Phase 3
Completed N=130
The Effect of Diflunisal on Familial Amyloidosis
Familial Amyloid Polyneuropathy · Familial Amyloidosis
Source: ClinicalTrials.gov NCT00294671 ↗
Enrolled (actual)
130
Serious AEs
5.4%
Results posted
Mar 2017
Primary outcomePrimary: Neurologic Impairment Score + 7 (NIS+7) — 8.2; 26.3; 6.2; 12.5 units on a scale — p=<0.001
◆ Published Evidence
Highly cited
702citations · ~54 / year
Repurposing diflunisal for familial amyloid polyneuropathy: a randomized clinical trial.
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)
Linked Publications
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Repurposing diflunisal for familial amyloid polyneuropathy: a randomized clinical trial.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Neurologic Impairment Score + 7 (NIS+7) |
8.2; 26.3; 6.2; 12.5 | <0.001 sig |
| SECONDARY Kumamoto Neurologic Scale; |
3.1; 8.0; 1.9; 4.1 | 0.002 sig |
| SECONDARY Modified Body Mass Index (mBMI); |
-33.7; -67.9; -18.7; -38.5 | 0.21 |
| SECONDARY Quality of Life Questionnaire: SF-36 Physical Component Score |
1.2; -4.9; 0.7; -1.9 | 0.001 sig |
| SECONDARY Quality of Life Questionnaire: SF-36 Mental Component Score |
3.5; -0.9; 2.5; 0.8 | 0.06 |
Eligibility 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
Data sourced from ClinicalTrials.gov (NCT00294671) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.