Trial | NCT02191826  Report issue

Title

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

  • Phase

    Phase 1/Phase 2

  • Study Type

    Interventional

  • Status

    Completed No Results Posted

  • Intervention/Treatment

    tolcapone ...

  • Study Participants

    17
Clinical proof of concept study to evaluate SOM0226 efficacy in TTR Amyloidosis.
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 Started
Jul 31
2014
Primary Completion
Nov 30
2015
Study Completion
Nov 30
2015
Last Update
Nov 30
2016
Estimate

Drug SOM0226

Oral

SOM0226 single dose Experimental

SOM0226 multiple doses Experimental

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
No Results Posted