The Effect of Diflunisal on Familial Amyloidosis

• Status: Completed
• Trial end date: 2012-12-01
• Target enrollment: 0
• Participant gender: All

Summary

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)

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Boston University
• Collaborators: Food and Drug Administration (FDA); National Institute of Neurological Disorders and Stroke (NINDS)
• Treatments: Diflunisal

Criteria

Inclusion Criteria:

• Age 18 to 75 years

• Biopsy proven amyloidosis

• Genotyping of variant transthyretin

• Signs of peripheral or autonomic neuropathy

Exclusion Criteria:

• Use of other non-steroidal anti-inflammatory drugs

• Other causes of sensorimotor polyneuropathy

• Anticipated survival <2 years or liver transplantation in <1 yr

• Liver transplantation

• Profound nerve, heart or kidney impairment

• Pregnancy or unwillingness to use contraception by women of childbearing age

• Active or recent gastrointestinal bleeding

• Non-steroidal or aspirin drug allergy/hypersensitivity