The Effect of Diflunisal on Familial Transthyretin Amyloidosis
- Conditions
- Amyloidosis
- Registration Number
- NCT01432587
- Lead Sponsor
- Umeå University
- Brief 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 55
- Biopsy and genetically proven systemic transthyretin amyloidosis caused by a TTR gene mutation. The amyloid shall be proven to be of transthyretin type, and the fibril composition settled.
- Age ≥ 18 years.
- Negative pregnancy test and contraception for sexually active women of child bearing potential.
- Concomitant use of non-study non-steroidal anti-inflammatory drugs (NSAIDs)
- Heart failure with symptoms at daily activities (NYHA class ≥III)
- Renal insufficiency (creatinine clearance < 30 ml calculated from the Cockcroft-Gault formula)
- Active non-haemorrhoidal bleeding within the last 18 month.
- Non-treated peptic ulcer disease.
- Anticoagulation therapy, low dose ASA permitted.
- Non-steroidal or aspirin allergy/hypersensitivity
- Thrombocytopenia (< 100,000 platelets/mm3)
- Inability or unwillingness of subject to give written informed consent
- By the investigator regarded as unable to follow the study guidelines and scheduled controls.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Changes in the Kumamoto scale 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.
- Secondary Outcome Measures
Name Time Method Changes in modified body mass index (mBMI) 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 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 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 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.
Trial Locations
- Locations (3)
Dept of Clinical Medicin, Ptieå Hospital
🇸🇪Piteå, Sweden
Dept of Clinical Medicine, Umeå University Hospital
🇸🇪Umeå, Sweden
Dept of clinical medicin, Skellefteå Hospital
🇸🇪Skellefteå, Sweden