The Effect of Diflunisal on Familial Transthyretin Amyloidosis
Status:
Completed
Trial end date:
2014-12-01
Target enrollment:
Participant gender:
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.