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Evaluate the safety and efficacy of ammonium tetrathiomolybdate alone and compared with trientine therapy as initial treatment in patients with Wilson disease presenting neurologically.
PROTOCOL OUTLINE: This a double blind, randomized study. Patients are randomized into one of two treatment arms.
Arm I: Patients receive tetrathiomolybdate (TM) 3 times a day with meals and 3 times a day between meals for 8 weeks in the absence of neurologic deterioration or unacceptable toxicity.
Arm II: Patients receive trientine therapy for 8 weeks in the absence of neurologic deterioration and unacceptable toxicity.
Additional therapy (off study): Patients in the TM group may receive maintenance zinc, while those in the trientine group may continue on trientine or switch to zinc.
Allocation: Randomized, Masking: Double-Blind, Primary Purpose: Treatment
University of Michigan
National Center for Research Resources (NCRR)
Published on BioPortfolio: 2010-07-15T17:00:00-0400
This is a retrospective study to assess the clinical efficacy and safety of trientine in Wilson's disease patients
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Wilson Disease (WD) is an autosomal recessive disorder of impaired copper (CU) transport caused by mutations in the ATP7B gene. WTX101 (bis-choline tetrathiomolybdate) is a first-in-class ...
This is a 24-month study to assess copper (Cu) parameters in subjects with Wilson Disease treated with standard of care (SOC) medications. Data will be collected during routinely scheduled...
Dysregulation of copper homeostasis in humans is primarily found in two genetic diseases of copper transport, Menkes and Wilson diseases, which show symptoms of copper deficiency or overload, respecti...
Since the seminal experience of Starzl et al (1), liver transplant (LT) has been performed for Wilson's disease patients with failing livers and more controversially for neurologic disease. The major...
Wilson's disease (WD), or hepatolenticular degeneration, is an autosomal recessive disorder with a prevalence of 1:50,000 to 1:100,000 live births.
Wilson disease is an inherited disorder of copper metabolism that leads to copper accumulation and toxicity in liver and brain. It is caused by mutations in the ATPase copper transporting beta gene (A...
Wilson's disease, also known as hepatolenticular degeneration, is an autosomal recessive genetic disorder due to a mutation of the ATP7B gene resulting in impaired hepatic copper excretion and copper ...
3-Mercapto-D-valine. The most characteristic degradation product of the penicillin antibiotics. It is used as an antirheumatic and as a chelating agent in Wilson's disease.
A rare autosomal recessive disease characterized by the deposition of copper in the BRAIN; LIVER; CORNEA; and other organs. It is caused by defects in the ATP7B gene encoding copper-transporting ATPase 2 (EC 22.214.171.124), also known as the Wilson disease protein. The overload of copper inevitably leads to progressive liver and neurological dysfunction such as LIVER CIRRHOSIS; TREMOR; ATAXIA and intellectual deterioration. Hepatic dysfunction may precede neurologic dysfunction by several years.
A cinnamon-colored strain of Long-Evans rats which carries a mutation causing fulminant hepatitis and jaundice, with an associated gross accumulation of copper in the liver. This strain is a model for Wilson's Disease (see HEPATOLENTICULAR DEGENERATION).
An ethylenediamine derivative used as stabilizer for EPOXY RESINS, as ampholyte for ISOELECTRIC FOCUSING and as chelating agent for copper in HEPATOLENTICULAR DEGENERATION.
An outbred strain of rats developed in 1915 by crossing several Wistar Institute white females with a wild gray male. Inbred strains have been derived from this original outbred strain, including Long-Evans cinnamon rats (RATS, INBRED LEC) and Otsuka-Long-Evans-Tokushima Fatty rats (RATS, INBRED OLETF), which are models for Wilson's disease and non-insulin dependent diabetes mellitus, respectively.