Status:
COMPLETED
Study of SOM0226 in Familial Amyloid Polyneuropathy
Lead Sponsor:
SOM Innovation Biotech SA
Collaborating Sponsors:
Hospital Vall d'Hebron
Conditions:
Familial Amyloid Polyneuropathy (FAP)
Eligibility:
All Genders
18+ years
Phase:
PHASE1
PHASE2
Brief Summary
Clinical proof of concept study to evaluate SOM0226 efficacy in TTR Amyloidosis.
Detailed 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 ...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
July 1 2014
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 1 2015
Estimated Enrollment :
17 Patients enrolled
Trial Details
Trial ID
NCT02191826
Start Date
July 1 2014
End Date
November 1 2015
Last Update
November 30 2016
Active Locations (1)
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1
Hospital Vall d'Hebron
Barcelona, Catalonia, Spain, 08035