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

SOM Innovation Biotech SL (SOM Biotech)0 个研究点开始时间: 2014年6月2日最近更新: 2015年12月7日

适应症

试验速览

暂无简介。

•- 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

•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

发起方

SOM Innovation Biotech SL (SOM Biotech)