Clinical proof of concept study to evaluate SOM0226 efficacy in TTR Amyloidosis.
This is an open label interventional Phase IIa proof of concept clinical trial designed in two phases separated by a washout period of 6 weeks (± 2 weeks) , to evaluate the TTR stabilization activity of SOM0226 in healthy volunteers (wild type), TTR-FAP patients and asymptomatic carriers. * Phase A (24 hours): SOM0226 single dose * Phase B (32 hours): SOM0226 multiple dose
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
17
Oral
Hospital Vall d'Hebron
Barcelona, Catalonia, Spain
TTR stabilization
TTR stabilization will be measured by determining the concentration of tetrameric TTR after denaturation. The ratio of TTR tetramer after denaturation / TTR tetramer before denaturation of plasma samples from treated subjects will be compared to their baseline ratio. TTR stabilization will be determined as percentage of the baseline ratio.
Time frame: 24 hours and 32 hours
Pharmacodynamics assessment
Determine the minimal molar ratio SOM0226:TTR that must be reached in plasma to confer maximal TTR stabilization
Time frame: 24 hours and 32 hours
Safety
Drug safety will be assessed by blood biochemistry and hematology analysis, patient exploration and adverse event reporting
Time frame: 24 hours
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