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The Effect of Diflunisal on Familial Transthyretin Amyloidosis — DFNS01  

Amyloidosis Clinical Trial

Official title:
The Effect of Diflunisal on Familial Transthyretin Amyloidosis: An Open Label Extension Study of "the Diflunisal Trial" (IND 68092), and an Open Label Observational Study on Previously Untreated Patients With Familial Transthyretin Amyloidosis.

Clinical Trial Summary

An ongoing trial of diflunisal has been closed for enrollment, thus, patients suitable for the study can no longer participate or receive treatment by diflunisal; and patients, who have participated in the trial can not continue their treatment. The investigators want to continue to monitor the effect of the drug on transthyretin (TTR) amyloidosis in an open label observational study.

Primary endpoint will be a composite score of the manifestations of the disease (Kumamoto scale) and secondary end points will be measurements of neurological impairment, heart involvement and nutritional status.

Clinical Trial Description

Duration of treatment in this study is dependent of the results from the ongoing IND 68092-study, which are planned to be presented 2013. ;

Study Design

Observational Model: Cohort, Time Perspective: Prospective

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT01432587
Study type Observational
Source Umeå University
Contact
Status Completed
Phase N/A
Start date August 2011
Completion date December 2014
View Details
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