Clinical Trial: Study of SOM0226 in Familial Amyloid Polyneuropathy
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: Study of SOM0226 in Familial Amyloid Polyneuropathy (FAP) Patients and Asymptomatic Carriers to Evaluate Protein Stabilization Activity
Brief Summary: Clinical proof of concept study to evaluate SOM0226 efficacy in TTR Amyloidosis.
Detailed Summary:
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
Sponsor: SOM Biotech SL
Current Primary Outcome: TTR stabilization [ Time Frame: 24 hours and 32 hours ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Pharmacodynamics assessment [ Time Frame: 24 hours and 32 hours ]Determine the minimal molar ratio SOM0226:TTR that must be reached in plasma to confer maximal TTR stabilization
- Safety [ Time Frame: 24 hours ]Drug safety will be assessed by blood biochemistry and hematology analysis, patient exploration and adverse event reporting
Original Secondary Outcome: Same as current
Information By: SOM Biotech SL
Dates:
Date Received: July 9, 2014
Date Started: July 2014
Date Completion:
Last Updated: November 29, 2016
Last Verified: November 2016