Study of SOM0226 in Familial Amyloid Polyneuropathy

Familial Amyloid Polyneuropathy (FAP) Clinical Trial

Official title: Study of SOM0226 in Familial Amyloid Polyneuropathy (FAP) Patients and Asymptomatic Carriers to Evaluate Protein Stabilization Activity

Status Completed

Clinical Trial Summary

Clinical proof of concept study to evaluate SOM0226 efficacy in TTR Amyloidosis.

Clinical Trial Description

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 Design

Allocation: Non-Randomized, Endpoint Classification: Pharmacodynamics Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Amyloid Neuropathies
• Amyloid Neuropathies, Familial
• Amyloidosis
• Familial Amyloid Polyneuropathy (FAP)
• Polyneuropathies

• NCT number: NCT02191826
• Study type: Interventional
• Source: SOM Biotech SL
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: July 2014
• Completion date: November 2015