TTR amyloidosis (wild type or hereditary) MedDRA version: 20.0 Level: PT Classification code 10007509 Term: Cardiac amyloidosis System Organ Class: 10007541 - Cardiac disorders
Conditions
Interventions
Trade Name: Vyndaqel
Pharmaceutical Form: Capsule, soft
INN or Proposed INN: Tafamidis
CAS Number: 594839-88-0
Other descriptive name: 2-(3,5-dichlorophenyl)-1,3-benzoxazole-6-carboxylic acid
Concentr
Sponsors
Centro Hospitalar de Trás-os-Montes e Alto Douro (CHTMAD), EPE
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Established diagnosis of TTR amyloidosis (wild type or hereditary) in accordance with institutional/site standard of care (SoC): - Diphosphonate scintigraphy with 99mTc-labelled radiotracers (grade 2 or 3 uptake) AND cardiac involvement confirmed by means of echocardiography, with an end-diastolic interventricular septal wall thickness exceeding 12 mm AND no evidence of a monoclonal protein by negative serum free light chain and negative serum and urine immunofixation OR - Extracardiac biopsy positive for ATTR amyloidosis AND cardiac involvement confirmed by means of echocardiography, with an enddiastolic interventricular septal wall thickness exceeding 12 mm OR - Cardiac biopsy positive for ATTR amyloidosis AND • Medical history of heart failure - NYHA I-III • Age > 18 years and =65 years) yes F.1.3.1 Number of subjects for this age range 25
Exclusion criteria
Exclusion criteria: • Confirmed diagnosis of light-chain amyloidosis • Familial amyloid polyneuropathy • NYHA IV • Heart failure due to other condition than CA in the opinion of the investigator • History of liver or cardiac transplant • Previous treatment with tafamidis or other alternatives in transthyretin amyloidosis • Severe malnutrition • Implanted cardiac device – pacemaker, defibrillator, cardiac resynchronization therapy • Devices or other material non-MRI conditional • Liver transaminases > 2 upper normal limit • Participating in another investigational study • Subjects requiring treatment with calcium channel blockers or digitalis • Subjects using non-steroidal anti-inflammatory drugs (NSAIDS), tauroursodeoxycholate and doxycycline • Investigator determines that the subject is not suitable for study participation for any other reason • Subjects who are pregnant females; breastfeeding females; males and females of childbearing potential who are unwilling or unable to use a highly effective method of contraception for the duration of the study and for at least 28 days, after last dose of tafamidis
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the evolution and correlation of CMR imaging parameters before and after treatment with tafamidis for 12 months.;Secondary Objective: To identify clinical, analytical and imaging predictors of treatment response. To assess the association of imaging markers evolution with mortality and heart failure related hospitalizations. To assess the association of imaging markers evolution with functional capacity and quality of life.;Primary end point(s): Change from baseline at each point in CMR imaging parameters, including: ventricular volumes, mass, ejection fraction; native T1 and T2 mapping; ECV; Look-Locker at 2, 5 and 10 minutes; LGE, Strain analysis and 4D flow;;Timepoint(s) of evaluation of this end point: Change from baseline | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Frequency of heart failure related hospitalization and all-cause mortality Change from baseline at each point in the Kansas City Cardiomyopathy Questionnaire overall score Change from baseline at each point in 6MWT distance Change from baseline at each point in NT-proBNP concentration Change from baseline at each point in NYHA classification Change from baseline at each point in echocardiographic parameters ;Timepoint(s) of evaluation of this end point: Change from baseline | — |
Countries
Portugal
Contacts
Public ContactCatarina Ferreira
Centro Hospitalar de Trás-os-Montes e Alto Douro (CHTMAD), EPE
Outcome results
None listed