A randomized, double-blind, placebo-controlled, international multi-center trial ofdiflunisal on neurologic disease progression in 200 familial amyloid subjects - The effect of diflunisal on familial amyloidosis

• Conditions: Hereditary neuropathic amyloidosis; MedDRA version: 11.Level: LLTClassification code 10019889Term: Hereditary neuropathic amyloidosis

• Registration Number: EUCTR2006-001066-16-GB
• Lead Sponsor: John L. Berk

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2010-06-18
• Last Update Posted: 19 March 2012

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 140

Inclusion Criteria

1. Biopsy proven amyloid deposition
2. Genotyping of variant TTR
3. Signs of peripheral or autonomic neuropathy -- detectable by a
neurologist (performance status = 3)
4. Age = 18 and = 75 years
5. Negative ßHCG testing and contraception for sexually active women of child-bearing potential
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Use of non-study NSAIDs after enrollment (low dose aspirin allowed)
2. Other causes of sensorimotor polyneuropathy
a. Vitamin B12 deficiency
b. HIV patients on anti-retroviral drugs
c. Diabetes mellitus (Hgb A1C > 6.2%)
d. Chronic alcoholism (> 6 ounces hard liquor daily for 10 or greater years)
3. Co-morbidities with anticipated survival <2 years or liver transplantation in <1 yr
4. Liver transplantation
5. End-stage neuropathic disease (performance status > 3, parenteral nutrition, bedsores)
6. NYHA class IV (cardiac symptoms at rest & with minimal exertion)
7. Pregnancy or unwillingness to use contraception by women of childbearing age
8. Renal insufficiency (creatinine clearance < 30 ml/min)
9. Active or recent non-hemorrhoidal GI bleeding (within past 18 months)
10. Current anti-coagulation therapy, non-study NSAID or aspirin use
11. AST, ALT or Total Bilirubin > twice the upper limit of normal lab value
12. Non-steroidal or aspirin allergy/hypersensitivity
13. Thrombocytopenia (< 100,000 platelets/mm3)
14. Previous participation in this study
15. Inability or unwillingness of subject or legal guardian/representative to give written informed consent

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified