The Effect of Diflunisal on Familial Transthyretin Amyloidosis (DFNS01)
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.
|Study Design:||Observational Model: Cohort
Time Perspective: Prospective
|Official Title:||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.|
- Changes in the Kumamoto scale [ Time Frame: Enrollment, 12 month and annual follow-up ]Composite score of the manifestations of the disease (Kumamoto Scale). Results at enrollment will be compared to results at 12 months and annual follow-ups.
- Changes in modified body mass index (mBMI) [ Time Frame: Enrollment, 12 month and annual follow-up ]Changes in nutritional status measured by mBMI.Results at enrollment will be compared to results at 12 months and annual follow-ups.
- Changes in paraneoplastic neurological disorders (PND) scale [ Time Frame: Enrollment, 12 month and annual follow-up ]Neurological impairment measured by the PND-score. Results at enrollment will be compared to results at 12 months and annual follow-ups.
- Changes in cardiac function [ Time Frame: Enrollment, 1 month, 2 month, 3 month, 6 month, 9 month 12 month, 18 month and annual follow-up ]Cardiac impairment is measure by echocardiographic measurement of septal thickness and by proBNP in blood samples. Results at enrollment will be compared to results during the study and annual follow-ups.
- Safety follow-up Blood Work [ Time Frame: 1 month, 3 month, 6 month, 9 month, 12 month and follow-up every 6 months ]To follow-up the patient safety during the study and follow-up the blood samples for (B-Hb), blood platelets, s-creatinine, liver enzymes [aspartate transaminase (ASAT),alanine aminotransferase (ALAT), s-bilirubin and alkaline phosphatase (ALP)],serum proBNP (S-proBNP) are drawn. Results at enrollment will be compares to results during study and every 6-month follow-ups.
Biospecimen Retention: Samples Without DNA
|Study Start Date:||August 2011|
|Study Completion Date:||December 2014|
|Primary Completion Date:||December 2014 (Final data collection date for primary outcome measure)|
Please refer to this study by its ClinicalTrials.gov identifier: NCT01432587
|Dept of Clinical Medicin, Ptieå Hospital|
|Piteå, Sweden, SE-941 28|
|Dept of clinical medicin, Skellefteå Hospital|
|Skellefteå, Sweden, SE-931 86|
|Dept of Clinical Medicine, Umeå University Hospital|
|Umeå, Sweden, SE-90185|
|Principal Investigator:||Ole B Suhr, MD PhD||Dept of Clinical Medicine and public Health, Umeå University|