Methods of treating huntington's disease using cysteamine compositions

A technology for Huntington's disease and cysteamine, applied in drug combinations, pharmaceutical formulations, nervous system diseases, etc., can solve problems such as inability to improve cognition and slow decline in motor function

Inactive Publication Date: 2017-08-29
HORIZON ORPHAN LLC
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

Tetrabenazine is the only drug approved for the treatment of HD in North America and some European countries, and tetrabenazine treats chorea associated with HD but does not improve cognition, slow motor decline, or show an effect on functional assessment ( 7)

Method used

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  • Methods of treating huntington's disease using cysteamine compositions
  • Methods of treating huntington's disease using cysteamine compositions
  • Methods of treating huntington's disease using cysteamine compositions

Examples

Experimental program
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Effect test

example 1

[0143] Clinical Trial Protocol

[0144] Research institute

[0145] In this double-blind, placebo-controlled, multicenter trial, the efficacy, safety, and tolerability of enteric-coated cysteamine in modulating the progression of HD as measured by the Unified Huntington's Disease Rating Scale ( Changes in the Total Motor Score (TMS) of the UHCRS). Patients were recruited from nine departments of neurology and genetics throughout France. Protocol and informed consent were approved by an institutional review board according to French law.

[0146] participant

[0147] Male and female HD patients aged 18 to 65 with CAG repeats >38 in the HTT gene were recruited. Inclusion criteria were minimum scores on both components of the Unified Huntington's Disease Rating Scale (UHDRS) (18): Total Motor Score (TMS) >5 and Total Functional Capacity (TFC) >10. The UHDRS is a validated scale for assessing clinical performance and abilities in four domains: motor function, cognitive function...

example 2

[0163] Twice-daily cysteamine improves motor scores in HD patients

[0164] From October 2010 to June 2012, a total of 96 subjects were randomized to treatment and constituted the ITT cohort. The baseline characteristics of the ITT and NoTBZ populations are shown in Table 1.

[0165] Table 1:

[0166]

[0167]

[0168] Efficacy on TMS

[0169] The ITT analysis of all 96 patients enrolled in the trial showed a slow positive trend toward progression to TMS (the primary endpoint of the study) for patients who received placebo and those who received RP103. The change in the primary endpoint from baseline to 18 months in TMS was 6.68 ± 7.98 for placebo and 4.55 ± 8.24 for RP103. Through the primary analysis method, the difference between the groups was 1.593±1.709, which was not statistically significant (95%CI[-5.000; 1.815]; p=0.3545). Supportive analyzes showed a between-group difference of 2.33±1.72 (95% CI [-5.750; 1.085]; p=0.1785) in the ITT population and 2.20±1.7...

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Abstract

The present disclosure relates in general to methods for the treatment of neurodegenerative disease, such as Huntington's Disease, using compositions comprising cysteamine or cystamine or salts or derivatives thereof.

Description

[0001] This application claims the benefit of priority to US Provisional Patent Application No. 62 / 075,536, filed November 5, 2014, which is incorporated herein by reference. technical field [0002] The present disclosure generally relates to methods of treating neurodegenerative diseases, such as Huntington's disease, using compositions comprising cysteamine or cystamine or salts or derivatives thereof. Background of the invention [0003] Treatment strategies for Huntington's disease (HD), an adult-onset neurodegenerative disorder, have helped alleviate some of the symptoms of HD, but still do not truly treat the disease effectively. HD is an autosomal dominant genetic disorder with an incidence of about 5-10 per 100,000 in the Caucasian population. Clinical signs include chorea and behavioral disturbances, but the disease is most troublesomely characterized by chronically progressive motor dysfunction and impaired cognitive function (1). Lesions in HD are characterized ...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): A61P25/28A61K31/145A61K31/13
CPCA61K31/145A61K31/4704A61K45/06A61P25/28A61K2300/00
Inventor P·里乌克斯
Owner HORIZON ORPHAN LLC
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