The Effect of Diflunisal on Familial Transthyretin Amyloidosis (DFNS01)
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|ClinicalTrials.gov Identifier: NCT01432587|
Recruitment Status : Completed
First Posted : September 13, 2011
Last Update Posted : August 24, 2015
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.
|Condition or disease||Intervention/treatment|
|Study Type :||Observational|
|Actual Enrollment :||55 participants|
|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.|
|Study Start Date :||August 2011|
|Actual Primary Completion Date :||December 2014|
|Actual Study Completion Date :||December 2014|
- Drug: Diflunisal
Film-coated tablet, 250 mg twice daily, orally for approximately 2 years
- 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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|