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 ]

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.


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