, moderate, and severe renal impairment on the pharmacokinetics (PK) of eliglustat. Secondary Objective: To assess the tolerabi...

PubMed Articles [14486 Associated PubMed Articles listed on BioPortfolio]

Progressive pulmonary hypertension in a patient with type 1 Gaucher disease.

Gaucher disease is the most common form of hereditary enzymopathies combined into a group of lysosomal storage diseases. The basis for the disease is a hereditary deficiency of the activity of acid β...

Four Gaucher disease type II patients with three novel mutations: a single centre experience from Turkey.

Gaucher disease is the most common lysosomal storage disorder due to glucosylceramidase enzyme deficiency. There are three subtypes of the disease. Neurological involvement accompanies visceral and ha...

Combined miglustat and enzyme replacement therapy in two patients with type 1 Gaucher disease: two case reports.

Intravenous enzyme replacement therapy is a first-line therapy for Gaucher disease type 1, and substrate reduction therapy represents an oral treatment alternative. Both enzyme replacement therapy and...

Involvement of hepcidin in iron metabolism dysregulation in Gaucher disease.

Gaucher disease is an inherited deficiency of glucocerebrosidase leading to accumulation of glucosylceramide in tissues such as the spleen, liver, and bone marrow. The resulting lipid-laden macrophage...

Gaucher's disease in a patient presenting with hip and abdominal pain.

Gaucher's disease is characterized by glucocerebroside accumulation in the cells of the reticuloendothelial system. There are three subtypes. The most common is type 1, known as the non-neuropathic fo...

Medical and Biotech [MESH] Definitions

One of several acid phosphatases in humans, other mammals, plants, and a few prokaryotes. The protein fold of tartrate-resistant acid phosphatase (TRAP) resembles that of the catalytic domain of plant purple acid phosphatase and other serine/threonine-protein phosphatases that also contain a metallophosphoesterase domain. One gene produces the various forms which include purple acid phosphatases from spleen and other tissues. Tartrate-resistant acid phosphatase is a biomarker for pathological states in which it is over-expressed. Such conditions include GAUCHER DISEASE; HODGKIN DISEASE; BONE RESORPTION; and NEOPLASM METASTASIS.

An autosomal recessive disorder caused by a deficiency of acid beta-glucosidase (GLUCOSYLCERAMIDASE) leading to intralysosomal accumulation of glycosylceramide mainly in cells of the MONONUCLEAR PHAGOCYTE SYSTEM. The characteristic Gaucher cells, glycosphingolipid-filled HISTIOCYTES, displace normal cells in BONE MARROW and visceral organs causing skeletal deterioration, hepatosplenomegaly, and organ dysfunction. There are several subtypes based on the presence and severity of neurological involvement.

A pharmaceutical preparation of brimonidine tartrate and timolol maleate. The combined ADRENERGIC ALPHA2 RECEPTOR AGONIST and ADRENERGIC BETA-ANTAGONIST activity of these drugs reduce INTRAOCULAR PRESSURE in GLAUCOMA patients.

A glycosidase that hydrolyzes a glucosylceramide to yield free ceramide plus glucose. Deficiency of this enzyme leads to abnormally high concentrations of glucosylceramide in the brain in GAUCHER DISEASE. EC 3.2.1.45.

Cerebrosides which contain as their polar head group a glucose moiety bound in glycosidic linkage to the hydroxyl group of ceramides. Their accumulation in tissue, due to a defect in beta-glucosidase, is the cause of Gaucher's disease.

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