Familial transthyretin amyloidosis MedDRA version: 13.1 Level: LLT Classification code 10019893 Term: Hereditary neuropathic amyloidosis, Swedish type System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: DIFLUNISAL
CAS Number: 22494-42-4
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 250-
Sponsors
Umea University
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Biopsy and genetically proven systemic transthyretin amyloidosis caused by a TTR gene mutation. The amyloid shall be proven to be of tranthyrein type, and the fibril composition settled. 2. Age = 18 years. 3. Negative pregnancy test 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 10 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 30
Exclusion criteria
Exclusion criteria: 1. Concomitant use of non-study NSAIDs 2. Heart failure with symptoms at daily activities (NYHA class =III) 3. Renal insufficiency (kreatinine clearence < 30 ml calculated from the Cocroft-Gault formula) 4. Active non-haemorrhoidal bleeding within the last 18 month. 5. Non-treated peptic ulcer disease. 6. Anticoagulation therapy, low dose ASA permitted. 7. Non-steroidal or aspirin allergy/hypersensitivity 8. Thrombocytopenia (< 100,000 platelets/mm3) 9. Inability or unwillingness of subject to give written informed consent 10. By the investigator regarded as unable to follow the study guidelines and scheduled controls.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To follow the development of neurological, nutritional and cardiac manifestations of transthyretin amyloidosis in patients treated by Diflunisal 250 mg twice daily.;Secondary Objective: collect and report adverse reactions;Primary end point(s): Changes in the Kumamoto scale;Timepoint(s) of evaluation of this end point: After 12 and 18 month treatment. Dependent of when results from the ongoing international study can be presented a 24 month evaluation can be performed in this study. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. changes in nutritional status measured by the modified body mass index (mBMI) 2. neurological impairment measured by the PND-score 3. cardiac impairment measured by echocardiographic measurement of septal thickness and by proBNP in blood samples. ;Timepoint(s) of evaluation of this end point: After 12 and 18 month of treatment. Dependent of when results from the ongoing international study can be presented a 24 month evaluation can be performed in this study. | — |
Countries
Sweden
Outcome results
None listed