Study of SOM0226 in Familial Amyloid Polyneuropathy

SOM Biotech

Status and phase

Completed

Phase 2

Phase 1

Conditions

Familial Amyloid Polyneuropathy (FAP)

Treatments

Drug: SOM0226

Study type

Interventional

Funder types

Other

Industry

Identifiers

NCT02191826

2014-001586-27 (EudraCT Number)

SOMCT01-C

Details and patient eligibility

About

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

Full 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

Enrollment

17 patients

Sex

All

Ages

18+ years old

Volunteers

Accepts Healthy Volunteers

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