The purpose of this study is to determine if diflunisal can prevent progressive lower leg
nerve damage in patients with familial amyloidosis polyneuropathy.
Funding Source - FDA Office of Orphan Products Development (OOPD); National Institute of
Neurological Disorders and Stroke (NINDS)
National Institute of Neurological Disorders and Stroke (NINDS)
Phase 2/Phase 3
2006-02-01
The purpose of this study is to determine if diflunisal can prevent progressive lower leg
nerve damage in patients with familial amyloidosis polyneuropathy.
Funding Source - FDA Office of Orphan Products Development (OOPD); National Institute of
Neurological Disorders and Stroke (NINDS)
The purpose of this study is to determine if diflunisal can prevent progressive lower leg
nerve damage in patients with familial amyloidosis polyneuropathy.
Funding Source - FDA Office of Orphan Products Development (OOPD); National Institute of
Neurological Disorders and Stroke (NINDS)
The Effect of Diflunisal on Familial Transthyretin Amyloidosis
Completed
Umeå University
2011-08-01
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.
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