Study of SOM0226 in familial amyloid polyneuropathy (FAP) patients and asymptomatic carriers to evaluate protein stabilization activity

Conditions: Patients with familial amyloid polyneuropathy (FAP), asymptomatic carriers and healthy volunteers
MedDRA version: 17.0Level: LLTClassification code 10057949Term: Familial amyloid polyneuropathySystem Organ Class: 100000004850
Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Registration Number: EUCTR2014-001586-27-ES

Lead Sponsor: SOM Innovation Biotech SL (SOM Biotech)

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: Not specified

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 time of consent.
- Two cohorts defined by TTR variant:
Wild type TTR: healthy patients
V30M TTR mutant: Asymptomatic carriers with documented
mutation V30M 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.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 20
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

The participant may not enter the study if ANY of the following apply:
- Female participants 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.
- Any other significant disease or disorder which, in the opinion of the Investigator, may either put the participants at risk because of participation in the study, or may influence the result of the study, or the participant?s ability to participate in 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 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