Overview

Study of SOM0226 in Familial Amyloid Polyneuropathy

Status:
Completed
Trial end date:
2015-11-01
Target enrollment:
0
Participant gender:
All
Summary
Clinical proof of concept study to evaluate SOM0226 efficacy in TTR Amyloidosis.
Phase:
Phase 1/Phase 2
Accepts Healthy Volunteers?
Accepts Healthy Volunteers
Details
Lead Sponsor:
SOM Biotech SL
Collaborator:
Hospital Vall d'Hebron
Treatments:
Tolcapone
Criteria
Inclusion Criteria:

- Participant is willing and able to give informed consent for participation in the
study

- Male or Female, aged 18 years or above at the time of consent

- Two cohorts defined by TTR variant (wild type: healthy volunteers; TTR mutant:
asymptomatic carriers with documented mutation in TTR or diagnosed TTR-FAP patients
(stage 1 or 2) not undergoing pharmacological treatment with Vyndaqel

- Body Mass Index (BMI) > 17.5 kg/m2

- Female participants of child bearing potential and male participants whose partner is
of child bearing potential must be willing to ensure that they or their partner use
effective contraception during the study and for 2 month thereafter

- Able (in the Investigators opinion) and willing to comply with all study requirements

Exclusion Criteria:

- Female participant who is pregnant, lactating or planning pregnancy during the course
of the study

- Evidence of history of clinically significant hepatic disease

- An ALT or AST measurement > 2 times the ULN (Upper Limit of Normal)

- Scheduled elective surgery or other procedures requiring general anaesthesia during
the study

- Donation of blood during the study or within the past 4 weeks

- Treatment (during the study or within the past 4 weeks) with a prescription or
investigational drug for the treatment of TTR amyloidosis

- Treatment with NSAIDs (nonsteroidal antiinflammatory drug) during the study or within
the past 4 weeks. The following NSAID are allowed: acetylsalicylic acid, etodolac,
ibuprofen, indomethacin, ketoprofen, nabumetone, naproxen, nimesulide, piroxicam and
sulindac