NCT01432587

The Effect of Diflunisal on Familial Transthyretin Amyloidosis

The Effect of Diflunisal on Familial Transthyretin Amyloidosis: An Open Label Extension Study of "the Diflunisal Trial" (IND 68092), and an Open Label Observational Study on Previously Untreated Patients With Familial Transthyretin Amyloidosis.

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 55 (actual)
• Sponsor: Umeå University · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Detailed description

Duration of treatment in this study is dependent of the results from the ongoing IND 68092-study, which are planned to be presented 2013.

Conditions

• Amyloidosis

Interventions

Type	Name	Description
DRUG	Diflunisal	Film-coated tablet, 250 mg twice daily, orally for approximately 2 years

Timeline

• Start date: 2011-08-01
• Primary completion: 2014-12-01
• Completion: 2014-12-01
• First posted: 2011-09-13
• Last updated: 2015-08-24

Locations

3 sites across 1 country: Sweden